2023 January Quarterly Pipeline

Table of CONTENTS

EDITORIAL STAFF Maryam Tabatabai, PharmD Editor-in-Chief Vice President, Clinical Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist

EDITOR-IN-CHIEF'S MESSAGE

2

Consultant Panel

PIPELINE DEEP DIVE

3

Robert Greer, RPh, BCOP Vice President, Clinical Strategy and Programs

KEEP ON YOUR RADAR

26

PIPELINE DRUG LIST

27

GLOSSARY

47

Michelle Booth, PharmD Director, Specialty Clinical Solutions

Andrea Henry, PharmD, MBA, BCPS Specialty Drug Information Pharmacist Katie Lockhart Manager, Forecasting and Pharmacoeconomics Simone Ndujiuba, PharmD, BCOP Director, Clinical Strategy and Innovation, Oncology Olivia Pane, PharmD, CDCES Drug Information Pharmacist Nothing herein is or shall be construed as a promise or representation regarding past or future events and Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Magellan Rx Management.

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Editor-in-Chief's MESSAGE Welcome to the MRx Pipeline. This quarterly publication offers clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline.

MRx Pipeline, our universal forecast, addresses trends applicable across market segments. Traditional and specialty drugs as well as agents under the pharmacy and medical benefits are featured. Also profiled in the report are new molecular entities, pertinent new and expanded indications for existing medications, and biosimilars.

Clinical analyses, financial outlook, and pre-regulatory status are considered. The products housed in the MRx Pipeline have been researched in detail. They have been developed in consultation with our internal team of clinical and analytics experts.

METHODOLOGY

Emerging therapeutics continue to grow and influence the clinical and financial landscape. Therefore, Magellan Rx Management has developed a systematic approach to determine the products with significant clinical impact. For the in-depth clinical evaluations, the products’ potential to meet an underserved need in the market by becoming the new standard of care, and the ability to replace existing therapies were investigated. The extent to which the pipeline drugs could shift market share on a formulary and their impact on disease prevalence were also important considerations. In order to assist payers with assessing the potential impact of these pipeline drugs, where available, a financial forecast has been included for select products. Primarily complemented by data from EvaluateTM, this pipeline report looks ahead at the 5-year projected annual US sales through the year 2027. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, including the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business.

REFLECTION

In 2022, the US FDA approved 37 novel drugs. This is lower compared to the recent past, with the last 5 years averaging 51 novel approvals per year. Notably, over half (54%) of novel approvals in 2022 were for rare or Orphan diseases.

Moreover, 65% of novel approvals underwent at least one of the Agency’s expedited programs to speed approval for serious conditions, with 16% as Accelerated Approval.

While numbers do not tell the entire story, they do represent significant innovation in patient care and advance public health for the American public.

ON THE HORIZON

As we look ahead, there is a continued trend towards the approval of specialty medications and drugs for rare conditions, with 66% and 36% of approvals expected, respectively, for agents with applications submitted to the FDA. There are 4 agents seeking FDA’s Accelerated Approval, which allows for earlier drug approval for serious conditions that fill an unmet need based on a surrogate endpoint reasonably likely to predict a clinical benefit. The first Humira® biosimilar is anticipated to launch on January 31, 2023. Approval of 2 new vaccines and a biologic for RSV prophylaxis plus a gene therapy for hemophilia A are expected. Other noteworthy pipeline trends to watch include the development of complex therapies, cell and gene therapies, oncology, immunology, immunotherapy, and therapeutic options for ultrarare hereditary diseases. Moreover, sprouting products for obesity and menopause vasomotor symptoms are being actively monitored through MRx Pipeline.

The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Innovative agents that show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm.

Maryam Tabatabai, PharmD Editor-in-Chief, MRx Pipeline

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Pipeline DEEP DIVE Objective evidence-based methodology was used to identify the Deep Dive drugs in the upcoming quarters. This section features a clinical overview and explores the potential place in therapy for these agents. Moreover, it addresses their FDA approval timeline and 5-year financial forecast.

SPECIALTY

PRIORITY REVIEW

BREAKTHROUGH THERAPY

88%

27%

18%

BIOSIMILAR

ORPHAN DRUG

58%

24%

pecialty drug names appear in  S magenta throughout the publication.

MUSCULOSKELETAL

delandistrogene moxeparvovec IV Sarepta/Genentech PROPOSED INDICATIONS

Duchenne muscular dystrophy (DMD) in ambulant patients

CLINICAL OVERVIEW

Delandistrogene moxeparvovec is a gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of shortened, functional dystrophin protein.

An analysis from three phase 1 and 2 trials (ENDEAVOR, SRP-9001-101, and SRP-9001-102) included data from > 80 boys with DMD who were 3 to 7 years of age. Patients received a single IV infusion of delandistrogene moxeparvovec. The analysis reported positive results across multiple time points, including 1-, 2-, and 4-years after treatment, with a consistent safety profile.

The ongoing, multinational, double-blind, phase 3 EMBARK trial is evaluating the safety and efficacy of delandistrogene moxeparvovec in 125 ambulatory boys 4 to 7 years old with DMD. Patients are randomized 1:1 to receive a single IV infusion of delandistrogene moxeparvovec 1.33x1014 vg/kg or matching placebo.

At week 52, patients who were previously treated with placebo will receive delandistrogene moxeparvovec, and patients who were previously treated with delandistrogene moxeparvovec will receive placebo. The primary endpoint is the change in NorthStar Ambulatory Assessment (NSAA) total score from baseline to week 52. Top-line results are expected in late 2023.

PLACE IN THERAPY

DMD is a rare X-linked neuromuscular disorder characterized by progressive muscle degeneration and weakness. An estimated 400 to 600 males are born with DMD each year in the US. In DMD, gene mutations lead to a lack of functional dystrophin protein involved in maintaining muscle fiber integrity. Onset of DMD occurs between 3 to 5 years of age. Most boys affected lose the ability to walk by age 12. Moreover, death due to respiratory or cardiac failure typically occurs before age 30.

Historically, select corticosteroids (prednisone) have been used to treat DMD; deflazacort (Emflaza®) is the only steroid FDA-approved for this use. While corticosteroids have been the SOC in DMD to delay progression of muscle weakness and improve respiratory function, their use is associated with side effects such as weight gain, slowed growth trajectories, bone fractures, and cataracts. Corticosteroids may be used in combination with the disease-modifying antisense oligonucleotides casimersen (Amondys 45), eteplirsen (Exondys 51), golodirsen (Vyondys 53), and viltolarsen (Viltepso®), which target exon skipping gene alterations to produce functional dystrophin protein.

If approved, delandistrogene moxeparvovec will be the first gene transfer therapy indicated to treat boys affected by DMD. Positive responses for up to 4 years have been reported after a single IV infusion. Pfizer’s single-dose fordadistrogene movaparvovec adeno-associated virus (AAV) gene therapy is also in a phase 3 trial in ambulatory patients 4 to 7 years old with DMD; primary completion is expected in January 2024.

The oral agent ataluren, which enables production of full-length, functional dystrophin, is also in phase 3 trials. After multiple attempts for FDA approval, new data for ataluren released in mid-2022 reported a 21% slowing of DMD progression and significant improvement in 6MWT.

FDA APPROVAL TIMELINE May 29, 2023  Fast Track

 Orphan Drug

 Priority Review

 RPD

 seeking Accelerated Approval

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$0

$532

$992

$1,503

$1,920

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IMMUNOLOGY

mirikizumab IV, SC Eli Lilly PROPOSED INDICATIONS

Moderate to severe active ulcerative colitis (UC)

CLINICAL OVERVIEW

Mirikizumab is an immunoglobulin G4 (IgG4) monoclonal antibody directed against the p19 subunit of interleukin-23 (IL-23).

The 12-week, double-blind, parallel-arm, phase 3 LUCENT-1 trial evaluated induction therapy with mirikizumab in 1,281 adults with moderately to severely active UC who failed or had intolerance to corticosteroids, immunosuppressants, biologics, or tofacitinib. Patients were randomized 3:1 to mirikizumab or placebo. At 12 weeks, among 1,162 patients included in the efficacy analysis, mirikizumab led to a significantly greater rate of clinical remission (primary endpoint) compared to placebo (24.2% versus 13.3%, respectively; p=0.00006).

Mirikizumab also led to statistically significant improvement compared to placebo in all secondary endpoints, including modified Mayo score, endoscopic remission, symptomatic response, symptomatic remission, histologic remission, endoscopic response, bowel urgency, and fecal calprotectin, an indicator of intestinal inflammation. Among patients who responded to treatment in the LUCENT-1 trial, 716 enrolled in the doubleblind LUCENT-2 maintenance therapy trial. In this trial, patients were re-randomized to mirikizumab or placebo (withdrawal group). At week 40, clinical remission was maintained in 49.9% of those who continued mirikizumab and 25.1% of patients who were switched to placebo (p<0.001). Mirikizumab was well-tolerated during both trials.

In LUCENT-1, mirikizumab 300 mg IV every 4 weeks for 12 weeks was administered as induction therapy. In LUCENT-2, mirikizumab 200 mg SC every 4 weeks was administered as maintenance therapy.

PLACE IN THERAPY

UC is a chronic autoimmune disorder characterized by inflammation and ulceration of the large intestine mucosa. Disease onset typically peaks in early adulthood. If left untreated, UC follows a relapsing and remitting disease course. Approximately 15% of patients experience aggressive disease and may require colectomy within 5 to 10 years after diagnosis.

There are several DMTs from various drug classes available for long-term management of moderate to severe UC. These include injectable TNF-α inhibitors, adalimumab (Humira; SC), golimumab (Simponi®; SC), infliximab (Remicade®, biosimilars; IV), the anti-integrin agent vedolizumab (Entyvio®; IV), and the IL-12/23 antagonist ustekinumab (Stelara®; IV). Oral agents for UC include the Janus kinase (JAK) inhibitors tofacitinib (Xeljanz®/ Xeljanz XR®) and upadacitinib (Rinvoq®), and the sphingosine 1-phosphate receptor modulator ozanimod (Zeposia®). In general, most drugs that are successful for induction of remission are continued as maintenance therapy.

If approved, mirikizumab will be the first anti-IL23p19 treatment for UC. It could compete with the IL12/23 inhibitor ustekinumab (Stelara) which is the only other agent that blocks IL-23 approved for UC. While ustekinumab is administered IV for both induction and maintenance dosing, mirikizumab offers the convenience of SC maintenance dosing. Guselkumab (Tremfya®) also targets IL-23p19 and is currently in phase 3 trials for UC via IV and SC administration. The IV- or SC-administered IL-23 inhibitor risankizumabrzaa (Skyrizi®) is also in phase 3 trials for UC.

FDA APPROVAL TIMELINE February to March, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$147

$274

$325

$387

$442

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ONCOLOGY

nogapendekin alfa inbakicept intravesical Immunitybio PROPOSED INDICATIONS

Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) carcinoma in situ (CIS) with or without Ta or T1 disease

CLINICAL OVERVIEW

Nogapendekin alfa inbakicept (NAI) is an interleukin-15 (IL-15) superagonist complex comprised of an IL-15 mutant (IL-15N72D) bound to an IL-15 receptor α/IgG1 Fc fusion protein.

The safety and efficacy of NAI was evaluated in the open-label, phase 3 QUILT-3.032 study in 171 adults with BCG-unresponsive NMIBC. Cohorts A (n=84) and C (n=10) included patients with CIS disease with or without Ta/T1 papillary disease. Cohort B enrolled patients (n=77) with high-grade Ta/T1 papillary disease. Patients in cohorts A and B were treated with NAI plus BCG. Those in cohort C received NAI alone. In cohort A, after a median follow-up of 23.9 months, 71% of patients achieved a complete response (primary endpoint); the probability of avoiding a cystectomy was 89.2% and probability of disease-specific survival was 100%. In cohort B, the estimated rate of disease-free survival (DFS) at 12 months was 55.4% (primary endpoint) and the median DFS was 19.3 months. In cohort C, at a median follow-up of 7.9 months, NAI monotherapy led to a complete response (primary endpoint) in 2 patients (20%); one patient maintained complete response at 6 months. The most common TEAEs reported with NAI were dysuria, pollakiuria, and hematuria, which are adverse events commonly reported with BCG. NAI 400 µg was administered intravesically (bladder instillation) once weekly for 6 consecutive weeks during the induction treatment period. In cohorts A and B, patients also received BCG 50 mg intravesically weekly for 6 weeks.

PLACE IN THERAPY

Bladder cancer is the sixth most common cancer in the US. It is estimated that 81,180 new cases of bladder cancer and 17,100 deaths due to the condition occurred in 2022. The average age at diagnosis is 73 years.

NMIBC accounts for approximately 75% of all new cases.

The standard initial treatment for NMIBC is transurethral tumor resection. This is followed by intravesical BCG (preferred) or intravesical chemotherapy (e.g., gemcitabine, mitomycin) in patients at intermediate or high risk of recurrence or progression. Up to 75% of cases will develop tumor recurrence after BCG therapy, and 20% have disease progression within 5 years. Notably, Merck is the sole supplier of the TICE attenuated, live BCG strain in the US and a number of other countries, and supply constraints have impacted the availability of BCG live.

Priority for BCG induction is given to patients with high-risk NMIBC (cT1 high grade or CIS). Radical cystectomy is recommended in patients who do not respond to intravesical BCG therapy. In patients who wish to preserve their bladder or who are ineligible for cystectomy, nonsurgical options are limited to IV pembrolizumab (Keytruda®) and intravesical valrubicin (Valstar®). Both options are indicated in patients with BCG-unresponsive CIS bladder cancer. In December 2022, the FDA approved the gene therapy nadofaragene firadenovec-vncg (Adstiladrin®) for the treatment of high-risk BCG-unresponsive NMIBC with CIS, with or without papillary tumors. In clinical trials, it demonstrated a 51% complete response rate with a median duration of 9.7 months.

Nogapendekin alfa inbakicept is a first-in-class IL-15 superagonist. It acts as an activation and proliferation factor for natural killer (NK) cells as well as effector and memory T cells, which may overcome BCG therapy failure.

NAI works locally in the bladder with no systemic IL-15 levels detected. In the clinical trial, it led to complete responses ranging from 71% to 89.2% when administered in combination with BCG in patients who previously failed BCG. If approved, NAI will provide an important option for patients with BCG-unresponsive NMIBC.

FDA APPROVAL TIMELINE May 23, 2023

 Breakthrough Therapy

 Fast Track

FINANCIAL FORECAST (reported in millions)

The financial forecast for nogapendekin alfa inbakicept is not currently available.

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ENDOCRINOLOGY

palopegteriparatide SC Ascendis PROPOSED INDICATIONS Hypoparathyroidism in adults

CLINICAL OVERVIEW

Palopegteriparatide is a long-acting prodrug of parathyroid hormone (PTH 1-34). It is designed to restore physiological levels of PTH 1-34 over 24 hours.

The 26-week, double-blind, phase 3 PaTHway trial evaluated palopegteriparatide in 82 patients with chronic hypoparathyroidism. Patients were randomized 3:1 to palopegteriparatide or placebo. The primary endpoint of serum calcium levels in the normal range (8.3 to 10.6 mg/dL) and independence from conventional therapy (active vitamin D and > 600 mg/day of calcium supplements) was achieved by significantly more patients in the palopegteriparatide group (78.7%) compared to placebo (4.8%) (p<0.0001). Statistically significant reductions in secondary endpoints of disease-specific physical and cognitive symptoms were also reported with palopegteriparatide compared to placebo. Notably, at week 26, the majority of patients (95%) treated with palopegteriparatide discontinued conventional treatment. Palopegteriparatide was well-tolerated, with injection site reaction and headache as the most common TEAEs reported. A 3-year, open-label extension period of the PaTHway trial, in which all patients enrolled (n=79) receive palopegteriparatide, is ongoing. The phase 2, PaTH Forward trial demonstrated a durable response through week 110. A normalized serum calcium level was maintained and 93% of palopegteriparatide-treated patients were independent of conventional therapy at this time point. The study also demonstrated that palopegteriparatide restored skeletal bone mineral density (BMD) toward sex- and age-expected values.

In the phase 3 PaTHway trial, palopegteriparatide was initiated at a self-administered fixed dose of 18 mcg/day SC using a prefilled pen. It was individually titrated to an optimal dose over 10 weeks, followed by individualized dosing up to 16 weeks.

PLACE IN THERAPY

Hypoparathyroidism is characterized by a decrease in function of the parathyroid glands, leading to reduced levels of PTH. This results in low serum calcium and elevated phosphorous levels. Hypoparathyroidism is typically caused by injury (surgical, autoimmune) to the parathyroid glands. It also manifests in the condition known as DiGeorge syndrome in which parathyroid glands are absent at birth. Hypoparathyroidism leads to an increased risk of bone loss, cataracts, kidney stones, Parkinson’s disease, and in the instance of autoimmune hypoparathyroidism, to Addison disease and pernicious anemia. The estimated prevalence of hypoparathyroidism in the US is 37 per 100,000 person-years.

Treatment focuses on restoring normal calcium and mineral levels in the body and includes lifelong calcium and vitamin D (e.g., calcitriol, alfacalcidol) supplementation. Recombinant human PTH 1-84 (Natpara®) via daily SC injection is approved for select patients, but carries a boxed warning for risk of osteosarcoma and has only been available through a Special Use program since September of 2019, when it was recalled due to reports of rubber particulates in the cartridge. If approved, palopegteriparatide will be the only hormonal replacement therapy indicated to treat hypoparathyroidism. Notably, palopegteriparatide is now available through an Expanded Access Program outside of clinical trials to select adults with hypoparathyroidism without other adequate treatment alternatives.

FDA APPROVAL TIMELINE April 30, 2023

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$33

$161

$289

$409

$542

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ONCOLOGY

quizartinib oral Daiichi Sankyo PROPOSED INDICATIONS

Acute myeloid leukemia (AML) in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation

CLINICAL OVERVIEW

Quizartinib is a highly potent and selective inhibitor of type II FMS-like tyrosine kinase 3 (FLT3), a protein expressed by hematopoietic stem cells that plays a key role in the life cycle of the cell.

The ongoing, double-blind, phase 3 QuANTUM-First trial evaluated quizartinib in 539 adults ages 18 to 75 years with newly diagnosed FLT3-ITD positive AML, including those who underwent prior HSCT. Patients were randomized 1:1 to quizartinib or placebo given in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation for up to 36 cycles. After a median follow-up of 39.2 months, the addition of quizartinib to chemotherapy led to a 22.4% reduction in the risk of death (primary endpoint) compared to standard chemotherapy alone (OS, 31.9 versus 15.1 months, respectively; HR=0.776; p=0.0324). The rates for complete response and complete response without hematologic recovery were 71.6% and 64.9%, respectively, and relapse-free survival was longer with the addition of quizartinib than with chemotherapy alone (HR=0.733).

The TEAEs with quizartinib were generally manageable and consistent with chemotherapy alone.

Quizartinib 40 mg/day was administered orally on days 8 through 21 of induction/consolidation and 30 to 60 mg/day was administered during continuation therapy for up to 3 years.

PLACE IN THERAPY

AML is a cancer involving the blood and bone marrow. It is the most common type of acute leukemia in adults, with an average age at diagnosis of 68 years. It is estimated that 20,050 new cases of AML and 11,540 deaths from the condition occurred in the US in 2022.

The FLT3 gene is involved in hematopoiesis. FLT3 mutations with internal tandem duplications (ITD) occur in approximately 30% of cases and are associated with shorter remissions and poorer survival outcomes (median OS, 6 to 12 months). Chemotherapy induction regimens in patients ages < 60 years with FLT3 mutation include standard dose cytarabine with daunorubicin and oral midostaurin. This regimen is followed by consolidation therapy with high-dose intermittent cytarabine with oral midostaurin. In those ≥ 60 years of age, venetoclax-based therapy (with azacitidine or decitabine) is preferred for induction followed by cytarabine with daunorubicin and or midostaurin. Post-induction therapy includes azacitidine or decitabine plus sorafenib. Recommended maintenance therapy in those with FLT3 mutation, regardless of age, is sorafenib.

If approved, quizartinib could compete with oral midostaurin (Rydapt®) for the treatment of newly diagnosed, FLT3 mutation-positive AML in combination with standard cytarabine and daunorubicin induction and consolidation therapy. In the phase 3 RATIFY trial (NCT00651261), midostaurin plus standard chemotherapy resulted in a similar improvement in OS compared with chemotherapy alone (HR, 0.77). In 2019, Daiichi Sankyo received a complete response letter (CRL) from the FDA for quizartinib for the treatment of R/R AML after the FDA’s Advisory Panel voted 8 to 3 against approval; the panel stated that quizartinib did not definitively demonstrate benefit that outweighed its potential risks.

FDA APPROVAL TIMELINE April 24, 2023

 Breakthrough Therapy

 Fast Track

 Orphan Drug

FINANCIAL FORECAST (reported in millions)

The financial forecast for quizartinib is not currently available.

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 Priority Review

INFECTIOUS DISEASE

respiratory syncytial virus (RSV) vaccines GSK3844766A IM GlaxoSmithKline PROPOSED INDICATIONS

RSV prevention in older adults (ages ≥ 60 years)

CLINICAL OVERVIEW

GSK3844766A (also known as RSVpreF3 OA) is a recombinant vaccine that contains a proprietary adjuvant system, which has been approved for use in other vaccines, including against shingles (Shingrix®) and malaria (Mosquirix®).

The safety and efficacy of GSK3844766A was evaluated in the ongoing multinational, randomized, observerblind, placebo-controlled, phase 3 AReSVi-006 trial in 26,665 adults ≥ 60 years of age. In part 1 of the study, GSK3844766A was administered as a single IM injection into the deltoid muscle prior to the RSV season. The study demonstrated the vaccine produced an overall vaccine efficacy (VE), the primary endpoint, of 82.6% against RSV lower respiratory tract disease (RSV-LRTD). In addition, the VE against severe RSV-LRTD, defined as presence of ≥ 2 lower respiratory signs or assessed as severe by the investigator, was 94.1%. Among select populations, the VE against RSV-LRTD was 94.6% in those with pre-existing comorbidities and 93.8% in those ages 70 to 79 years. Efficacy was similar and robust against both the RSV A and RSV B strains. GSK3844766A was well tolerated. In part 2 of the ongoing study, individuals will receive annual revaccination prior to each subsequent RSV season.

FDA APPROVAL TIMELINE May 3, 2023  Fast Track

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$68

$254

$479

$772

$1,007

PF-06928316 injectable Pfizer PROPOSED INDICATIONS

RSV prevention in older adults (ages ≥ 60 years)

CLINICAL OVERVIEW

PF-06928316 (also known as RSVpreF) is a recombinant bivalent prefusion F-based vaccine that contains equal parts RSV types A and B. The safety and efficacy of PF-06928316 was evaluated in the multinational, randomized, double-blind, placebo-controlled, phase 3 RENOIR trial in 37,630 adults ≥ 60 years of age. Interim data demonstrated an overall VE of 66.7% against RSV-lower respiratory tract infection (RSV-LRTI) (defined by ≥ 2 symptoms) and a VE of 85.7% against severe RSV-LRTI (defined by ≥ 3 symptoms). The vaccine was well tolerated. No safety concerns were observed.

PF-06928316 was administered as a single 120 µg injection.

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respiratory syncytial virus (RSV) vaccines (cont.)

PF-06928316 (cont.)

FDA APPROVAL TIMELINE May 2023

 Breakthrough Therapy

 Fast Track

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$8

$163

$208

$312

$438

PLACE IN THERAPY

RSV is a common contagious respiratory virus that usually causes mild, cold-like symptoms lasting 1 to 2 weeks. However, RSV can cause serious illness, particularly in infants and older adults in whom bronchiolitis and pneumonia can occur and may require hospitalization. Older adults are at higher risk of serious illness due to weakened immunity and underlying cardiac or pulmonary conditions. In the US, RSV causes 60,000 to 120,000 hospitalizations and 6,000 to 10,000 deaths each year among adults ages ≥ 65 years. Typically, the RSV season in the US occurs during approximately November through April but may vary by region. However, since the start of the COVID-19 pandemic the regular RSV circulation pattern has been disrupted and began to rise during the Spring of 2021.

There is no agent FDA-approved to treat RSV infection. Treatment consists of symptom management, including antipyretics and analgesics. Palivizumab (Synagis®), an RSV F protein inhibitor monoclonal antibody, is the only agent FDA-approved for the prevention of RSV. It is indicated for use only in infants and young children.

No vaccine has been FDA-approved for the prevention of RSV. If approved, GlaxoSmithKline’s RSV vaccine GSK3844766A could be the first vaccine in the US available to prevent lower respiratory tract disease due to RSV infection in adults ≥ 60 years of age, closely followed

by Pfizer’s PF-06928316 vaccine. Both are recombinant vaccines containing a subunit prefusion RSV F glycoprotein (RSVpref) antigen and target the viral fusion protein that the virus uses to enter the host cell; GlaxoSmithKline’s product also contains an adjuvant to boost its activity. Both vaccines are expected to require repeat dosing prior to each RSV season. RSV vaccines by Bavarian, Janssen, Novavax, and Moderna (mRNA) for use in older adults are also in phase 3 trials.

Notably, on the pediatric RSV pipeline front, PF-06928316 is in phase 3 investigation (MATISSE trial) for maternal administration during pregnancy to protect the infant from RSV after birth. An interim analysis reports favorable safety and efficacy (VE, 81.8% and 69.4% through first 3 and 6 months of life, respectively).

PF-06928316 has the potential to be the first RSV vaccine approved to protect infants from RSV-LRTI. Novavax’s RSV vaccine is also in phase 3 research for maternal administration. In addition, AstraZeneca submitted a BLA for the single-dose monoclonal antibody nirsevimab for prevention of RSV-LRTI in children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season. The FDA decision is expected in Q3, 2023. Merck’s monoclonal antibody clesrovimab is also in a phase 3 trial for use in patients < 1 year of age who are at increased risk for severe RSV.

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IMMUNOLOGY

ritlecitinib oral Pfizer PROPOSED INDICATIONS

Alopecia areata in patients ages ≥ 12 years

CLINICAL OVERVIEW

Ritlecitinib is a dual inhibitor of Janus kinase (JAK) 3 and the tyrosine kinase expressed in hepatocelluar carcinoma (TEC) family of tyrosine kinases. TEC proteins play a key role in T-cell-receptor signaling.

The double-blind, phase 2b/3, dose-finding ALLEGRO trial evaluated the safety and efficacy of ritlecitinib in 718 patients ≥ 12 years of age with alopecia areata, including alopecia totalis and alopecia universalis.

Patients had ≥ 50% scalp hair loss, based on the Severity of Alopecia Tool (SALT) score (≥ 50%). Patients were excluded if they had other types of alopecia or other diseases that can cause hair loss. Patients were randomized to oral once daily ritlecitinib 50 mg or 30 mg (with or without 4 weeks of initial treatment with ritlecitinib 200 mg once daily), ritlecitinib 10 mg once daily, or placebo, across 7 study arms. After 24 weeks, significantly more patients treated with ritlecitinib 30 mg or 50 mg (with or without the loading dose) compared to those given placebo experienced the primary endpoint of hair regrowth, as indicated by a SALT score of ≤ 20%. The response was dose-dependent, ranging from 14.3% with ritlecitinib 30 mg with no loading dose to 30.7% with ritlecitinib 50 mg with a loading dose, compared to 1.54% with placebo (p<0.000001 for each compared to placebo). The ritlecitinib 10 mg dose was included for dose ranging purposes only and did not provide meaningful benefit. At week 48, durable dose-dependent responses (SALT ≤ 20%) were seen in 31.2% to 39.5% of patients who received 30 mg or 50 mg (with or without loading doses), respectively. The most common TEAEs were nasopharyngitis, headache, and upper respiratory tract infection. Among patients treated with ritlecitinib, 8 patients developed shingles, 1 patient developed pulmonary embolism, and 2 patients developed breast cancer. No deaths, MACE, or opportunistic infections were reported.

PLACE IN THERAPY

Alopecia areata is an autoimmune condition that attacks hair follicles causing hair loss. Patchy baldness can develop anywhere on the scalp, face, and body. Onset typically occurs during childhood, adolescence, or during the patient's 20s or 30s. Approximately half of individuals experience hair regrowth within a few months without treatment. Alopecia may reoccur with unpredictable cycles. Pharmacotherapies for alopecia areata include corticosteroids, immunosuppressants, and agents that stimulate hair regrowth. In June 2022, the JAK inhibitor baricitinib (Olumiant®) received FDA approval as the first medication to treat alopecia areata in adults. Baricitinib carries a boxed warning for serious infections, mortality, malignancy, MACE, and thrombosis.

If approved, ritlecitinib will be the first-in-class of agents that inhibit both JAK3 and TEC tyrosine kinases. It could compete with baricitinib to treat alopecia areata in adults; however, ritlecitinib will be the only agent approved for use in adolescents in this setting. While ritlecitinib has high selectivity for JAK3, baricitinib has greater inhibitory potency for JAK1 and JAK2. Both agents are administered orally once daily. In noncomparison trials, ritlecitinib and baricitinib produced similar dose-dependent hair regrowth; for baricitinib, SALT score ≤ 20% at week 36 occurred in 17% to 35% of patients. Unlike baricitinib, serious events such as death, MACE, and serious infections were not reported with ritlecitinib. Ritlecitinib is also in phase 2 trials for vitiligo, RA, CD, and UC.

FDA APPROVAL TIMELINE April to June 2023

 Breakthrough Therapy

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$25

$73

$87

$133

$155

* Forecasted sales reported for all investigational indications

11 | MAGELLANRX.COM

INFECTIOUS DISEASE

SER-109 oral Seres PROPOSED INDICATIONS

Prevention of recurrent Clostridioides difficile-associated diarrhea/infection (CDI)

CLINICAL OVERVIEW

SER-109 is an oral live microbiome therapeutic product that consists of purified Firmicutes spores, which are typically found in a healthy microbiome. Stool donations are from healthy, screened individuals.

The double-blind, placebo-controlled, phase 3 ECOSPOR-III study evaluated SER-109 in adults with recurrent CDI (≥ 3 CDI episodes within 12 months). The 182 enrolled patients had ≥ 3 unformed bowel movements over 2 consecutive days, a positive C. difficile stool toxin test, and had responded to SOC antibiotic treatment (vancomycin and/or fidaxomicin). Patients were randomized 1:1 to SER-109 or placebo and were monitored for 8 weeks for recurrence. SER-109 demonstrated superiority over placebo, as it reduced the risk of CDI recurrence (primary endpoint) by 68% (12% versus 40%, respectively; p<0.001). In addition, response was sustained through week 8 in 88% of patients treated with SER-109 compared with 60% who received placebo. No serious adverse events were reported. The most common adverse events were GI in nature and of mild to moderate severity, and occurred at similar rates in both groups. The open-label extension ECOSPOR-IV study (n=263) included patients from ECOSPOR-III and additional patients with only 1 prior CDI recurrence. The study also included patients with multiple comorbidities (e.g., CVD, tumors, diabetes, COPD, CKD). It reported similar findings, with a 91% sustained clinical response at 8 weeks and 86% at 24 weeks.

SER-109, at an approximate dose of 3 x 107 spore colony-forming units, was administered orally as 4 capsules once daily for 3 consecutive days.

PLACE IN THERAPY

C. difficile is a bacterium that causes diarrhea and colitis. Nearly 500,000 cases of CDI are reported in the US each year. CDI typically occurs during or shortly after a course of antibiotic therapy. Risk factors include age ≥ 65 years, recent hospital or nursing home stay, immunocompromise, and previous CDI. Recurrence of CDI is reported in approximately 15% to 30% of patients who initially respond to therapy; the rates of second and subsequent recurrences are 40% and 45% to 65%, respectively. It is estimated that 15,000 to 30,000 deaths each years in the US are attributed to CDI. The IDSA recommends proper antibiotic stewardship to control CDI rates based on the local epidemiology and the C. difficile strains present. Appropriate restriction of fluoroquinolones, clindamycin, and cephalosporins should be considered, and use of the inciting antibiotic agent(s) should be discontinued as soon as possible.

Data are insufficient to recommend use of probiotics for primary prevention of CDI. Oral vancomycin (Firvanq®, Vancocin®, generics) or fidaxomicin (Dificid®) is strongly recommended for the treatment of initial and recurrent CDI episodes in adults. Fecal microbiota transplantation is strongly recommended in patients who experience multiple recurrences and fail appropriate antibiotic treatments. The monoclonal antibody bezlotoxumab (Zinplava™) is indicated to reduce recurrence of CDI in adults on antibiotic CDI therapy who are at high risk for recurrence.

SER-109 alters the disrupted microbiome of the GI tract to resist growth of C. difficile. If approved, it will be the first oral live microbiome therapy approved in the US to prevent recurrent CDI; this follows the November 2022 approval of Ferring's rectally-administered live microbiota preparation (Rebyota®).

FDA APPROVAL TIMELINE April 26, 2023

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$15

$38

$72

$110

$150

12 | MAGELLANRX.COM

ENDOCRINOLOGY

sotagliflozin oral Lexicon PROPOSED INDICATIONS

Heart failure (HF) » To reduce the risk of CV death, hospitalization for HF, and urgent HF visit in adults with HF, including those with acute or worsening HF » To reduce the risk of CV death, hospitalization for HF, urgent HF visit, nonfatal MI, and nonfatal stroke in adults with T2DM, CKD, and other CV risk factors, including a history of HF

CLINICAL OVERVIEW

Sotagliflozin is a dual inhibitor of sodium-glucose co-transporter (SGLT) types 1 and 2. SGLT1 is responsible for glucose absorption in the GI tract and SGLT2 mediates glucose reabsorption by the kidneys.

The double-blind, phase 3 SCORED trial enrolled 10,584 adult patients with T2DM, CKD, and other CV risk factors. Patients were randomized 1:1 to sotagliflozin or placebo, both added to SOC. After a median of 16 months, the rate of the composite primary endpoint of the total number of deaths from CV causes, hospitalizations for HF, and urgent visits for HF was significantly less with sotagliflozin (5.6 events/100 patient-years) compared to placebo (7.5 events/100 patients-years) (HR, 0.74; p<0.001). This was primarily driven by a reduction in hospitalizations and urgent visits for HF (HR 0.67; p<0.001); there was no significant difference in the rate of CV death (p=0.35).

The double-blind, phase 3 SOLOIST-WHF trial enrolled 1,222 adults with T2DM who had recently been hospitalized for worsening HF. Patients were randomized 1:1 to sotagliflozin or placebo; both were added to SOC. After a median of 9 months, the primary endpoint of total number of deaths from CV causes, hospitalizations for HF, and urgent visits for HF was significantly lower with sotagliflozin than placebo (51 versus 76.3 events/100 patient-years; HR, 0.67; p<0.001). The rate of hospitalizations and urgent visits for HF was significantly lower with sotagliflozin (HR, 64; p<0.001). The rate of CV death was numerically lower with sotagliflozin compared to placebo, but not to a significant level (10.6 versus 12.5 total events; HR, 0.84; p=0.36). Adverse events reported more often with sotagliflozin than placebo were diarrhea, genital mycotic infections, volume depletion, and diabetic ketoacidosis.

In both trials, sotagliflozin was initiated at a dosage of 200 mg orally once daily and, if tolerated, increased to 400 mg orally once daily.

PLACE IN THERAPY

An estimated 6.2 million adults in the US suffer from HF. It occurs when the heart fails to pump blood at an adequate rate to support the organs of the body. A wide range of pharmacotherapies are used for the management of HF. While several mechanisms have been proposed, the HF benefit that SGLT2 inhibitors provide appears to be independent of glucose lowering. The AHA, ACC, and HFSA recommend the use of SGLT2 inhibitors in patients with T2DM and either established CVD or at high CV risk to prevent hospitalization for HF. SGLT2 inhibitors are also recommended in patients with HF, with or without T2DM, to reduce hospitalization and CV mortality. SGLT2 inhibitors already approved to reduce the risk of CV death and hospitalization due to HF include canagliflozin (Invokana® and Invokamet®/XR-in patients with T2DM only), dapagliflozin (Farxiga®, Xigduo® XR), and empagliflozin (Jardiance®). If approved, sotagliflozin will be the first agent to target both SGLT1 and SGLT2 for use in this setting. Whether additional CV benefit is provided from SGLT1 inhibition beyond that provided by SGLT2 inhibition has not been established.

FDA APPROVAL TIMELINE May 31, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales*

$134

$267

$460

$626

$748

*Financial data reflected for all investigational indications combined.

13 | MAGELLANRX.COM

NEUROLOGY

tofersen intrathecal Biogen PROPOSED INDICATIONS

Amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations

CLINICAL OVERVIEW

Tofersen is an antisense oligonucleotide designed to inhibit the production of the SOD1 protein.

The efficacy and safety of tofersen were evaluated in the 28-week, double-blind, placebo-controlled, phase 3 VALOR trial (n=108) that randomly assigned patients 2:1 to tofersen or placebo, and in the ongoing, open-label extension (OLE) study (n=95). In the OLE trial, all patients received tofersen, and the study compared earlystart with delayed-start of tofersen. All patients enrolled in the studies had ALS with a SOD1 mutation. Among patients in VALOR, 60 were predicted to have ALS with faster progression. At 28 weeks, treatment with tofersen resulted in greater reductions in SOD1 concentrations in the cerebrospinal fluid (CSF) and neurofilament light chains levels in plasma (both secondary endpoints) compared to placebo; this was maintained through week 52 of the OLE trial. Approximately, 33% and 51% of reductions in SOD1 and neurofilament light chains, respectively, were measured after 52 weeks of tofersen treatment. While improvements in these 2 biomarkers were observed, it did not correspond to a significant difference between the tofersen and placebo groups in the primary endpoint of total ALS Functional Rating Scale–Revised score (ALSFRS-R; range, 0 to 48). At 28 weeks, among patients predicted to have faster progression, the primary analysis subgroup, the change from baseline in ALSFRS-R for tofersen and placebo was -6.98 and -8.14 points, respectively (p=0.97). At 52 weeks, the change from the VALOR baseline in the ALSFRS-R total score was -6 points in patients originally randomized to tofersen (early-start cohort) and -9.5 points among those originally randomized to placebo (delayed-start cohort). The median time to death or permanent ventilation could not be estimated due to the small number of events. During the 52 weeks, serious adverse events were reported in 37% of tofersentreated patients. These included (≥ 5%) neurologic adverse events (e.g., myelitis, meningitis, radiculopathy, increased intracranial pressure, papilledema), respiratory failure, and pneumonia aspiration.

Tofersen 100 mg was administered intrathecally over 24 weeks, as 3 doses once every 2 weeks, followed by 5 doses once every 4 weeks.

PLACE IN THERAPY

ALS is a rare, progressive motor neuron disease characterized by voluntary muscle weakness, atrophy, and paralysis. Eventually respiratory failure and premature death occur. The age at diagnosis is typically between 55 to 75 years. Once symptoms develop, life expectancy is 2 to 5 years. It is estimated that 31,000 people in the US have ALS, with approximately 5,000 new cases occurring each year. ALS occurs at similar rates in men and women. The exact cause of ALS is unknown. About 5% to 10% of cases appear to be inherited, and 12% to 20% of familial cases are due to SOD1 gene mutations.

There is no cure for ALS. FDA-approved DMTs include oral riluzole (generic, Exservan™, Rilutek®, Tiglutik®) and sodium phenylbutyrate/taurursodiol (Relyvrio™), and IV-administered edaravone (Radicava®). If approved, tofersen will be the first SOD1-directed DMT for ALS. In clinical trials, it reduced SOD1 concentrations in the CSF, an indirect marker of target engagement. It also reduced neurofilament light chains in the plasma, an indicator of axonal injury and neurodegeneration. While tofersen was associated with a smaller numeric decline in the ALSFRS-R score compared to placebo, the difference was not significant. The benefit of earlier versus delayed treatment continues to be assessed in the OLE trial. Evaluation of tofersen in presymptomatic SOD1 ALS is also underway.

FDA APPROVAL TIMELINE April 25, 2023

 Orphan Drug

 Priority Review

 seeking Accelerated Approval

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1

$2

$3

$4

$4

14 | MAGELLANRX.COM

NEUROLOGY

trofinetide oral Acadia PROPOSED INDICATIONS Rett syndrome

CLINICAL OVERVIEW

Trofinetide is a synthetic analog of the amino‐terminal tripeptide of insulin-like growth factor 1 (IGF-1) that has the potential to stimulate synaptic maturation and overcome the synaptic and neuronal immaturities that are observed with Rett syndrome. This may lead to reduced neuroinflammation and support of synaptic function.

The randomized, double-blind, placebo-controlled, phase 3 LAVENDER trial enrolled 187 females ages 5 to 20 years diagnosed with classic Rett syndrome. After 12 weeks, trofinetide significancy decreased the coprimary endpoint of the caregiver-rated Rett Syndrome Behavior Questionnaire (RSBQ) score compared to placebo (−5.1 versus −1.7, respectively; p=0.0175). The other primary endpoint of Clinical Global Impression of Improvement (CGI-I) was also significantly improved with trofinetide (mean, 3.5 versus 3.8, respectively; p=0.003). Trofinetide also resulted in less decline compared to placebo in the key secondary endpoint of Communication and Symbolic Behavior Scales Developmental Profile Infant-Toddler Checklist–Social composite score (−0.1 versus −1.1, respectively; p=0.0064). Diarrhea was reported in 80.6% of patients treated with trofinetide and led to treatment discontinuation in 17.2% of patients in this group. The majority of patients (95%) continued treatment in the 40-week open-label LILAC extension study; results are pending.

Trofinetide oral solution was administered as a weight-based dose twice daily by mouth or gastrostomy tube (G-tube).

PLACE IN THERAPY

Rett syndrome is a rare progressive neurodevelopmental disorder that is seen almost exclusively in females.

In ≥ 90% of cases, a sporadic (non-inherited) mutation of the MECP2 gene, located on the X chromosome and is integral for brain development, is detected. Boys with this gene mutation typically die shortly after birth.

Classic Rett syndrome is characterized by normal early growth and development, but after 6 to 18 months of age affected children experience a slowing of development, loss of purposeful use of the hands, distinctive hand movements (e.g., wringing, clapping, rubbing), slowed brain and head growth, difficulty walking, seizures, and intellectual disability. The estimated incidence of Rett syndrome in the US is 1 in 10,000 girls by age 12.

The average life expectancy is 24 years; however, patients can live into their 40s or 50s with proper care, but suffer severe disability.

There is no cure for Rett syndrome. Therapy focuses on symptom management and support of physical functioning. If approved, trofinetide will be the first DMT available to treat the condition. In short-term (12week) clinical trials, it demonstrated significant benefit based on behavioral and clinical measures. Longterm data is pending. Avavex’s sigma-1 receptor agonist, blarcamesine, is also in phase 3 development for neurodegenerative and neurodevelopmental disorders, including Rett syndrome in adults. Topline data reported significant improvement in behavioral and clinical measures.

FDA APPROVAL TIMELINE March 12, 2023  Fast Track

 Orphan Drug

 Priority Review

 RPD

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$29

$76

$131

$187

$230

15 | MAGELLANRX.COM

HEMATOLOGY

valoctocogene roxaparvovec IV Biomarin PROPOSED INDICATIONS Severe hemophilia A

CLINICAL OVERVIEW

Valoctocogene roxaparvovec (val-rox) is an adeno-associated virus (AAV) vector gene therapy for the treatment of severe hemophilia A in adults.

The open-label, single-group, phase 3 GENEr8-1 study evaluated val-rox in adult males with severe hemophilia A (factor VIII level ≤ 1 IU/dL). Patients were receiving prophylactic factor VIII therapy for ≥ 1 year and were negative for factor VIII inhibitors. A total of 132 patients received a dose of val-rox 6×1013 vector genomes/kg of body weight and were HIV-negative. At weeks 49 to 52 after the val-rox infusion, significant changes from baseline were reported in factor VIII activity, the primary efficacy endpoint; mean and median increases from baseline were 41.9 IU/dL (p<0.001) and 22.9 IU/dL, respectively. At weeks 49 through 52, a median factor VIII activity level ≥ 40 IU/dL (equivalent to nonhemophilic) was reported in 37.9% of patients, levels between 5 and 40 IU/dL (equivalent to mild hemophilia) were reported in 50% of patients, and levels < 5 IU/dL were reported in 12.1% of patients. Among 112 patients enrolled from a prospective noninterventional study, the mean annualized factor VIII concentrate use decreased by 98.6% and treated bleeding decreased by 83.8% (p<0.001 for both). The most common adverse events were ALT elevations (85.8%), headache (38.1%), nausea (37.3%), and AST elevations (35.1%).

In addition, an ongoing open-label, phase 1/2 study demonstrated sustained hemostatic efficacy for 5 and 6 years after administration of val-rox at doses of 4×1013 and 6×1013 vector genomes/kg, respectively. Until the time of data cutoff, all but 1 patient remained off factor VIII treatment (the 1 patient resumed factor VIII for 1 month).

PLACE IN THERAPY

Hemophilia A is a congenital X-linked bleeding disorder that affects 1 in 5,000 male births. It is characterized by coagulation factor VIII deficiency leading to chronic spontaneous bleeding into muscles and joints that can progress to debilitating arthropathy.

The SOC for hemophilia A is routine infusion of factor VIII replacement, including extended half-life products.

A major advancement in the hemophilia space is the SC monoclonal antibody emicizumab-kxwh (Hemlibra®), which provides routine prophylaxis in hemophilia A patients with or without inhibitors using a once every 1-, 2-, or 4-week dosing regimen.

If approved, val-rox will be the first gene therapy in the US for the treatment of severe hemophilia A. In clinical trials, it resulted in near elimination of bleeds with a single dose, with efficacy demonstrated for up to 6 years. Other products in phase 3 development for hemophilia A include the AAV gene therapies giroctocogene fitelparvovec (Pfizer) and SPK-8011 (Spark), and the bispecific monoclonal antibody Mim8 (Novo Nordisk/Genmab).

FDA APPROVAL TIMELINE March 31, 2023

 Breakthrough Therapy

 Orphan Drug

 RMAT

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$139

$292

$449

$614

$751

16 | MAGELLANRX.COM

METABOLIC

velmanase alfa IV Chiesi PROPOSED INDICATIONS Alpha-mannosidosis

CLINICAL OVERVIEW

Velmanase alfa (VA) is a recombinant human alpha-mannosidase intended as an enzyme replacement therapy (ERT).

The 52-week, double-blind, phase 3 rhLAMAN-05 trial evaluated VA in 25 patients ages 5 to 35 years old, with a confirmed diagnosis of alpha-mannosidase as defined by alpha-mannosidase activity < 10% of normal activity in blood leukocytes. Eligible patients were able to walk without support. The study was conducted in 5 countries across Europe. Patients were randomized 3:2 to VA or placebo. After 52 weeks, patients either continued receiving VA or switched from placebo to VA. The coprimary endpoints were change from baseline to week 52 in serum oligosaccharides and the 3-minute stair climb test (3MSCT). At week 52, the mean relative change in serum oligosaccharide concentration was significantly greater with VA than with placebo (-77.6% versus -24.1%, respectively; adjusted mean difference, -70.5%; p<0.001). At the last observation (long-term outcome), the mean relative change from baseline was -62.9% with the VA group and -55.7% in the placebo-VA switch group. At week 52, there was no significant change in the 3MSCT from baseline with VA compared to placebo (mean change, -1.1% versus -0.0%, respectively; adjusted mean difference, +3; p=0.648). At the last observation timepoint, the mean relative change was +3.9% in the VA group and +9% in the placebo-VA switch group. In addition, a small increase in the secondary endpoint of change from baseline in 6MWT at week 52 was seen in the VA group compared with a small decline in the placebo group; the difference was not significant. Five serious TEAEs were reported, one of which was considered related to VA in a patient who received long-term ibuprofen who experienced acute renal failure; the patient recovered after VA interruption and was able to restart therapy without incident.

The single-center, open-label, long-term (up to 4 years) phase 3 rhLAMAN-10 trial assessed VA in 33 patients (14 adults, 19 pediatrics) with confirmed alpha-mannosidosis who had previously participated in phase 1/2 and phase 3 trials. The coprimary endpoint of serum oligosaccharide level was significantly reduced in the overall population at 12 months (mean change, -72.7%; p<0.001) which was reported through the last observation timepoint (mean change, -62.8%; p<0.001). An improvement in the other coprimary endpoint of change from baseline in 3MSCT was also observed at 12 months (mean change, +9.3%; p=0.013) and continued through the last observation (mean change, +13.8%; p= 0.004).

VA was administered via IV infusion once weekly at a dose of 1 mg/kg of body weight.

PLACE IN THERAPY

Alpha-mannosidosis is a rare genetic disorder caused by a mutation in the MAN2B1 gene that is responsible for the production of lysosomal alpha-mannosidase. Lysosomal enzymes, such as alpha-mannosidase, breakdown complex carbohydrates (oligosaccharides) in cell membranes. A deficiency in functional alphamannosidase results in mannose accumulation leading to tissue and organ damage. The global prevalence of alpha-mannosidosis is estimated to be 1 in every 500,000 individuals. Those affected by alpha-mannosidosis may experience hydrocephalus, intellectual disability, ataxia, myopathy, distinctive facial features, and skeletal abnormalities, as well as other complications. Manifestations of the condition can range from mild to severe. Three types of alpha-mannosidosis are identified. Type 1, the mildest form, progresses slowly and may appear during the teenage years. Type 2 is moderate in severity, may appear prior to 10 years of age, and progresses slowly. Type 3 is severe, may occur during infancy, with rapid progression and death during childhood. With milder forms of the disease, patients may survive through middle age (50s).

There are no FDA-approved medications to treat alpha-mannosidosis. Current therapy is supportive in nature and focuses on symptom management and prevention of complications. If approved, VA will be the first and only DMT available to treat alpha-mannosidosis. In clinical trials, it demonstrated a marked reduction in serum oligosaccharide level and mixed results regarding the effect on 3MSCT. Positive changes in endurance and pulmonary function with VA, particularly in pediatric patients, might suggest that greater clinical benefits could be achieved with initiation of therapy early in the disease course.

17 | MAGELLANRX.COM

velmanase alfa (cont.)

FDA APPROVAL TIMELINE January to June 2023  Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions)

The financial forecast for velmanase alfa is not currently available.

18 | MAGELLANRX.COM

Biosimilar Overview CLINICAL OVERVIEW

Biosimilars are very different from generic drugs in that they are not exact duplicates of their reference biologic product. The FDA approval process for biosimilars is designed to ensure that the biosimilar product is highly similar to the reference product without having any meaningful clinical differences. Moreover, an interchangeable biological product is a biosimilar that is expected to produce the same clinical result as the reference product in any given patient. Switching or alternating between the reference and interchangeable products should have been evaluated and should not negatively impact the safety and efficacy of therapy.

Many controversies have surrounded biosimilars, and regulatory and litigation hurdles remain. The FDA has issued final and draft guidances. Select FDA biosimilar guidances are noted here. In January 2017, the agency issued final guidance on the nonproprietary naming of biologic products, which also applies to biosimilars.

The biological products must bear a core name followed by a distinguishing 4-letter, lowercase, hyphenated suffix that is devoid of meaning. The international nonproprietary name (INN) impacts interchangeability as it affects pharmacists’ ability to substitute an interchangeable biosimilar for the reference product.

The FDA withdrew the September 2017 draft industry guidance on determining similarity of a proposed biosimilar product to its reference product to allow for further consideration of the most current and relevant scientific methods in evaluating analytical data. The agency focuses on providing flexibility for the efficient development of biosimilars while maintaining high scientific standards. In July 2018, the FDA finalized its guidance on labeling biosimilars. The guidance pertains to prescribing information (PI) but does not contain specific recommendations on interchangeability in the labeling. The labeling guidance provides recommendations on how to include, identify, and differentiate the biosimilar and the reference product in various sections of the PI. The basic premise remains that the originator product’s safety and effectiveness can be relied upon for HCPs to make prescribing decisions; therefore, a biosimilar should include relevant data from the originator in its PI. In May 2019, the agency released its final guidance on interchangeability.

Most states have enacted biosimilar substitution laws. An interchangeable product may be substituted for the originator at the pharmacy without the involvement of the prescriber. In December 2022, the FDA announced a pilot regulatory science program that focuses on advancing the development of interchangeable products and improving the efficiency of biosimilar product development. The Purple Book is an FDA database of licensed biological products that lists biosimilar and interchangeable products. The FDA has approved 3 biosimilars for interchangeability to their reference product: insulin glargine-yfgn (Semglee®), adalimumabadbm (Cyltezo®), and ranibizumab-eqrn (Cimerli™).

Biosimilars can receive extrapolation to gain an indication without direct trials of the biosimilar for the eligible indication(s) of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will need to be considered individually. The FDA historically regulated insulins as small molecules. However, effective March 23, 2020, drugs such as insulin and growth hormone were deemed biologics and transitioned from the drug pathway to the biologic pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.

PLACE IN THERAPY

The patents of several biologic drugs are set to expire in the next few years, opening the US market for biosimilar entry; however, patent litigation has resulted in significant launch delays of FDA-approved biosimilars. In June 2017, the US Supreme Court issued 2 landmark rulings: (1) allowing a biosimilar manufacturer to provide launch notice of commercial marketing to the originator manufacturer before or after FDA approval of the biosimilar product and (2) eliminating any federal requirement for disclosure, also known as the “patent dance.” Some states, however, mandate disclosure. These decisions may bring biosimilars to the market sooner and potentially create price competition in the marketplace.

In July 2018, the FDA unveiled its Biosimilar Action Plan (BAP), a series of 11 steps to encourage biosimilar market competition, some of which were previously announced or underway. The BAP contains 4 key strategies: (1) improve the biosimilar development and approval process; (2) maximize scientific and regulatory clarity for sponsors; (3) provide effective communications for patients, clinicians, and payers; and (4) reduce unfair tactics that may delay market approval and entry. The BAP strives to promote access to biosimilar products and reduce healthcare costs.

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BIOSIMILAR OVERVIEW continued To date, a total of 40 biosimilars have received FDA approval. Of these, only 27 have entered the market.

APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zaxio® (filgrastim-sndz)

Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer

April 2016

Erelzi® (etanercept-szzs)

Sandoz

August 2016

Amjevita™* (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Merck/Organon

May 2017

Cyltezo* (adalimumab-adbm)

Boehringer Ingelheim

August 2017

Mvasi® (bevacizumab-awwb)

Amgen

September 2017

Ixifi™ ‡ (infliximab-qbtx)

Pfizer

December 2017

Ogivri® (trastuzumab-dkst)

Mylan/Biocon

December 2017

Retacrit® (epoetin alfa-epbx)

Pfizer/Vifor/ Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Mylan/Biocon

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz®* (adalimumab-adaz)

Sandoz

October 2018

Udenyca® (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Cephalon/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Merck

January 2019

Trazimera™ (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Merck

April 2019

Kanjinti® (trastuzumab-anns)

Amgen

June 2019

Zirabev® (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™*† (adalimumab-bwwd)

Organon

July 2019

Ruxience® (rituximab-pvvr)

Pfizer

July 2019

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Interchangeable -

Commercially Available

 

Originator (Manufacturer) Neupogen® (Amgen) Remicade® (Janssen)

-

-

Enbrel® (Amgen)

-

-

Humira (Abbvie)

-



Remicade (Janssen)



-

Humira (Abbvie)

-



Avastin® (Genentech)

-

-

Remicade (Janssen)

-

    -

     -

 

Herceptin® (Genentech) Epogen® (Amgen) Procrit® (Janssen) Neulasta® (Amgen) Neupogen (Amgen) Humira (Abbvie) Neulasta (Amgen) Rituxan® (Genentech) Herceptin (Genentech) Herceptin (Genentech) Herceptin (Genentech) Enbrel (Amgen) Herceptin (Genentech) Avastin (Genentech)

-

-

Humira (Abbvie)

-



Rituxan (Genentech)

BIOSIMILAR OVERVIEW continued APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Abrilada™* (adalimumab-afzb)

Pfizer

November 2019

Ziextenzo® (pegfilgrastim-bmez)

Sandoz

November 2019

Avsola® (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer

June 2020

Semglee (insulin glargine-yfgn)

Mylan/Biocon

July 2021

Hulio®* (adalimumab-fkjp)

Mylan/Biocon

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Byooviz™ (ranibizumab-nuna)

Biogen

September 2021

Rezvoglar™ (insulin glargine-aglr)

Eli Lilly

December 2021

Yusimry™* (adalimumab-aqvh)

Coherus

December 2021

Releuko® (filgrastim-ayow)

Amneal

March 2022

Alymsys® (bevacizumab-maly)

Amneal

April 2022

Fylnetra® (pegfilgrastim-pbbk)

Amneal

May 2022

Cimerli (ranibizumab-eqrn)

Coherus

August 2022

Stimufend® (pegfilgrastim-fpgk)

Fresenius Kabi

September 2022

Vegzelma® (bevacizumab-adcd)

Celltrion

September 2022

Idacio® (adalimumab-aacf)

Fresenius Kabi

December 2022

Interchangeable

Commercially Available

-

-



   

-

-

-

-

 

Originator (Manufacturer) Humira (Abbvie) Neulasta (Amgen) Remicade (Janssen) Neulasta (Amgen) Lantus® (Sanofi-Aventis) Humira (Abbvie) Rituxan (Genentech) Lucentis® (Genentech)



-

Lantus (Sanofi)

-

-

Humira (Abbvie)

-

 -

    

Neupogen (Amgen) Avastin (Genentech) Neulasta (Amgen) Lucentis (Genentech) Neulasta (Amgen)

-

-

Avastin (Genentech)

-

-

Humira (Abbvie)

* Abbvie’s adalimumab (Humira) is available in 50 mg/mL (with citric acid/citrate) and 100 mg/mL (citrate-free) concentrations. All biosimilars for the product are approved as 50 mg/mL concentrations. † Adalimumab-bwwd (Hadlima) by Organon is also approved as a 100 mg/mL high concentration citrate-free formulation (approved in August 2022). ‡ Pfizer already has Inflectra on the market and has not announced plans to launch Ixifi.

Also available are Eli Lilly’s Basaglar® insulin glargine, a follow-on to Sanofi’s Lantus, and Sanofi’s Admelog® insulin lispro approved as a follow-on to Eli Lilly’s Humalog®.

Specialty medications, which include biologics, continue to grow and constitute a large part of drug spend. In the US, it is estimated that biosimilars will cost approximately 15% to 35% less than the originator product, although price dynamics vary.

Further, the potential cost savings can vary based on the market segment where brand contracts can play a role. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play an important role in market adoption of biosimilars.

The first biosimilar version of Abbvie’s adalimumab (Humira) is anticipated to be available in the US in January 2023. It is anticipated that multiple Humira biosimilars (including citrate-free and high-concentration formulations) will enter the market in July 2023.

21 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

adalimumab SC Alvotech, Celltrion, and Sandoz are seeking approval for their investigational biosimilars to Abbvie’s citrate-free, high-concentration (100 mg/mL) Humira. Abbvie’s Humira is a tumor necrosis factor alpha (TNF-α) blocker indicated for the treatment of autoimmune disorders, including rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), ankylosing spondylitis (AS), plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD) in adults and children, ulcerative colitis (UC), hidradenitis suppurativa (HS), and non-infectious uveitis.

Pfizer is seeking interchangeability of FDA-approved adalimumab-afzb (Abrilada) 50 mg/mL.

FDA APPROVAL TIMELINE

50 mg/mL • Pfizer (Abrilada) – Pending for interchangeability 100 mg/mL • Alvotech (AVT02) – April 13, 2023 for interchangeability • Celltrion (Yuflyma) – Pending • Sandoz (Hyrimoz) – March to April 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$11,242

$6,894

$5,203

$4,199

$3,508

The forecast is a projection of total US sales per year for the branded originator product.

OPHTHALMOLOGY

aflibercept intravitreal Biocon/Janssen Biocon/Janssen are seeking approval of their investigational biosimilar to Regeneron’s Eylea®, a vascular endothelial growth factor (VEGF) inhibitor indicated for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), and diabetic retinopathy (DR).

FDA APPROVAL TIMELINE Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$6,401

$5,992

$5,147

$4,441

$3,933

The forecast is a projection of total US sales per year for the branded originator product.

22 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

ONCOLOGY

bevacizumab IV Bio-Thera Solutions/Sandoz, Centus, Samsung Bioepis/Organon, and Biocon/Janssen are seeking approval for their investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth factor (VEGF)-specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, nonsquamous non-small cell lung cancer, glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA APPROVAL TIMELINE

• Bio-Thera Solutions/Sandoz (BAT1706) – Pending • Centus (FKB238) – Pending • Samsung Bioepis/Organon (Aybintio) – Pending • Biocon/Janssen (Bmab-100) – Pending

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$505

$428

$379

$341

$308

The forecast is a projection of total US sales per year for the branded originator product.

BLOOD MODIFIER

filgrastim IV, SC Apotex and Tanvex are seeking approval of their investigational biosimilars to Amgen’s Neupogen, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs; following induction or consolidation chemotherapy for acute myeloid leukemia (AML); with nonmyeloid malignancies in patients who are undergoing myeloablative chemotherapy followed by bone marrow transplantation; to mobilize autologous hematopoietic progenitor cells for collection by leukapheresis; with symptomatic congenital neutropenia‚ cyclic neutropenia‚ or idiopathic neutropenia; and in patients who are acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).

FDA APPROVAL TIMELINE

• Apotex (Grastofil) – Pending • Tanvex (TX01) – February 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$65

$59

$52

$47

$43

The forecast is a projection of total US sales per year for the branded originator product.

23 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

NEUROLOGY

natalizumab IV Polypharma/Sandoz Polypharma/Sandoz are seeking approval for their investigational biosimilar to Biogen’s Tysabri®, an integrin receptor antagonist indicated for treatment of multiple sclerosis (MS) and Crohn’s disease (CD).

FDA APPROVAL TIMELINE May to June 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,117

$1,109

$1,097

$1,098

$1,036

The forecast is a projection of total US sales per year for the branded originator product.

BLOOD MODIFIER

pegfilgrastim SC Apotex and Lupin are seeking approval for their investigational biosimilars to Amgen’s Neulasta, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs and in patients acutely exposed to myelosuppressive doses of radiation (HSARS).

Coherus is seeking approval of Udenyca OBI®, an investigational biosimilar to Amgen’s Neulasta Onpro®.

FDA APPROVAL TIMELINE

• Apotex (Lapelga) – Pending • Lupin (Lupifil-P) – Pending • Coherus (Udenyca OBI) – October 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$768

$657

$571

$515

$465

The forecast is a projection of total US sales per year for the branded originator product.

24 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

tocilizumab IV, SC Fresenius Kabi and Biogen/Bio-Thera Solutions are seeking approval for their investigational biosimilars to Genentech’s Actemra®, an interleukin-6 (IL-6) receptor antagonist indicated for the treatment of rheumatoid arthritis (RA), giant cell arteritis, systemic sclerosis-associated interstitial lung disease, polyarticular and systemic juvenile idiopathic arthritis (JIA), and cytokine release syndrome.

FDA APPROVAL TIMELINE

• Fresenius Kabi (MSB11456) – April to June 2023 • Biogen/Bio-Thera Solutions (BIIB800 or BAT1806) – October 9, 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$1,009

$822

$660

$542

$440

The forecast is a projection of total US sales per year for the branded originator product.

IMMUNOLOGY

ustekinumab SC Alvotech Alvotech is seeking approval for their investigational biosimilar (AVT04) to Janssen’s Stelara, an interleukin 12/23 (IL-12/23) antagonist indicated for the treatment of plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD), and ulcerative colitis (UC).

FDA APPROVAL TIMELINE July to December 2023

FINANCIAL FORECAST (reported in millions) Year

2023

2024

2025

2026

2027

Projected Total US Sales

$6,389

$4,768

$3,606

$2,362

$2,351

The forecast is a projection of total US sales per year for the branded originator product.

25 | MAGELLANRX.COM

Keep on Your RADAR Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the MRx Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2027, are displayed. The financials are projected total annual US sales, reported in millions. zuranolone

avacincaptad pegol

$943

$765

Behavioral health

vamorolone

Ophthalmology

Musculoskeletal

bimekizumab Immunology

$299

$1,037

tabelecleucel

datopotamab deruxtecan

Oncology

$410

Oncology

$1,036 talquetamab

epcoritamab

Oncology

Oncology

$604

$625

resmetirom

etrasimod

Endocrine

Immunology

$686

$618 exagamglogene autotemcel

nirsevimab

Infectious disease

$855

Hematology/Gene therapy

$495

lenadogene nolparvovec

fidanacogene elaparvovec

Ophthalmology/Gene therapy

$51

Hematology/Gene therapy

lebrikizumab Immunology

$944

giroctocogene fitelparvovec

$263

Hematology/Gene therapy

$132

pecialty drug names appear in  S magenta throughout the publication.

Pipeline DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2024. It is not intended to be a comprehensive inventory of all drugs in the pipeline; emphasis is placed on drugs in high-impact categories. Investigational drugs with a Complete Response Letter (CRL).

APPLICATION APPLICATION SUBMITTED SUBMITTED TO THE FDA

PHASE33 IN PHASE TRIALS TRIALS

66%

64%

34%

36%

36%

35%

28%

Specialty

22%

13%

12%

9%

Traditional

Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar

pecialty drug names appear in  S magenta throughout the publication.

PIPELINE DRUG LIST  Specialty drug names appear in magenta throughout the publication.

NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

Submitted (New Drugs) pirtobrutinib

Eli Lilly

Mantle cell lymphoma

Oral

Submitted − NDA; Priority Jan-Feb 2023 Review

naloxone

Amphastar

Opioid overdose

Intranasal

Submitted − 505(b)(2) NDA

Jan-Mar 2023

zavegepant

Pfizer

Migraine treatment

Intranasal

Submitted − NDA

Jan-Mar 2023

risperidone ER

Teva

Schizophrenia

SC

Submitted − 505(b)(2) NDA

Jan-Jun 2023

tislelizumab

Beigene/Novartis

Esophageal squamous cell carcinoma (unresectable or metastatic, 2nd-line)

IV

Submitted − BLA; Orphan Drug

Jan-Jun 2023

velmanase alfa

Chiesi

Alpha-mannosidosis

IV

Submitted − BLA; Orphan Drug; Priority Review

Jan-Jun 2023

filgrastim (biosimilar to Amgen’s Neupogen)

Tanvex

Neutropenia/leukopenia

IV, SC

Submitted − BLA

February 2023

mirikizumab

Eli Lilly

UC (moderate to severe)

IV, SC

Submitted − BLA

Feb-Mar 2023

daprodustat

GlaxoSmithKline

Anemia due to CKD (dialysis-dependent, dialysis-independent)

Oral

Submitted − NDA

02/01/2023

elacestrant

Menarini

Breast cancer (ER+/HER2advanced or metastatic)

Oral

Submitted − NDA; Fast Track; Priority Review

02/17/2023

sparsentan

Travere

Immunoglobulin A nephropathy (Berger’s disease)

Oral

Submitted − NDA; seeking Accelerated Approval; Orphan Drug; Priority Review

02/17/2023

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Submitted − NDA; Priority 02/22/2023 Review

pegcetacoplan

Apellis

Geographic atrophy (secondary to AMD)

Intravitreal

Submitted − sNDA; Fast Track; Priority Review

02/26/2023

efanesoctocog alfa

Sanofi

Hemophilia A

IV

Submitted − BLA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

02/28/2023

omaveloxolone

Reata/Abbvie

Friedreich’s ataxia

Oral

Submitted − NDA; Fast Track; Orphan Drug; Priority Review; RPD

02/28/2023

omecamtiv mecarbil

Cytokinetics

HFrEF

Oral

Submitted − NDA; Fast Track

02/28/2023

tixagevimab/cilgavimab (Evusheld™)

AstraZeneca

COVID-19 treatment

IM

Submitted − BLA

March 2023

nirogacestat

Springworks

Desmoid tumors

Oral

Submitted − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RTOR

Mar-Dec 2023

trofinetide

Acadia

Rett syndrome

Oral

Submitted − NDA; Fast Track; Orphan Drug; Priority Review; RPD

03/12/2023

efgartigimod/ hyaluronidase

Argenx

Myasthenia gravis

SC

Submitted − BLA; Orphan Drug; Priority Review

03/20/2023

28 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

biafungin

Melinta

Candidemia/invasive candidiasis

IV

Submitted − NDA; Fast Track; Orphan Drug; Priority Review; QIDP

03/22/2023

rezafungin

Cidara

Candidemia/invasive candidiasis

IV

Submitted − NDA; Fast Track; Orphan Drug; Priority Review; QIDP

03/22/2023

leniolisib

Pharming/Novartis

Activated PI3K-delta syndrome

Oral

Submitted − NDA; Orphan Drug; Priority Review

03/29/2023

valoctocogene roxaparvovec

Biomarin

Hemophilia A (severe)

IV

Submitted − BLA; Breakthrough Therapy; Orphan Drug; RMAT

03/31/2023

anthrax vaccine, adjuvanted

Emergent

Anthrax infection (postexposure prophylaxis)

IM

Submitted − BLA; Fast Track

April 2023

bimekizumab

UCB

PSO

SC

Submitted − BLA

Apr-Jun 2023

ritlecitinib

Pfizer

Alopecia areata (ages ≥ 12 years)

Oral

Submitted − NDA; Breakthrough Therapy

Apr-Jun 2023

rozanolixizumab

UCB

Myasthenia gravis

SC

Submitted − BLA; Orphan Drug; Priority Review

Apr-Jun 2023

tocilizumab (biosimilar to Genentech’s Actemra)

Fresenius Kabi

RA; Polyarticular JIA; Systemic JIA

IV, SC

Submitted − BLA

Apr-Jun 2023

epinephrine

ARS

Anaphylaxis

Intranasal

Submitted − 505(b)(2) NDA; Fast Track

Apr-Sep 2023

adalimumab 100 mg/mL (biosimilar to Abbvie’s Humira)

Alvotech

RA; AS; PSO; PsA; JIA; CD; UC

SC

Submitted − BLA for interchangeability

04/13/2023

rizatriptan film

Gensco

Migraine treatment

Oral transmucosal

Submitted − 505(b)(2) NDA

04/17/2023

quizartinib

Daiichi Sankyo

AML (in combination with cytarabine &/or anthracycline induction & consolidation, and monotherapy following consolidation)

Oral

Submitted − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

04/24/2023

tofersen

Biogen

ALS (superoxide dismutase 1)

Intrathecal

Submitted − NDA; seeking Accelerated Approval; Orphan Drug; Priority Review

04/25/2023

SER-109

Seres

C. difficile-associated diarrhea prevention of recurrence

Oral

Submitted − BLA; Breakthrough Therapy; Orphan Drug; Priority Review

04/26/2023

aripiprazole 2-month

H. Lundbeck

Bipolar disorder; Schizophrenia

IM

Submitted − 505(b)(2) NDA

04/27/2023

palopegteriparatide

Ascendis

Hypoparathyroidism (adults)

SC

Submitted − NDA; Orphan Drug; Priority Review

04/30/2023

RSV pre-fusion protein vaccine (PF-06928316)

Pfizer

RSV prevention (ages ≥ 60 years)

Injectable

Submitted − BLA; May 2023 Breakthrough Therapy; Fast Track; Priority Review

natalizumab (biosimilar to Biogen’s Tysabri)

Polypharma/Sandoz

CD; MS

IV

Submitted − BLA

29 | MAGELLANRX.COM

May-Jun 2023

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

omidubicel

Gamida Cell

HSCT (allogenic)

IV

Submitted − BLA; Breakthrough Therapy; Orphan Drug; Priority Review

05/01/2023

RSV pre-fusion protein vaccine, adjuvanted (GSK3844766A)

GlaxoSmithKline

RSV prevention (older adults, ages ≥ 60 years)

IM

Submitted − BLA; Fast Track; Priority Review

05/03/2023

phenylephrine/ tropicamide (2.5%/1%)

Eyenovia

Mydriasis (in-office)

Ophthalmic

Submitted − 505(b)(2) NDA

05/08/2023

(vic-)trastuzumab duocarmazine

Byondis

Breast cancer (HER2+, unresectable locally advanced or metastatic)

IV

Submitted − BLA; Fast Track

05/12/2023

beremagene geperpavec

Krystal

Epidermolysis bullosa (dystrophic)

Topical

Submitted − BLA; Fast Track; Orphan Drug; Priority Review; RPD; RMAT

05/19/2023

foscarbidopa/foslevodopa

Abbvie

Parkinson’s disease motor SC fluctuations

Submitted − NDA

05/20/2023

epcoritamab

Genmab/Abbvie

DLBCL (R/R, ≥ 3rd-line)

SC

Submitted − BLA; Priority Review

05/21/2023

dengue tetravalent vaccine, live, attenuated

Takeda

Dengue fever prevention (ages 4-60 years)

SC

Submitted − BLA; Fast Track; Priority Review

05/22/2023

nalmefene

Opiant

Opioid overdose

Intranasal

Submitted −505(b)(2) NDA; Fast Track; Priority Review

05/22/2023

buprenorphine ER (weekly, monthly dosing)

Braeburn

Opioid use disorder (moderate to severe)

SC

Submitted − 505(b)(2) NDA; Fast Track

05/23/2023

nogapendekin alfa inbakicept

Immunitybio

Bladder cancer (BCG-unresponsive, non-muscle invasive carcinoma in situ, in combination with BCG)

Intravesical

Submitted − BLA; Breakthrough Therapy; Fast Track

05/23/2023

nirmatrelvir/ritonavir (Paxlovid™)

Pfizer

COVID-19 treatment (high-risk individuals)

Oral

Submitted − NDA

05/28/2023

delandistrogene moxeparvovec

Sarepta/Genentech

DMD (ambulatory patients)

IV

Submitted − BLA; seeking Accelerated Approval; Fast Track; Orphan Drug; Priority Review; RPD

05/29/2023

durlobactam/sulbactam

Innoviva

Acinetobacter baumannii infection

IV

Submitted − NDA; Fast Track; Priority Review; QIDP

05/29/2023

sotagliflozin

Lexicon

Heart failure (with or without T2DM)

Oral

Submitted − NDA

05/31/2023

landiolol

Eagle

Supraventricular tachycardia (short-term reduction of ventricular rate)

IV

Submitted − NDA

06/01/2023

cyclosporine A

Novaliq

DED

Ophthalmic

Submitted − 505(b)(2) NDA

06/08/2023

momelotinib

GlaxoSmithKline

Myelofibrosis

Oral

Submitted − NDA; Fast Track; Orphan Drug

06/16/2023

30 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

olorofim

F2G

Fungal infections (invasive)

Oral

Submitted − NDA; Breakthrough Therapy; LPAD; Orphan Drug; QIDP

06/17/2023

perfluorohexyloctane

Bausch Health

DED associated with Meibomian gland dysfunction

Ophthalmic

Submitted − NDA

06/28/2023

carbidopa/levodopa ER

Amneal

Parkinson’s disease

Oral

Submitted − 505(b)(2) NDA

06/30/2023

nirsevimab

AstraZeneca

RSV prevention (newborns/infants in 1st RSV season, ages ≥ 24 months at risk through 2nd RSV season)

IM

Submitted − BLA; Breakthrough Therapy; Fast Track

Jul-Sep 2023

sofpironium

Botanix

Axillary hyperhidrosis

Topical

Submitted − NDA

Jul-Sep 2023

eplontersen

Ionis/AstraZeneca

Transthyretin amyloid polyneuropathy

SC

Submitted − NDA; Orphan Drug

Jul-Dec 2023

etrasimod

Pfizer

UC

Oral

Submitted − NDA; Orphan Drug

Jul-Dec 2023

hypericin

Soligenix

Cutaneous T-cell lymphoma (CTCL; early stages)

Topical

Submitted − NDA; Fast Track; Orphan Drug

Jul-Dec 2023

ustekinumab (biosimilar to Janssen’s Stelara)

Alvotech

PSO; PsA; CD; UC

SC

Submitted − BLA

Jul-Dec 2023

glofitamab

Genentech

DLBCL (after ≥ 3rd-line)

IV

Submitted − BLA; Priority Review

07/01/2023

brimonidine tartrate 0.35%

Visiox

Glaucoma/ocular hypertension

Ophthalmic

Submitted − 505(b)(2) NDA

08/04/2023

bevacizumab-vikg

Outlook

Wet AMD

Intravitreal

Submitted − BLA

08/29/2023

lebrikizumab

Eli Lilly

Atopic dermatitis (moderate-severe)

SC

Submitted − BLA; Fast Track

September 2023

lotilaner

Tarsus

Demodex blepharitis

Ophthalmic

Submitted − NDA

09/07/2023

motixafortide

Biolinerx

Stem cell mobilization for autologous BMT for multiple myeloma

SC

Submitted − NDA; Orphan Drug

09/09/2023

meningococcal pentavalent Pfizer vaccine (PF-06886992)

Meningococcal immunization

IM

Submitted − BLA

October 2023

infliximab SC

Celltrion

IBS

SC

Submitted − BLA

Oct-Dec 2023

methotrexate

Aldeyra

Primary vitreoretinal lymphoma

Intravitreal

Submitted − 505(b)(2) NDA; Orphan Drug

Oct-Dec 2023

zilucoplan

UCB

Myasthenia gravis

SC

Submitted − NDA; Orphan Drug

Oct-Dec 2023

phentolamine 0.7%

Ocuphire

Pharmacologicallyinduced mydriasis reversal

Ophthalmic

Submitted − 505(b)(2) NDA

10/06/2023

tocilizumab (biosimilar to Genentech’s Actemra)

Biogen/Bio-Thera Solutions

RA; Polyarticular JIA; Systemic JIA

IV

Submitted − BLA

10/09/2023

vamorolone

Santhera

DMD

Oral

Submitted − NDA; Fast Track; Orphan Drug

10/26/2023

zuranolone

Sage

MDD; Post-partum depression

Oral

Submitted − NDA; Breakthrough Therapy; Fast Track

12/06/2023

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

talquetamab

Janssen

Multiple myeloma

SC

Submitted − BLA; Breakthrough Therapy; Orphan Drug

12/11/2023

aprocitentan

Janssen

Hypertension (resistant)

Oral

Submitted − NDA

12/20/2023

avacincaptad pegol

Iveric Bio

Dry AMD-related geograhic atrophy

Intravitreal

Submitted − NDA; Breakthrough Therapy; Fast Track

12/20/2023

chikungunya vaccine monovalent, live, attenuated

Valneva

Chikungunya prevention (single dose, adults)

IM

Submitted − BLA; Breakthrough Therapy; Fast Track

12/22/2023

pozelimab

Regeneron

Chaple syndrome

IV, SC

Submitted − BLA; Orphan Drug

12/31/2023

berdazimer

Novan

Molluscum contagiosum

Topical

Submitted − NDA

Jan-Mar 2024

pilocarpine 0.4%

Orasis

Presbyopia

Ophthalmic

Submitted − 505(b)(2) NDA

01/03/2024

cosibelimab

Checkpoint

Cutaneous squamous cell carcinoma (metastatic)

IV

Submitted − BLA

01/04/2024

adalimumab 100 mg/mL (biosimilar for Abbvie’s Humira)

Celltrion

RA; AS; PSO; PsA; JIA; CD; UC

SC

Submitted − BLA

Pending

aflibercept (biosimilar to Regeneron’s Eylea)

Biocon/Janssen

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Submitted − BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera Solutions/ Sandoz

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Submitted − BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Biocon/Janssen

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Submitted − BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Centus

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Submitted − BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Samsung Bioepis/ Organon

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Submitted − BLA

Pending

casirivimab/imdevimab (REGEN-COV)

Regeneron

COVID-19 treatment (outpatient); COVID-19 prevention

IM, IV, SC

Submitted − BLA; Priority Review

Pending

cipaglucosidase alfa

Amicus

Pompe disease (in combination with oral miglustat)

IV

Submitted − BLA; Breakthrough Therapy; Orphan Drug

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted − BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

SC

Submitted − BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Lupin

Neutropenia/leukopenia

SC

Submitted − BLA

Pending

sabizabulin

Veru

COVID-19 treatment

Oral

Submitted − EUA; Fast Track

Pending

tislelizumab

Beigene/Novartis

Esophageal squamous cell carcinoma (unresectable or metastatic, 2nd-line)

IV

Submitted − sBLA; Orphan Drug

Pending

32 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME toripalimab

MANUFACTURER Coherus

DOSAGE FORM

CLINICAL USE Nasopharyngeal cancer (advanced recurrent/ metastatic, 1st-line with gemcitabine & cisplatin, subsequent monotherapy)

IV

DEVELOPMENT STATUS

FDA DECISION

Submitted − BLA; Breakthrough Therapy; Orphan Drug

Pending

Submitted (Supplementals) lanadelumab-flyo (Takhzyro®)

Takeda

HAE prophylaxis (ages 2 to < 12 years)

SC

Submitted − sBLA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

Jan-Jun 2023

pembrolizumab (Keytruda)

Merck

NSCLC (post surgical resection; stage 1B, II, or IIIA)

IV

Submitted − sBLA

01/29/2023

relugolix/estradiol/ norethindrone (Myfembree®)

Myovant

Uterine fibroids (premenopausal women)

Oral

Submitted − sNDA

01/29/2023

asparaginase erwina chrysanthemi (recombinant)-rywn (Rylaze®)

Jazz

ALL

IV

Submitted − sBLA; Fast Track; Orphan Drug; RTOR

February 2023

sacituzumab govitecanhziy (Trodelvy®)

Gilead

Breast cancer (unresectable, locally advanced or metastatic, HR+/HER2-, post endocrine therapy and ≥ 2 additional lines of systemic metastatic therapy)

IV

Submitted − sBLA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

February 2023

aflibercept (Eylea)

Regeneron

Retinopathy of prematurity (preterm infants)

Intravitreal

Submitted − sBLA; Orphan Drug; Priority Review

02/11/2023

aflibercept (Eylea)

Regeneron

Diabetic retinopathy (16-week maintenance regimen)

Intravitreal

Submitted − sBLA

02/28/2023

olaparib (Lynparza®)

AstraZeneca

Prostate cancer (mCRPC, Oral combination with prednisone ± abiraterone)

Submitted − sNDA; Breakthrough Therapy; Priority Review

March 2023

adalimumab-adaz 100 mg/mL (Hyrimoz) (biosimilar to Abbvie’s Humira)

Sandoz

RA; AS; PSO; PsA; JIA; CD; UC

SC

Submitted − sBLA

Mar-Apr 2023

pegcetacoplan (Empaveli®) injector

Apellis

PNH

SC

Submitted − sNDA

03/15/2023

avapritinib (Ayvakit®)

Blueprint

Mastocytosis (indolent, systemic)

Oral

Submitted − sNDA; Breakthrough Therapy; Orphan Drug; Priority Review

03/22/2023

naloxone (Narcan®)

Emergent

Opioid overdose

Intranasal

Submitted − sNDA for Rx- 03/29/2023 to-OTC; Priority Review

evinacumab-dgnb (Evkeeza®)

Regeneron

HoFH (ages 5-11 years, adjunct)

IV

Submitted − sBLA; Breakthrough Therapy; Orphan Drug; Priority Review

03/30/2023

cobimetinib (Cotellic®)

Genentech

Histiocytosis

Oral

Submitted − sNDA; Breakthrough Therapy

April 2023

33 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

pneumococcal conjugate vaccine candidate (20-valent) (Prevnar®)

Pfizer

Invasive pneumococcal disease prevention & related otitis media (ages 6 weeks-17 years)

IM

Submitted − sBLA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

April 2023

somapacitan-beco (Sogroya®)

Novo Nordisk

Growth hormone deficiency (pediatric)

SC

Submitted − sBLA

Apr-Jun 2023

polatuzumab vedotin-piiq (Polivy®)

Genentech

DLBCL (1st-line, combination with rituximab/ cyclophosphamide or doxorubicin/prednisone)

IV

Submitted − sBLA; Breakthrough Therapy; Orphan Drug

04/02/2023

atogepant (Qulipta™)

Abbvie

Migraine prevention

Oral

Submitted − sNDA

04/21/2023

enfortumab vedotin-ejfv (Padcev®)

Astellas

Urothelial cancer (advanced, 1st-line)

IV

Submitted − sBLA; Breakthrough Therapy; Priority Review

04/21/2023

elexacaftor/tezacaftor/ ivacaftor (Trikafta®)

Vertex

CF (ages 2-5 years, F508del mutation or responsive mutation)

Oral

Submitted − sNDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

04/28/2023

durvalumab (Imfinzi®)

AstraZeneca

HCC (unresectable)

IV

Submitted − sBLA; Orphan Drug

04/30/2023

ivacaftor (Kalydeco®)

Vertex

CF (ages 1 to < 4 months, CFTR gene mutation responsive to ivacaftor)

Oral

Submitted − sNDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review

05/03/2023

brexpiprazole (Rexulti®)

Otsuka

Alzheimer’s disease

Oral

Submitted − sNDA; Fast Track; Priority Review

05/10/2023

Abbvie

CD (moderate to severe)

Oral

Submitted − sNDA

05/26/2023

atezolizumab (Tecentriq )

Genentech

Sarcoma (alveolar soft part)

IV

Submitted − sBLA

June 2023

mavacamten (Camzyos®)

Bristol-Myers Squibb

Cardiomyopathy (reduce need for septal reduction therapy)

Oral

Submitted − sNDA; Breakthrough Therapy; Orphan Drug

06/16/2023

obeticholic acid (Ocaliva®)

Intercept

Pre-cirrhotic liver fibrosis due to NASH

Oral

Submitted − sNDA; seeking Accelerated Approval; Breakthrough Therapy

06/22/2023

brentuximab vedotin (Adcetris®)

Seagen

Hodgkin’s Lymphoma (untreated advanced)

IV

Submitted − sBLA; Breakthrough Therapy; Fast Track; Orphan Drug

July 2023

odevixibat (Bylvay®)

Albireo

Alagille syndrome-related Oral cholestatic pruritus

Submitted − sNDA; Orphan Drug

Jul-Dec 2023

vosoritide (Voxzogo®)

Biomarin

Achondroplasia (ages < 5 years)

SC

Submitted − sNDA; Orphan Drug

Jul-Dec 2023

rucaparib (Rubraca®)

Clovis

Ovarian cancer (1st-line, post 1st-line platinum chemotherapy)

Oral

Submitted − sNDA; Breakthrough Therapy; Orphan Drug

07/12/2023

denileukin diftitox (Ontak®) Citius

Cutaneous T cell lymphoma (recurrent)

IV

Submitted − sBLA; Orphan Drug

07/28/2023

aflibercept (Eylea) 8 mg dose

Regeneron

Wet AMD (extended dose intervals)

Intravitreal

Submitted − sBLA; Priority Review

August 2023

daxibotulinumtoxinA-lanm (Daxxify™)

Revance

Cervical dystonia

IM

Submitted − sBLA; Orphan Drug

08/19/2023

upadacitinib (Rinvoq) ®

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

valbenazine (Ingrezza®)

Neurocrine Biosciences

Huntington’s disease

Oral

Submitted − sNDA; Orphan Drug

08/20/2023

empagliflozin (Jardiance)

Boehringer Ingelheim/Eli Lilly

CKD (reduce the risk of kidney disease progression & CV death)

Oral

Submitted − sNDA; Fast Track

Sep-Oct 2023

ravulizumab-cwvz (Ultomiris®)

AstraZeneca

Neuromyelitis optica (Devic’s syndrome)

IV, SC

Submitted − sBLA

09/30/2023

pegfilgrastim-cbqv onbody injector (Udenyca OBI) (biosimilar to Amgen’s Neulasta Onpro)

Coherus

Neutropenia/leukopenia

SC

Submitted − sBLA

October 2023

phentolamine (Nyxol®)

Ocuphire

Pharmacologicallyinduced mydriasis reversal

Ophthalmic

Submitted − 505(b)(2) sNDA

10/06/2023

linaclotide (Linzess®)

Ironwood

Functional constipation (ages 6-17 years)

Oral

Submitted − sNDA

10/16/2023

roflumilast (Zoryve™)

Arcutis

PSO (ages 2-11 years)

Topical

Submitted − sNDA

10/19/2023

secukinumab (Cosentyx®)

Novartis

Hidradenitis suppurativa

IV, SC

Submitted − sBLA

10/25/2023

bupivacaine/meloxicam (Zynrelef®)

Heron

Postsurgical pain (soft tissue and orthopedic surgical procedures)

Instillation

Submitted − 505(b)(2) sNDA; Breakthrough Therapy; Fast Track

10/27/2023

dupilumab (Dupixent®)

Sanofi

Urticaria (chronic, spontaneous)

SC

Submitted − sBLA

10/31/2023

adalimumab-afzb 50 mg/mL (Abrilada) (biosimilar to Abbvie’s Humira)

Pfizer

RA; AS; PSO; PsA; JIA; CD; UC

SC

Submitted − PAS BLA for interchangeability

Pending

norgestrel (Opill®)

Perrigo

Contraception

Oral

Submitted − sNDA for Rx-to-OTC

Pending

vonoprazan/amoxicillin/ clarithromycin (Takecab®)

Phathom

Erosive esophagitis

Oral

Submitted − sNDA

Pending

Subretinal

Phase 3 − BLA; Orphan Drug

TBD

Phase 3 (New Drugs) AAV8-ranibizumab

Regenxbio

Wet AMD

abelacimab

Anthos

Stroke prevention in atrial IV, SC fibrillation; VTE

Phase 3 − BLA; Fast Track

TBD

abiraterone

Tavanta

Prostate cancer

Oral

Phase 3 − NDA

TBD

acoramidis

Bridgebio/ AstraZeneca

Transthyretin amyloid cardiomyopathy (ATTRCM)

Oral

Phase 3 − NDA

TBD

adapalene/benzoyl peroxide/clindamycin

Bausch Health

Acne

Topical

Phase 3 − NDA

TBD

adintrevimab

Invivyd/Biocon

COVID-19

IM

Phase 3 − BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Amgen

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

SamChunDang

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

35 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

aflibercept (biosimilar to Regeneron’s Eylea)

Samsung Bioepis/ Biogen

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Santo/Formycon

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

aloradine

Vistagen

Social anxiety disorder

Intranasal

Phase 3 − NDA; Fast Track TBD

amubarvimab/ romlusevimab

Brii

COVID-19

IV

Phase 3 − BLA

TBD

anti-betv1 antibody (REGN- Regeneron 5713-5714-5715)

Birch allergy

SC

Phase 3 − BLA

TBD

apolipoprotein A1 (human)

CSL

Atherosclerosis

IV

Phase 3 − BLA

TBD

AR-15512

Aerie

DED

Ophthalmic

Phase 3 − NDA

TBD

arimoclomol

Kempharm

Niemann-Pick disease

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RPD

TBD

ARO-APOC3

Arrowhead

Familial chylomicronemia syndrome

SC

Phase 3 − NDA; Orphan Drug

TBD

asundexian

Bayer

Stroke prevention in atrial Oral fibrillation

Phase 3 − NDA

TBD

ataluren

PTC

DMD

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors

Brainstorm

ALS

Intrathecal

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

avasopasem manganese

Galera

Mucositis

IV

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

bamlanivimab

Eli Lilly

COVID-19

IV

Phase 3 − BLA

TBD

batiraxcept

Aravive

Ovarian cancer

IV

Phase 3 − BLA; Fast Track

TBD

bentracimab

Phasebio/ AstraZeneca

Ticagrelor (Brilinta®) reversal

IV

Phase 3 − BLA; Breakthrough Therapy

TBD

bevacizumab (biosimilar to Genentech’s Avastin)

Kyowa Kirin

Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC

IV

Phase 3 − BLA

TBD

bimekizumab

UCB

Axial spondyloarthritis; Hidradenitis suppurativa; PsA

SC

Phase 3 − BLA

TBD

bis-choline tetrathiomolybdate

AstraZeneca

Wilson’s disease

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

botaretigene sparoparvovec

Janssen

Retinitis pigmentosa

Subretinal

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

BPR277

Lifemax

Congenital ichthyosis

Topical

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

brensocatib

Insmed/AstraZeneca

Bronchiectasis

Oral

Phase 3 − NDA; Breakthrough Therapy

TBD

buntanetap

Annovis

Parkinson’s disease

Oral

Phase 3 − NDA

TBD

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION TBD

cannabidiol gel

Zynerba

Fragile X syndrome

Topical

Phase 3 − NDA; Fast Track; Orphan Drug

capsaicin

Centrexion

Osteoarthritis pain (knee)

Intraarticular

Phase 3 − NDA; Fast Track TBD

ceftobiprole medocaril

Basilea

Bacteremia; CAP; HAP; SCLC

IV

Phase 3 − NDA; Fast Track; QIDP

TBD

ceftriaxone wearable pump scPharmaceuticals

Gram+/Gram- infection

SC

Phase 3 − NDA

TBD

clobetasol propionate

Formosa

Ocular pain/inflammation

Ophthalmic

Phase 3 − 505(b)(2) NDA

TBD

CM-AT (pancreatic enzyme)

Curemark

Autism spectrum disorders

Oral

Phase 3 − BLA; Fast Track

TBD

cobitolimod

Index/Merck

UC

Rectal

Phase 3 − NDA; Orphan Drug

TBD

colistimethate sodium

Zambon

Bronchiectasis

Inhaled

Phase 3 − NDA; Breakthrough Therapy; Fast Track; QIDP

TBD

concizumab

Novo Nordisk

Hemophilia A and B

SC

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

copper histidine

Zydus

Menkes disease

SC

Phase 3 − NDA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

COVID-19 vaccine (C19VAZ; formerly AZD1222; ChAdOx1)

AstraZeneca

COVID-19

IM

Phase 3 − BLA

TBD

COVID-19 vaccine (JNJ78436735; formerly Ad26.

COV2-S)

Janssen

COVID-19

IM

Phase 3 − BLA

TBD

COVID-19 vaccine (MT2766)

GlaxoSmithKline

COVID-19

IM, SC

Phase 3 − BLA; Fast Track

TBD

COVID-19 vaccine (SCB2019)

Clover

COVID-19

SC

Phase 3 − BLA

TBD

COVID-19 vaccine (SP0253)

Sanofi/ GlaxoSmithKline

COVID-19

IM

Phase 3 − BLA

TBD

COVID-19 vaccine, adjuvanted

Novavax

COVID-19

IM

Phase 3 − BLA

TBD

crinecerfont

Neurocrine

Congenital adrenal hyperplasia

Oral

Phase 3 − NDA; Orphan Drug

TBD

crovalimab

Genentech

Hemolytic uremic syndrome; PNH

IV, SC

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

cyclobenzaprine

Tonix

Fibromyalgia

SL

Phase 3 − 505(b)(2) NDA

TBD

dabocemagene autoficel

Castle Creek

Epidermolysis bullosa

Intradermal

Phase 3 − BLA; Fast Track; Orphan Drug; RMAT

TBD

danicopan

AstraZeneca

PNH

Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug

TBD

darvadstrocel

Takeda

CD

IV

Phase 3 − BLA; Orphan Drug

TBD TBD

datopotamab deruxtecan

NSCLC; Breast Cancer

IV

Phase 3 − BLA

delgocitinib

Leo

Atopic dermatitis

Topical

Phase 3 − NDA; Fast Track TBD

denosumab (biosimilar to Amgen’s Prolia®)

Biocon

Osteoporosis/osteopenia

SC

Phase 3 − BLA

37 | MAGELLANRX.COM

TBD

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

denosumab (biosimilar to Amgen’s Prolia)

Celltrion

Osteoporosis/osteopenia

SC

Phase 3 − BLA

TBD

denosumab (biosimilar to Amgen’s Prolia)

Gedeon Richter

Osteoporosis/osteopenia

SC

Phase 3 − BLA

TBD

denosumab (biosimilar to Amgen’s Prolia)

Sandoz

Osteoporosis/osteopenia

SC

Phase 3 − BLA

TBD

denosumab (biosimilar to Amgen’s Prolia)

Teva

Osteoporosis/osteopenia

SC

Phase 3 − BLA

TBD

dersimelagon

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

difamilast

Medimetriks

Atopic dermatitis

Topical

Phase 3 − NDA

TBD

difelikefalin

Cara

Atopic dermatitis; Pruritus Oral

Phase 3 − NDA

TBD

difluprednate XR

Visiox

Ocular pain/inflammation

Ophthalmic

Phase 3 − NDA

TBD

dirloctocogene samoparvovec

Genentech

Hemophilia A

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers (chronic non-healing)

IM

Phase 3 − BLA

TBD

doravirine/islatravir

Merck

HIV-1 infection treatment

Oral

Phase 3 − NDA

TBD

dovitinib lactate

Allarity

Breast cancer

Oral

Phase 3 − NDA

TBD

dust mite immunotherapy tablet

Stallergenes Greer

Allergic rhinitis

SL

Phase 3 − BLA

TBD

EB-101 (gene therapy)

Abeona

Epidermolysis bullosa

Surgical application

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug; RMAT

TBD

ebselen

Sound

Meniere’s disease

Oral

Phase 3 − NDA; Fast Track TBD

eculizumab (biosimilar to Alexion’s Soliris®)

Amgen

PNH

IV

Phase 3 − BLA

TBD

efgartigimod/ hyaluronidase

Argenx

Bullous pemphigoid; ITP; Pemphigus vulgaris

SC

Phase 3 − BLA; Orphan Drug

TBD

efzofitimod

Atyr

Sarcoidosis

IV

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

elamipretide

Stealth

Barth syndrome

SC

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

IV

Phase 3 − NDA; Fast Track; QIDP

TBD

ensifentrine

Verona

COPD

Inhaled

Phase 3 − NDA

TBD

ensovibep

Novartis

COVID-19

IV

Phase 3 − BLA; Fast Track

TBD

epinephrine

Bryn

Anaphylaxis

Intranasal

Phase 3 − NDA; Fast Track TBD

eplontersen

Ionis/AstraZeneca

Transthyretin amyloid cardiomyopathy

SC

Phase 3 − NDA; Orphan Drug

TBD

esreboxetine

Axsome/Pfizer

Fibromyalgia

Oral

Phase 3 − NDA

TBD

etanercept (biosimilar to Amgen’s Enbrel)

Coherus

RA; Polyarticular JIA; AS; PSO; PsA

SC

Phase 3 − BLA

TBD

etavopivat

Novo Nordisk

SCD

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

etesevimab

Eli Lilly

COVID-19

IV

Phase 3 − BLA

TBD

evobrutinib

Merck

MS

Oral

Phase 3 − NDA

TBD

38 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

exagamglogene autotemcel

Vertex

SCD; Thalassemia

IV

Phase 3 − BLA; Breakthrough Therapy; Fast Track; Orphan Drug; RMAT

TBD

factor VIII mimetic bispecific antibody

Novo Nordisk

Hemophilia A

SC

Phase 3 − BLA; Orphan Drug

TBD

fasinumab

Regeneron

Osteoarthritis pain (knee)

SC

Phase 3 − BLA

TBD

Feld1 antibody (REGN1908-1909)

Regeneron

Allergic rhinitis

SC

Phase 3 − BLA

TBD

fexapotide triflutate

Nymox

BPH

Intraprostatic

Phase 3 − NDA

TBD

fidanacogene elaparvovec

Pfizer/Genentech

Hemophilia B

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

CD; UC

Oral

Phase 3 − NDA

TBD

fitusiran

Sanofi

Hemophilia A and B

SC

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

fluticasone propionate

Ellodi

Esophagitis

Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

follitropin alfa (biosimilar to EMD Serono’s Gonal-F®)

Abbvie

Female reproductive disorder

SC

Phase 3 − BLA

TBD

garadacimab

CSL

HAE

SC

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

gavorestat

Applied Therapeutics Galactosemia

Oral

Phase 3 − NDA; Fast Track; Orphan Drug; RPD

TBD

GBT601

Pfizer

SCD

Oral

Phase 3 − NDA; Orphan Drug

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3 − NDA; QIDP

TBD

giredestrant

Genentech

Breast cancer

Oral

Phase 3 − NDA; Fast Track TBD

giroctocogene fitelparvovec

Pfizer

Hemophilia A

IV

Phase 3 − BLA; Fast Track; Orphan Drug; RMAT

TBD

glatiramer acetate depot

Viatris

MS

IM

Phase 3 − 505(b)(2) NDA

TBD

gold nanocrystal

Clene

ALS

Oral

Phase 3 − NDA; Orphan Drug

TBD

imetelstat

Geron

Myelodysplastic syndrome

IV

Phase 3 − NDA

TBD

inavolisib

Genentech

Breast cancer

Oral

Phase 3 − NDA

TBD

inclacumab

Pfizer

SCD

IV

Phase 3 − BLA; Orphan Drug

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; CD

IV

Phase 3 − BLA

TBD

influenza nanoparticle vaccine

Novavax

Seasonal influenza prevention

IM

Phase 3 − BLA; Fast Track

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog®)

Amphastar

T1DM; T2DM

SC

Phase 3 − BLA

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Sanofi

T1DM; T2DM

SC

Phase 3 − BLA

TBD

insulin glargine (biosimilar to Sanofi’s Lantus)

Gan & Lee

T1DM; T2DM

SC

Phase 3 − BLA

TBD

insulin icodec (onceweekly)

Novo Nordisk

T2DM

SC

Phase 3 − BLA

TBD

39 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

iodine-131 apamistamab

Actinium

AML

IV

Phase 3 − BLA; Orphan Drug

TBD

ipatasertib

Genentech

Prostate cancer

Oral

Phase 3 − NDA

TBD

iptacopan

Novartis

Complement 3 (C3) glomerulopathy; Hemolytic uremic syndrome; Immunoglobulin A nephropathy (Berger’s disease); PNH

Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug

TBD

isotretinoin

Timber

Congenital ichthyosis

Topical

Phase 3 − 505(b)(2) NDA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

itepekimab

Regeneron/Sanofi

COPD

SC

Phase 3 − BLA

TBD

JDQ-443

Novartis

NSCLC

Oral

Phase 3 − NDA

TBD

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3 − BLA; Orphan Drug

TBD

lanifibranor

Inventiva

NASH

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

lazertinib

Genosco

NSCLC

Oral

Phase 3 − NDA

TBD

lenadogene nolparvovec

Gensight

Leber’s hereditary optic neuropathy

Intravitreal

Phase 3 − BLA; Orphan Drug

TBD

lenzilumab

Humanigen

COVID-19

IV

Phase 3 − BLA

TBD

leriglitazone

Minoryx

Adrenoleukodystrophy

Oral

Phase 3 − NDA; Fast Track; Orphan Drug; RPD

TBD

leukocyte interleukin

CEL-SCI

SCCHN

Peritumor/ perilymphatic

Phase 3 − BLA; Orphan Drug

TBD

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

SC

Phase 3 − 505(b)(2) NDA

TBD

ligelizumab

Novartis

Food allergies; Urticaria

SC

Phase 3 − BLA; Breakthrough Therapy

TBD

litifilimab

Biogen

SLE

SC

Phase 3 − BLA

TBD

lovotibeglogene autotemcel

Bluebird Bio

SCD

IV

Phase 3 − BLA; Fast Track; Orphan Drug; RMAT

TBD

Lyme disease vaccine

Valneva

Lyme disease prevention

IM

Phase 3 − BLA; Fast Track

TBD

magrolimab

Gilead

Myelodysplastic syndrome

IV

Phase 3 − BLA; Breakthrough Therapy; Fast Track; Orphan Drug

TBD

marstacimab

Pfizer

Hemophilia A and B

IV, SC

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

masitinib

AB Science

ALS; Alzheimer’s disease; Asthma (eosinophilic); Mastocytosis; MS

Oral

Phase 3 − NDA; Orphan Drug

TBD

mavorixafor

X4

Warts, Oral hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome

Phase 3 − NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RPD

TBD

melphalan

Delcath

Uveal melanoma (hepatic-dominant)

Phase 3 − NDA

TBD

40 | MAGELLANRX.COM

Percutaneous hepatic perfusion

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

meningococcal vaccine (GSK3536819A)

GlaxoSmithKline

Meningococcal immunization

IM

Phase 3 − BLA

TBD

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

IV

Phase 3 − BLA; Orphan Drug; RMAT

TBD

midomafetamine

Multidisciplinary Association for Psychedelic Studies

PTSD

Oral

Phase 3 − NDA; Breakthrough Therapy

TBD

minocycline

Journey

Rosacea

Oral

Phase 3 − 505(b)(2) NDA

TBD

minocycline/edetate/ethyl alcohol

Citius

Catheter-related bloodstream infection

IV

Phase 3 − 505(b)(2) NDA; Fast Track; QIDP

TBD

mirikizumab

Eli Lilly

CD

IV, SC

Phase 3 − BLA

TBD

molnupiravir

Merck

COVID-19

Oral

Phase 3 − NDA

TBD

mometasone furoate

Lyra

Chronic rhinosinusitis

Implant

Phase 3 − 505(b)(2) NDA

TBD

nabiximols

Jazz

MS-related spasticity

Oral transmucosal

Phase 3 − NDA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 − NDA; Fast Track TBD

naloxone

Orexo

Opioid overdose

Intranasal

Phase 3 − 505(b)(2) NDA

TBD

narsoplimab

Omeros

Hemolytic uremic syndrome

IV, SC

Phase 3 − BLA; Fast Track

TBD

navitoclax

Abbvie/Genentech

Myelofibrosis

Oral

Phase 3 − NDA; Orphan Drug

TBD

nedosiran

Novo Nordisk

Hyperoxaluria

SC

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug; RPD

TBD

nemolizumab

Galderma

Atopic dermatitis

SC

Phase 3 − BLA

TBD

nipocalimab

Janssen

Autoimmune hemolytic anemia (warm)

IV

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

NIS793

Novartis

Pancreatic cancer

IV

Phase 3 − BLA; Orphan Drug

TBD

nomacopan

Akari

HSCT-associated thrombotic microangiopathy; PNH

SC

Phase 3 − BLA; Fast Track; Orphan Drug; RPD

TBD

obefazimod

Abivax

UC

Oral

Phase 3 − NDA

TBD

olezarsen

Akcea

Familial chylomicronemia syndrome

SC

Phase 3 − NDA

TBD

omalizumab (biosimilar for Genentech’s Xolair®)

Teva

Asthma; Nasal polyps; Urticaria

SC

Phase 3 − BLA

TBD

OPT-302

Opthea

Wet AMD

Intravitreal

Phase 3 − BLA; Fast Track

TBD

OTL-103

Orchard

Wiskott-Aldrich syndrome IV

Phase 3 − BLA; Orphan Drug; RMAT

TBD

Oxalobacter formigenes

Oxthera

Hyperoxaluria

Oral

Phase 3 − BLA; Orphan Drug; RPD

TBD

padeliporfin

Steba

Bladder cancer

IV

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

paliperidone palmitate

Luye

Schizophrenia

IM

Phase 3 − 505(b)(2) NDA

TBD

paltusotine

Crinetics

Acromegaly

Oral

Phase 3 − NDA; Orphan Drug

TBD

pamrevlumab

Fibrogen

COVID-19; DMD; IV Idiopathic pulmonary fibrosis; Pancreatic cancer

Phase 3 − BLA; Fast Track; Orphan Drug; RPD

TBD

41 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

papillomavirus therapeutic vaccine (VGX-3100)

Inovio

Cervical dysplasia (human IM papillovirus-positive)

Phase 3 − BLA

TBD

parsaclisib

Incyte

Autoimmune hemolytic anemia (warm)

Oral

Phase 3 − NDA; Orphan Drug

TBD

pegadricase synthetic vaccine particle

Swedish Orphan Biovitrum

Gout

IV

Phase 3 − BLA

TBD

pegzilarginase

Aeglea

Arginase 1 deficiency

IV

Phase 3 − BLA; Breakthrough Therapy; Fast Track; Orphan Drug; RPD

TBD

phentolamine 0.7%

Ocuphire

Dim light/night vision disturbances; Presbyopia

Ophthalmic

Phase 3 − 505(b)(2) NDA

TBD

piclidenoson

Can-Fite

Psoriasis

Oral

Phase 3 − NDA

TBD

PL-9643

Palatin

DED

Ophthalmic

Phase 3 − NDA

TBD

plastoquinone derivative

Mitotech

DED

Ophthalmic

Phase 3 − NDA

TBD

plinabulin

Beyondspring

Chemotherapy-induced neutropenia prevention; NSCLC

IV

Phase 3 − NDA; Breakthrough Therapy

TBD

plonmarlimab

I-Mab

COVID-19

IV

Phase 3 − BLA

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

SC

Phase 3 − BLA

TBD

pollinex quattro ragweed

Allergy Therapeutics

Allergic rhinitis

SC

Phase 3 − BLA

TBD

potassium citrate/ potassium bicarbonate

Advicenne

Renal tubular acidosis

Oral

Phase 3 − 505(b)(2) NDA; Orphan Drug

TBD

pozelimab

Regeneron

PNH

IV, SC

Phase 3 − BLA

TBD

pramipexole/rasagiline

Pharma Two B

Parkinson’s disease

Oral

Phase 3 − 505(b)(2) NDA

TBD

pritelivir

Aicuris Anti-infective Cures

Herpes simplex virus treatment

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

prothrombin complex

Octapharma

Hemostasis

IV

Phase 3 − BLA

TBD

proxalutamide

Kintor

COVID-19

Oral

Phase 3 − NDA

TBD

ralinepag

United

PAH

Oral

Phase 3 − NDA; Orphan Drug

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Stada Arzneimittel/ Bausch

Diabetic retinopathy; DME; Myopic choroidal neovascularization; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

rapamycin (high-strength)

Palvella

Pachyonychia congenita

Topical

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 − NDA; Orphan Drug

TBD

remibrutinib

Novartis

Urticaria

Oral

Phase 3 − NDA

TBD

reproxalap

Aldeyra

Allergic conjunctivitis; DED

Ophthalmic

Phase 3 − NDA

TBD

resmetirom

Madrigal

NASH

Oral

Phase 3 − NDA; Fast Track TBD

RGX-121

Regenxbio

Mucopolysaccharidosis II (Hunter syndrome)

Intracisternally

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 − NDA; Fast Track; QIDP

TBD

rocatinlimab

Amgen

Atopic dermatitis

IV

Phase 3 − BLA

TBD

roflumilast foam

Arcutis

Atopic dermatitis (scalp); PSO

Topical

Phase 3 − NDA

TBD

42 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

roluperidone

Minerva

Schizophrenia (negative symptoms)

Oral

Phase 3 − NDA

TBD

roxadustat

AstraZeneca

Anemia due to cytotoxic chemotherapy

Oral

Phase 3 − NDA

TBD

RSV pre-fusion F protein vaccine (JNJ-64400141)

Janssen

RSV prevention (ages ≥ 60 years)

IM

Phase 3 − BLA; Breakthrough Therapy

TBD

RSV pre-fusion F protein vaccine (mRNA-1345)

Moderna

RSV prevention (ages ≥ 60 years)

IM

Phase 3 − BLA; Fast Track

TBD

ruxolitinib (deuterated)

Concert

Alopecia areata

Oral

Phase 3 − NDA; Breakthrough Therapy; Fast Track

TBD

sabatolimab

Novartis

Myelodysplastic syndrome

IV

Phase 3 − BLA; Fast Track

TBD

saroglitazar

Zydus

Primary biliary cholangitis Oral

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

sebetralstat

Kalvista

HAE

Phase 3 − NDA; Fast Track; Orphan Drug

TBD

seladelpar

Cymabay

Primary biliary cholangitis Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug

TBD

seltorexant

Janssen

MDD

Oral

Phase 3 − NDA

TBD

sotatercept

Merck

PAH

SC

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

sparsentan

Travere

Focal segmental glomerulosclerosis

Oral

Phase 3 − NDA; Orphan Drug

TBD

sulopenem

Iterum

Intra-abdominal bacterial infection

IV, Oral

Phase 3 − NDA

TBD

tabelecleucel

Atara

Epstein-Barr virus-associated post-transplant lymphoproliferative disease

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

TAK-755

Takeda

Thrombotic thrombocytopenic purpura

IV

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

tamibarotene

Syros

Myelodysplastic syndrome

Oral

Phase 3 − NDA; Orphan Drug

TBD

tanfanercept

Hanall

DED

Ophthalmic

Phase 3 − BLA

TBD

tarcocimab tedromer

Kodiak

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Phase 3 − BLA

TBD

tebipenem pivoxil

Spero/ GlaxoSmithKline

UTI (complicated)

Oral

Phase 3 − NDA; Fast Track; QIDP

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 − NDA

TBD

telisotuzumab vedotin

Abbvie

NSCLC

IV

Phase 3 − BLA; Breakthrough Therapy

TBD

tiragolumab

Genentech

Esophageal cancer; NSCLC

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

43 | MAGELLANRX.COM

Oral

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

tiratricol

Rare Thyroid Therapeutics

Resistance to thyroid hormone type beta (RTH-b)

Oral

Phase 3 − NDA; Fast Track; Orphan Drug; RPD

TBD

tislelizumab

Beigene

Gastric cancer; HCC; NSCLC

IV

Phase 3 − BLA; Orphan Drug

TBD

tixagevimab/cilgavimab (Evusheld)

AstraZeneca

COVID-19

IM

Phase 3 − BLA

TBD

tominersen

Genentech

Huntington’s disease

Intrathecal

Phase 3 − NDA; Orphan Drug

TBD

tovorafenib

Day One

Brain cancer

Oral

Phase 3 − NDA; Breakthrough Therapy; Orphan Drug; RPD

TBD

tradipitant

Vanda/Eli Lilly

Atopic dermatitis; COVID-19; Emesis; Gastroparesis; Pruritus

Oral

Phase 3 − NDA

TBD

travoprost implant

Glaukos

Glaucoma/ocular hypertension

Intraocular

Phase 3 − 505(b)(2) NDA

TBD

tusamitamab ravtansine

Sanofi

NSCLC

IV

Phase 3 − BLA

TBD

upifitamab rilsodotin

Mersana

Ovarian cancer

IV

Phase 3 − BLA; Fast Track

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Amgen

PSO; PsA; CD; UC

IV, SC

Phase 3 − BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO; PsA; CD; UC

IV, SC

Phase 3 − BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Hikma

PSO; PsA; CD; UC

IV, SC

Phase 3 − BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Intas

PSO; PsA; CD; UC

IV, SC

Phase 3 − BLA

TBD

venglustat

Sanofi

Gaucher’s disease; GM2 gangliosidoses (TaySachs disease, Sandhoff disease, AB variant)

Oral

Phase 3 − NDA; Orphan Drug

TBD

Viaskin peanut

DBV

Peanut allergy

Transdermal

Phase 3 − BLA; Breakthrough Therapy; Fast Track

TBD

von Willebrand factor concentrate

LFB

von Willebrand disease

IV

Phase 3 − BLA; Orphan Drug

TBD

xanomeline/trospium

Karuna

Schizophrenia

Oral

Phase 3 − NDA

TBD

zanidatamab

Zymeworks

Gastric cancer

IV

Phase 3 − BLA; Fast Track; Orphan Drug

TBD

zinpentraxin alfa

Genentech

Idiopathic pulmonary fibrosis

IV

Phase 3 − BLA; Breakthrough Therapy; Orphan Drug

TBD

zolbetuximab

Astellas

Gastric cancer

IV

Phase 3 − BLA; Orphan Drug

TBD

zoliflodacin

Innoviva

Gonorrhea

Oral

Phase 3 − NDA; Fast Track; QIDP

TBD

Phase 3 (Supplementals) atezolizumab (Tecentriq)

Genentech

SCCHN

IV

Phase 3 − sBLA

TBD

baricitinib (Olumiant)

Eli Lilly

JIA; Uveitis

Oral

Phase 3 − sNDA

TBD

44 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

benralizumab (Fasenra®)

AstraZeneca

ANCA-associated vasculitis; Bronchiectasis; Bullous pemphigoid; Esophagitis

SC

Phase 3 − sBLA; Orphan Drug

TBD

brexpiprazole (Rexulti)

Otsuka

PTSD

Oral

Phase 3 − sNDA

TBD

dupilumab (Dupixent)

Sanofi

Allergic fungal rhinosinusitis; Bullous pemphigoid; Chronic rhinosinusitis; COPD

SC

Phase 3 − sBLA; Orphan Drug

TBD

efgartigimod (Vyvgart®)

Argenx

ITP

IV

Phase 3 − sBLA; Orphan Drug

TBD

empagliflozin (Jardiance)

Boehringer Ingelheim/Eli Lilly

Diabetic nephropathy

Oral

Phase 3 − sNDA

TBD

ferric carboxymaltose (Injectafer®)

Daiichi Sankyo

Anemia in heart failure

IV

Phase 3 − sNDA

TBD

ferric derisomaltose (Monoferric®)

Pharmacosmos

Anemia in heart failure

IV

Phase 3 − sNDA

TBD

fluticasone propionate (Xhance®)

Optinose

Chronic rhinosinusitis

Intranasal

Phase 3 − sNDA

TBD

fostamatinib (Tavalisse®)

Rigel

Autoimmune hemolytic anemia (warm)

Oral

Phase 3 − sNDA; Fast Track; Orphan Drug

TBD

guselkumab (Tremfya)

Janssen

UC

SC

Phase 3 − sBLA

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 − sNDA

TBD

immune globulin (human) 10% (Octagam®)

Octapharma

COVID-19

IV

Phase 3 − sBLA

TBD

inebilizumab-cdon (Uplizna®)

Horizon

Immunoglobulin G4-related disease; Myasthenia gravis

IV

Phase 3 − sBLA

TBD

irinotecan liposomal (Onivyde®)

Ipsen

SCLC

IV

Phase 3 − sNDA; Fast Track; Orphan Drug

TBD

maralixibat (Livmarli®)

Mirum

Progressive familial intrahepatic cholestasis

Oral

Phase 3 − sNDA; Breakthrough Therapy; Orphan Drug

TBD

mepolizumab (Nucala®)

GlaxoSmithKline

COPD

IV, SC

Phase 3 − sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

IV

Phase 3 − sNDA; QIDP

TBD

mitapivat (Pyrukynd®)

Agios

SCD; Thalassemia

Oral

Phase 3 − sNDA; Orphan Drug

TBD

nitazoxanide (Alinia®)

Lupin

COVID-19; Influenza

Oral

Phase 3 − sNDA

TBD

omalizumab (Xolair)

Genentech

Food allergies

SC

Phase 3 − sBLA; Breakthrough Therapy

TBD

patisiran (Onpattro®)

Alnylam

Transthyretin amyloid cardiomyopathy (wild type or hereditary)

IV

Phase 3 − sNDA; Orphan Drug

TBD

pegcetacoplan (Empaveli)

Apellis

Autoimmune hemolytic anemia (warm)

SC

Phase 3 − sNDA; Orphan Drug

TBD

rimegepant (Nurtec ODT®)

Pfizer

Chronic rhinosinusitis; Nasal polyposis

Oral

Phase 3 − sNDA

TBD

risankizumab-rzaa (Skyrizi)

Abbvie

UC

IV, SC

Phase 3 − sBLA

TBD

rivaroxaban (Xarelto )

Janssen

COVID-19

Oral

Phase 3 − sNDA

TBD

roflumilast (Zoryve)

Arcutis

Atopic dermatitis

Topical

Phase 3 − sNDA

TBD

®

45 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

romiplostim (Nplate®)

Amgen

Chemotherapy-induced thrombocytopenia

SC

Phase 3 − sBLA; Orphan Drug

TBD

ropeginterferon alfa-2b -njft (Besremi®)

Pharmaessentia

Essential thrombocythemia

SC

Phase 3 − sBLA; Orphan Drug

TBD

talazoparib (Talzenna®)

Pfizer

Prostate cancer (metastatic, castrationresistant, in combination with enzalutamide)

Oral

Phase 3 − sNDA

TBD

tapinarof (Vtama®)

Roivant

Atopic dermatitis

Topical

Phase 3 − sNDA

TBD

tezepelumab-ekko (Tezspire™)

Amgen

Chronic rhinosinusitis; Nasal polyposis

SC

Phase 3 − sBLA

TBD

tirzepatide (Mounjaro™)

Eli Lilly

Obesity; Overweight with weight-related comorbidities

SC

Phase 3 − sNDA; Fast Track

TBD

upadacitinib (Rinvoq)

Abbvie

Giant cell arteritis

Oral

Phase 3 − sNDA

TBD

Complete Response Letter (CRL) NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

bulevirtide

Gilead

Hepatitis D infection treatment (with compensated liver disease)

SC

CRL

TBD

donanemab

Eli Lilly

Alzheimer’s disease (early)

IV

CRL

TBD

omburtamab

Y-mAbs

Brain cancer (CNS/ leptomeningeal metastasis from neuroblastoma)

Intracerebroventricular

CRL

TBD

palovarotene

Ipsen

Fibrodysplasia ossificans Oral progressiva (prevention of heterotropic ossification)

CRL

TBD

poziotinib

Spectrum

NSCLC (HER2 exon 20 insertion mutations, locally advanced or metastatic, previously treated)

Oral

CRL

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Sandoz

Breast cancer; Gastric/ gastroesophageal cancer

IV

CRL

TBD

46 | MAGELLANRX.COM

GLOSSARY 6MWT 6 Minute Walking Test

CAP Community Acquired Pneumonia

ABSSSI Acute Bacterial Skin and Skin Structure Infection

CAR T Chimeric Antigen Receptor T Cell

ACC American College of Cardiology

CDC Centers for Disease Control and Prevention

ACEI Angiotensin-Converting Enzyme Inhibitor ACR20 American College of Rheumatology 20% Improvement ACR50 American College of Rheumatology 50% Improvement

CD Crohn's Disease CF Cystic Fibrosis CHF Congestive Heart Failure CI Confidence Interval CKD Chronic Kidney Disease

ACR70 American College of Rheumatology 70% Improvement

CLL Chronic Lymphocytic Leukemia

ADC Antibody-Drug Conjugate

CMS Centers for Medicare & Medicaid Services

ADHD Attention Deficit Hyperactivity Disorder

CNS Central Nervous System

ADL Activities of Daily Living

COPD Chronic Obstructive Pulmonary Disease

AED Anti-Epileptic Drug

COVID-19 Coronavirus Disease 2019

AHA American Heart Association

CRC Colorectal Cancer

ALK Anaplastic Lymphoma Kinase

CRL Complete Response Letter

ALL Acute Lymphoblastic Leukemia

CRR Complete Response Rate

ALS Amyotrophic Lateral Sclerosis

CSF Colony Stimulating Factor

ALT Alanine Transaminase

CV Cardiovascular

AMD Age-Related Macular Degeneration

CVD Cardiovascular Disease

AML Acute Myeloid Leukemia ANCA Antineutrophil Cytoplasmic Antibodies

DAS28-CRP Disease Activity Score-28 with C Reactive Protein

ANDA Abbreviated New Drug Application

DCR Disease Control Rate

ARB Angiotensin II Receptor Blocker

DEA Drug Enforcement Administration

ARNI Angiotensin Receptor-Neprilysin Inhibitor

DED Dry Eye Disease

ART Antiretroviral Therapy

DLBCL Diffuse Large B Cell Lymphoma

ARV Antiretroviral

DMARD Disease Modifying Antirheumatic Drug

AS Ankylosing Spondylitis

DMD Duchenne Muscular Dystrophy

ASCVD Atherosclerotic Cardiovascular Disease

DME Diabetic Macular Edema

AST Aspartate Aminotransferase

DMT Disease Modifying Therapy

BCG Bacillus Calmette-Guérin

DNA Deoxyribonucleic Acid

BCVA Best Corrected Visual Acuity

DOR Duration of Response

BLA Biologics License Application

DPI Dry Powder for Inhalation

BMI Body Mass Index

DPP-4 Dipeptidyl Peptidase 4

BMT Bone Marrow Transplant

DR Delayed-Release

BPH Benign Prostatic Hyperplasia

EASI-75 Eczema Area and Severity Index ≥ 75% Reduction

BSA Body Surface Area BsUFA Biosimilar User Fee Act CABP Community Acquired Bacterial Pneumonia

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CML Chronic Myeloid Leukemia

ECOG Eastern Cooperative Oncology Group EDSS Expanded Disability Status Scale

GLOSSARY continued eGFR estimated Glomerular Filtration Rate

HSV Herpes Simplex Virus

EGFR Epidermal Growth Factor Receptor

HTN Hypertension

ER Extended-Release

IBS Irritable Bowel Syndrome

ESA Erythropoietin Stimulating Agent

IBS-C Irritable Bowel Syndrome, Constipation Predominant

ESRD End-Stage Renal Disease

ICU Intensive Care Unit

EUA Emergency Use Authorization

IDSA Infectious Diseases Society of America

FDA Food and Drug Administration

IGA Investigator's Global Assessment

FH Familial Hypercholesterolemia

IgG Immunoglobulin G

FLT3 FMS-Like Tyrosine Kinase-3

IL-12 Interleukin-12

FMS Feline McDonough Sarcoma

IL-17 Interleukin-17

G-CSF Granulocyte Colony Stimulating Factor

IL-23 Interleukin-23

GI Gastrointestinal

IM Intramuscular

GIST Gastrointestinal Stromal Tumor

IR Immediate-Release

GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist

IRB Internal Review Board

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

ITP Immune Thrombocytopenic Purpura

GVHD Graft Versus Host Disease

IV Intravenous

H Half HAART Highly Active Antiretroviral Therapy HAE Hereditary Angioedema HAM-D Hamilton Depression Rating Scale

ITT Intention-To-Treat JIA Juvenile Idiopathic Arthritis LDL Low-Density Lipoprotein LDL-C Low-Density Lipoprotein Cholesterol

HAP Healthcare-Associated Pneumonia

LPAD Limited Population Pathway for Antibacterial and Antifungal Drugs

Hb Hemoglobin

LVEF Left Ventricular Ejection Fraction

HbA1c Hemoglobin A1c

mAb Monoclonal Antibody

HBV Hepatitis B Virus

MACE Major Adverse Cardiovascular Events

HCC Hepatocellular Carcinoma

MADRS Montgomery – Åsberg Depression Rating Scale

HCP Healthcare Professional

MAOI Monoamine Oxidase Inhibitor

HCV Hepatitis C Virus

MDD Major Depressive Disorder

HER Human Epidermal Growth Factor Receptor

MDI Metered Dose Inhaler

HER2 Human Epidermal Growth Factor Receptor 2

MDR Multi-Drug Resistant

HF Heart Failure

MECP2 Methyl-CpG Binding Protein 2

HFA Hydrofluoroalkane

mITT modified Intention-To-Treat

HFpEF Heart Failure with preserved Ejection Fraction

MRI Magnetic Resonance Imaging

HFSA Heart Failure Society of America

MRSA Methicillin-Resistant Staphylococcus Aureus

HIT Heparin Induced Thrombocytopenia

MS Multiple Sclerosis

HIV Human Immunodeficiency Virus

N/A Not Applicable

HIV-1 Human Immunodeficiency Virus-1

NASH Non-Alcoholic Steatohepatitis

HPV Human Papilloma Virus

NCCN National Comprehensive Cancer Network

HR Hazard Ratio

NCT National Clinical Trials

HSCT Hematopoietic Stem Cell Transplant

NDA New Drug Application

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GLOSSARY continued NHL Non-Hodgkin Lymphoma NIAID National Institute of Allergy and Infectious Diseases

R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone RA Rheumatoid Arthritis

NSAID Non-Steroidal Anti-Inflammatory Drug

RBC Red Blood Cell

NSCLC Non-Small Cell Lung Cancer

RCC Renal Cell Carcinoma

NYHA New York Heart Association

REMS Risk Evaluation and Mitigation Strategy

ODT Orally Disintegrating Tablet

RMAT Regenerative Medicine Advanced Therapy

OR Odds Ratio

RNA Ribonucleic Acid

ORR Overall/Objective Response Rate

RPD Rare Pediatric Disease

OS Overall Survival

RRR Relative Risk Reduction

OTC Over-the-Counter

RSV Respiratory Syncytial Virus

PAH Pulmonary Arterial Hypertension

RTOR Real-Time Oncology Review

PARP Poly(ADP-ribose) Polymerase

RVO Retinal Vein Occlusion

PAS Prior Approval Supplement PASI Psoriasis Area and Severity Index

SARS-CoV-2 Severe Acute Respiratory SyndromeAssociated Coronavirus-2

PASI 50 Psoriasis Area and Severity Index 50% Reduction

sBLA supplemental Biologics License Application SC Subcutaneous

PASI 75 Psoriasis Area and Severity Index 75% Reduction

SCCHN Squamous Cell Cancer of the Head and Neck

PASI 90 Psoriasis Area and Severity Index 90% Reduction

SCLC Small Cell Lung Cancer

PASI 100 Psoriasis Area and Severity Index 100% Reduction

SCD Sickle Cell Disease SCT Stem Cell Transplant SGLT2 Sodium-Glucose Co-Transporter 2

PCI Percutaneous Coronary Intervention

SL Sublingual

PCSK9 Proprotein Convertase Subtilisin Kexin 9

SLE Systemic Lupus Erythematosus

PD-1 Programmed Death Protein 1

SLL Small Lymphocytic Lymphoma

PD-L1 Programmed Death-Ligand 1

sNDA supplemental New Drug Application

PDUFA Prescription Drug User Fee Application

SNRI Serotonin and Norepinephrine Reuptake Inhibitor

PFS Progression-Free Survival PGA Physician Global Assessment PI3K Phosphatidylinositol-3-kinase PNH Paroxysmal Nocturnal Hemoglobinuria PsA Psoriatic Arthritis PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty

SOC Standard of Care sPGA static Physician Global Assessment SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection T1DM Type 1 Diabetes Mellitus T2DM Type 2 Diabetes Mellitus

PTSD Post-Traumatic Stress Disorder

TBD To Be Determined

Q Quarter

TEAE Treatment-Emergent Adverse Event

QIDP Qualified Infectious Diseases Product

TNBC Triple Negative Breast Cancer

QOL Quality of Life

TNF Tumor Necrosis Factor

R/R Relapsed or Refractory

TNFα Tumor Necrosis Factor-alpha UA Unstable Angina

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GLOSSARY continued UC Ulcerative Colitis US United States UTI Urinary Tract Infection VAS Visual Analog Scale VEGF Vascular Endothelial Growth Factor VTE Venous Thromboembolism WBC White Blood Cell WHO World Health Organization XR Extended-Release

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MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS

JANUARY 2022

2023 Magellan Rx Management, LLC. All rights reserved. MRX1119_0123