MRx Pipeline - October 2020

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October 2020

MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS


TABLE OF CONTENTS Introduction Pipeline Deep Dive

EDITORIAL STAFF Maryam Tabatabai, PharmD Editor in Chief Senior Director, Drug Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist

Keep on Your Radar

Consultant Panel

Pipeline Drug List

Michelle Booth, PharmD Director, Medical Pharmacy Strategy

Daphne Atria, PharmD, BCPS, CPE Strategic Clinical Pharmacist Consultant

Becky Borgert, PharmD, BCOP Director, Clinical Oncology Product Development

Glossary

Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist Robert Greer, RPh, BCOP Senior Director, Clinical Strategy and Programs Brian MacDonald, PharmD Senior Manager, Specialty Clinical Programs Troy Phelps Senior Director, COAR - Analytics

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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INTRODUCTION 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, agents under the pharmacy and medical benefits, new molecular entities, pertinent new and expanded indications for existing medications, and biosimilars are profiled in the report. Clinical analyses, financial outlook, and pre-regulatory status are considered as part of the evaluation process. The products housed in the MRx Pipeline have been researched in detail and developed in collaboration and in consultation with our internal team of clinical and analytics experts. 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 2024. 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, such as the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business. So far in 2020, a total of 42 novel drugs have received FDA approval, including the first treatment for COVID-19. Last year at approximately the same timepoint, only 33 novel drugs had been approved, already representing a 21% increase in approvals in 2020. For the remainder of this year, 26 notable drugs filed with the agency are profiled, each of which has an anticipated FDA decision in 2020. The progress of these agents is being actively monitored through MRx Pipeline. In the past few years, game changers, such as chimeric antigen receptor (CAR) T therapies, have transformed the pipeline by delivery of drug therapy directly to the site of action in the body. As we look ahead, a continued trend toward the approval of specialty medications, drugs for rare and ultra rare diseases, growth of biosimilars, new treatment modalities using gene therapy, and additional CAR-T therapies are expected. Noteworthy pipeline trends to watch in 2020 include much-needed therapies and vaccines for COVID-19. There are also a number of first-time approvals anticipated in 2020 – the first-in-class oral agent for anemia in chronic kidney disease, first treatment for genetic deficiency obesity, first oral agent for hereditary angioedema, and the first injectable for dyslipidemia administered every 6 months. In the upcoming quarters, development of complex therapies, therapeutic options for rare hereditary diseases, oncology, immunology, neurology, and investigational agents will be on the MRx radar. Moreover, sprouting products for cardiology, ophthalmology, hematology, and women’s health await on the horizon. The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Novel agents that apply innovation to show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm.

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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

88%

47% BIOSIMILAR

47%

BREAKTHROUGH THERAPY

12% ORPHAN DRUG

18%

pecialty drug names appear in ï‚« S magenta throughout the publication.

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NEUROLOGY

aducanumab IV Biogen/Eisai PROPOSED INDICATION Alzheimer’s disease (AD)

CLINICAL OVERVIEW

Aducanumab is a human monoclonal antibody that selectively binds to amyloid beta fibrils and soluble oligomers. Two identical 18-month, double-blind, phase 3 trials, EMERGE and ENGAGE, evaluated the efficacy and safety of aducanumab in patients 50 to 85 years of age with early AD (defined as mild cognitive impairment [MCI] due to AD and mild AD dementia). A total of 3,285 patients were randomized 1:1:1 to one of two dosing regimens of aducanumab (low-dose group, 3 mg/kg in patients who carry apolipoprotein E-e4 [APO-e4] gene variant or 6 mg/kg in patients who do not carry APO-e4; high-dose group, 6 mg/kg in patients who carry APOE-e4 or 10 mg/kg in patients who do not) or to placebo; at 18-months, the dose was increased to 10 mg/kg in all APO-e4 carriers. In March 2019, the initial interim analysis based on data from 1,748 patients who completed 18-months of therapy predicted that both trials would not meet their primary endpoint of Clinical Dementia Rating–Sum of Boxes (CDR-SB) score; this led to termination of both trials. However, further analyses revealed EMERGE demonstrated a significant reduction in CDR-SB with the highest dose of aducanumab 10 mg/kg compared to placebo, which was in part due to study protocol amendments that allowed more patients to receive the 10 mg/kg dose. Consequently, the trials were resumed. With 3 additional months of study data in the EMERGE trial, the high-dose aducanumab regimen demonstrated a significant 22% reduction in clinical decline (based on CDR-SB) compared to placebo (p=0.01). In addition, significant differences favoring the high-dose aducanumab versus placebo were seen with the following secondary measures: Alzheimer’s Disease Assessment Scale–Cognitive Subscale (difference, 27%; p=0.01), Alzheimer’s Disease Cooperative Study–Activities of Daily Living Inventory–Mild Cognitive Impairment (difference, 40%; p=0.001), and Mini Mental State Exam (difference, 18%; p=0.05). In ENGAGE, while trends in slowing decline of AD were seen with some secondary study endpoints, the aducanumab high-dose regimen failed to demonstrate a significant benefit in the primary endpoint of CDR-SB. In EMERGE and ENGAGE (reported as incidence in each trial, respectively), the most common TEAE with the aducanumab 10 mg/kg dose was transient amyloid-related imaging abnormalities-edema/effusion (ARIA-E; 34% and 35.5%), headache (19.4% and 20.4%), and amyloid-related imaging abnormalitiesmicrohemorrhage (ARIA-H; 18.6% and 17.6%). In the trials, 8.8% and 11.5% of patients, respectively, permanently discontinued treatment due to TEAEs, of which 6.6% and 7.3%, respectively, were due to ARIA. In each study, the incidence of all-cause death was low among patients treated with high-dose aducanumab (1.1% and 0.4%, respectively). Aducanumab was infused IV once monthly.

PLACE IN THERAPY

Alzheimer’s disease is a neurodegenerative disorder characterized by cognitive and memory decline, impairment of ADLs, and behavioral disturbances. It is the most common form of dementia. It is estimated to affect 5 million elderly people in the US, and the incidence is expected to nearly triple by 2050. Moreover, the presence of the APOE-e4 gene variant is considered a risk factor for developing AD. The gene variant is found in approximately 28% of the general public, and it is estimated to exist in 40% to 65% of patients diagnosed with AD. Presence of amyloid plaque as well as abnormalities of cholinergic, glutamate, and serotonin systems in the brain are thought to contribute to the development of AD. Current pharmacotherapy for AD includes oral acetylcholinesterase inhibitors (donepezil, galantamine, rivastigmine [also transdermal]), oral N-methylD-aspartate (NMDA) receptor antagonists (memantine), and a fixed-dose combination of the two classes (donepezil/memantine); all single-agent products are available as generics. Although troubled by mixed clinical results, if approved, aducanumab will be the first agent to treat early AD that reduces amyloid plaque. However, clearance of plaque is associated with vasogenic brain edema that limits the tolerable dose and 4 | magellanrx.com


aducanumab cont. PLACE IN THERAPY cont.

can lead to treatment discontinuation, particularly in patients with the APOE-e4 gene variant. As part of the FDA approval process, on November 6, 2020, the Peripheral and Central Nervous System Drugs Advisory Committee will review data for aducanumab as it relates to its clinical benefits and risks. Other agents that target amyloid beta protein in late-stage development for AD include gantenerumab, solanezumab, and BAN2401.

FDA APPROVAL TIMELINE March 7, 2021 ďƒź Fast Track

ďƒź Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$0

$253

$595

$1,051

$1,651

The forecast is a projection of total US sales per year.

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MUSCULOSKELETAL

casimersen IV Sarepta PROPOSED INDICATION

Duchenne muscular dystrophy (DMD) with mutations amenable to exon 45 skipping

CLINICAL OVERVIEW

Casimersen is a phosphorodiamidate morpholino oligomer (PMO) that binds to exon 45 of dystrophin premRNA. The ongoing, double-blind, placebo-controlled, phase 3 ESSENCE trial is evaluating the safety and efficacy of casimersen in male patients with DMD. The baseline mean dystrophin protein level was 0.925% of normal. Based on interim data, casimersen therapy has resulted in a significant increase in mean dystrophin protein level to 1.736% of normal (p<0.001 compared to baseline); statistically significant differences from placebo have been observed at 48 weeks (p=0.009). Changes in functional abilities (e.g., 6MWT) and safety data were reported with this interim analysis. After 96 weeks of therapy, patients in the study are allowed to enter a 48-week, open-label extension phase during which all patients will receive active treatment. In the clinical trial, patients received an IV infusion of casimersen 30 mg/kg once per week for up to 96 weeks.

PLACE IN THERAPY

DMD is a rare, X-linked neuromuscular disorder characterized by progressive muscle degeneration and weakness. An estimated 400 to 600 boys 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 years. Moreover, death due to respiratory or cardiac failure typically occurs before age 30. The standard of care for DMD includes systemic corticosteroids (prednisone, deflazacort [Emflaza®; the only FDA-approved steroid for DMD]) to delay progression of muscle weakness and improve respiratory function; however, side effects such as weight gain, bone fractures, and cataracts are associated with corticosteroid therapy. Approximately 8% of individuals diagnosed with DMD carry the exon 45 mutation. Casimersen allows sections of defective genetic code with the exon 45 mutation to be bypassed during the dystrophin manufacturing process, creating a partially functional dystrophin protein. Sarepta brought the first antisense oligonucleotide treatments for DMD to the US market, including eteplirsen (Exondys 51®) and golodirsen (Vyondys 53™), which target exons 51 and 53, respectively. Viltolarsen (Viltepso™) by Nippon Shinyaku is also approved and targets exon 53. Interim data reveals that casimersen increases the surrogate marker of mean dystrophin protein by 0.811 percentage points; this increase is in line with the the change in dystrophin protein that supported golodirsen’s FDA approval. Sarepta is seeking Accelerated Approval for casimersen. If casimersen is approved, it will be the first antisense oligonucleotide available in the US to treat DMD with mutations amenable to exon 45 skipping.

FDA APPROVAL TIMELINE February 25, 2021  Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$0

$94

$136

$157

$187

The forecast is a projection of total US sales per year.

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CARDIOLOGY

inclisiran SC Novartis/The Medicines Company PROPOSED INDICATION

Treatment of dyslipidemia for secondary prevention of patients with atherosclerotic cardiovascular disease (ASCVD) and familial hypercholesterolemia (FH)

CLINICAL OVERVIEW

Inclisiran is a small interfering RNA (siRNA) therapeutic agent that inhibits hepatic production of PCSK9. The randomized, double-blind, placebo-controlled, phase 3 ORION trial program demonstrated LDL-C lowering with inclisiran in addition to background antilipid therapy in patients with ASCVD or FH and elevated LDL-C despite maximally tolerated statin therapy. In the ORION-10 (n=1,561) and ORION-11 (n=1,617) trials, inclisiran reduced LDL-C (primary endpoint) by 51.3% and 49.9%, respectively, compared to placebo at day 510 (p<0.001 for both). Decreased total cholesterol, non-HDL-C, apolipoprotein B, and triglycerides, as well as increased HDL-C, were reported with inclisiran compared to placebo. In patients with heterozygous FH (HeFH), the ORION-9 trial (n=482) reported a 47.9% reduction in LDL-C with inclisiran compared to placebo (p<0.001). The most common adverse effect reported with inclisiran was transient mild to moderate injection site reactions. In ORION-10 and ORION-11, overall deaths occurred at similar rates with inclisiran and placebo, and a prespecified analysis reported a lower rate of a CV composite endpoint with inclisiran compared to placebo (inclisiran, 7.4% and 7.8%, respectively; placebo, 10.2% and 10.3%, respectively); however, data were too limited to establish meaningful CV outcome conclusions. Inclisiran was HCP-administered as SC doses of 284 mg on days 1 and 90, and every 6 months thereafter.

PLACE IN THERAPY

CVD is the leading cause of death in the US. Individuals with established ASCVD are at high risk for a CV event. Also, FH occurs in approximately 830,000 Americans and is characterized by an elevated LDL-C and premature ASCVD. LDL-C lowering is a main objective of secondary prevention. Statins are the drugs of choice to reduce LDL-C levels, but their use is limited by resistance and intolerance to therapy. When target LDL-C is not achieved despite optimized oral statin therapy, oral ezetimibe or bempedoic acid (Nexletol®) or an injectable PCSK9-inhibiting mAb (alirocumab [Praluent®], evolocumab [Repatha®]) may be added. The phase 3 ORION program demonstrated robust LDL-C lowering with inclisiran as adjunct to background antilipid therapy. If approved, inclisiran will be the first-in-class siRNA-targeting PCSK9 agent and an alternative to PCSK9-inhibiting mAbs in patients who are not at goal with lifestyle modification and maximally tolerated statin therapy. Inclisiran offers every 6-month HCP-administered SC maintenance dosing, while PCSK9-inhibiting mAbs allow for once-monthly SC self-administration. Although there are no head-to-head phase 3 trials between the 2 antilipid classes, noncomparative study data report slightly less LDL-C lowering with inclisiran (range, 48% to 53%) versus PCSK9-inhibitor mAbs (range, 55% to 59%). Unlike statins, no liver, muscle, or kidney toxicity or need for dose adjustments in patients with renal impairments have been reported with inclisiran. While the PCSK9-inhibiting mAbs demonstrate risk reduction of CV events, CV outcomes trials with inclisiran are ongoing. In the United Kingdom (UK), Novartis is collaborating with the UK National Health Service on CV outcomes, and inclisiran will be provided to UK patients for secondary prevention through a population-level agreement pending UK regulatory approval and National Institute for Health and Care Excellence (NICE) assessment.

FDA APPROVAL TIMELINE December 2020  Orphan Drug

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$0

$136

$248

$376

$575

The forecast is a projection of total US sales per year.

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ONCOLOGY

naxitamab IV Y-mAbs Therapeutics PROPOSED INDICATION

Relapsed/refractory, high-risk neuroblastoma

CLINICAL OVERVIEW

Naxitamab is a humanized 3F8 monoclonal antibody (mAb) that targets ganglioside GD2, a cell surface antigen found on neuroblastoma tumors. Cytokines, such as GM-CSF, enhance the antibody-dependent cellular cytotoxicity (ADCC). Submission to the FDA for naxitamab was supported by a phase 2 clinical study (12-230) in patients with high-risk neuroblastoma. Patients were stratified into 3 groups. Among patients with primary refractory disease (n=28), naxitamab therapy resulted in an ORR of 78% and a 2-year PFS rate of 50%. In patients who relapsed and were resistant to salvage therapy (n=30), naxitamab led to an ORR of 37% and PFS of 36%. Lastly, in patients in second or later complete remission (n=44), naxitamab demonstrated a 52% PFS rate; however, this group was not evaluable for ORR. All patients received naxitamab 3 mg/kg administered IV over 30 minutes on days 1, 3, and 5 of each cycle. GM-CSF was also administered per the study protocol. Treatment was continued for 5 monthly cycles after complete response or very good partial response was achieved. Treatment was administered in an outpatient setting.

PLACE IN THERAPY

Neuroblastoma is a solid cancer that originates in immature neurons (neuroblasts) of the sympathetic nervous system. The rare disease accounts for approximately 6% of all childhood cancers and is the most common cancer in infants. The average age at diagnosis is 1 to 2 years, with the majority (90%) of cases identified by ≤ 5 years of age. Metastatic disease is often detected at diagnosis. Aggressive treatment is used for high-risk neuroblastoma. Induction and consolidation therapies include surgery, high-dose chemotherapy, radiation, and autologous stem cell transplant, while maintenance therapy includes isotretinoin, immune-activating cytokines (GM-CSF, interleukin-2), and GD2-targeting mAbs. If approved, naxitamab will be the second GD2-targeting mAb available to treat high-risk neuroblastoma, following dinutuximab (Unituxin®), a chimeric anti-GD2 mAb. Dinutuximab, given in combination with GMCSF, intereukin-2, and isotretinoin, is associated with serious infusion reactions, neurotoxicity, and antidrug antibodies (ADA; 20%). Naxitamab has high affinity for GD2 and was designed to reduce ADAs and boost ADCC while retaining complement mediated cytotoxicity (CMC). Low-immunogenicity with the product has been confirmed, and it appears to have an improved safety profile. Additionally, naxitamab may offer a more convenient administration regimen compared to dinutuximab; naxitamab is infused IV over 30 minutes as 3 doses/cycle, compared to 4 doses/cycle administered IV over 10 to 20 hours for dinutuximab. Notably, naxitamab has the potential to be administered on an outpatient basis, while dinutuximab must be given in a hospital setting.

FDA APPROVAL TIMELINE November 30, 2020

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$7

$27

$63

$111

$165

The forecast is a projection of total US sales per year.

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 Rare Pediatric Disease


ONCOLOGY

paclitaxel/encequidar oral Athenex PROPOSED INDICATION Breast cancer

CLINICAL OVERVIEW

Athenex's oral formulation of paclitaxel, a microtubule inhibitor, is co-administered with encequidar, a GI tract specific P-glycoprotein (P-gp) pump inhibitor that is designed to allow for oral absorption of paclitaxel. In a pivotal phase 3 trial, oral paclitaxel/encequidar (OPE) was compared to IV paclitaxel in patients with metastatic breast cancer, including patients with HR+/HER2-, HR+/HER2+, TNBC, and unknown HR/HER2 receptor status. Patients were randomized 2:1 to monotherapy with oral and IV formulations, respectively. In the intent-to-treat population (n=360), the primary endpoint of overall tumor response rate (ORR) was significantly higher with OPE compared to the IV paclitaxel (40.4% versus 25.6%; p=0.01). While analyses are ongoing, to date, the reported median duration of confirmed response is 39 weeks with OPE and 30.1 weeks with IV paclitaxel, and overall survival and PFS are favoring the oral over the IV formulation (p=0.035 and p=0.77, respectively). OPE was associated with less neuropathy; however, higher rates of hematologic adverse effects, including grade 3 neutropenia (14.8% versus 6.9% with IV paclitaxel) and one incidence of grade 5 anemia were reported with OPE. GI adverse events were more prevalent with the oral formulation compared to IV paclitaxel, including grade 3 diarrhea (4.9% versus 1.5%), nausea (3.8% versus 0.2%), and vomiting (4.9% versus 0.7%); the study protocol was amended to allow for use of medications to mitigate GI toxicities. OPE was administered 3 consecutive days per week as a 30 mg capsule of solubilized paclitaxel plus a 15 mg tablet of encequidar. The IV formulation was given every 3 weeks.

PLACE IN THERAPY

It is estimated that 279,100 people in the US will be diagnosed with breast cancer and 42,690 will die from the disease in 2020. It is the most common malignancy and second leading cause of death in American women. Taxanes are the cornerstone of breast cancer treatment. Paclitaxel is currently approved as an IV formulation for the treatment of metastatic breast cancer after failure of anthracycline-containing chemotherapy as well as first-line treatment for advanced or metastatic NSCLC and metastatic pancreatic cancers. In the pivotal trial, OPE demonstrated significant improvement in ORR compared to IV paclitaxel in patients with metastatic breast cancer. OPE leads to significantly less peripheral neuropathy, a long-term and potentially debilitating effect of IV paclitaxel. OPE is, however, associated with short-term hematologic and GI adverse effects. Intravenous paclitaxel is administered in combination with chemotherapy and/or immunomodulator therapy as every 1-week (fewer side effects) or 3-week regimens. OPE will replace IV paclitaxel within the chemotherapy regimen. It is also anticipated to reduce the need for adjunctive pretreatment with corticosteroids, which are typically given to mitigate the hypersensitivity reactions of IV paclitaxel solubilized with cremophor. OPE does have a complex oral administration regimen that requires fasting for 9 hours per day, 3 consecutive days per week. Oral paclitaxel is in phase 2 trials for cutaneous angiosarcoma and advanced solid tumors. The oral formulation has not been compared in clinical trials to the solvent-free IV nanoparticle albumin-bound (nab)-paclitaxel (AbraxaneÂŽ) formulation designed to reduce solvent-related side effects and improve tumor uptake.

FDA APPROVAL TIMELINE February 28, 2021 ďƒź Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$0

$33

$116

$218

$322

The forecast is a projection of total US sales per year.

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ONCOLOGY/WOMEN’S HEALTH

relugolix oral Myovant

PROPOSED INDICATIONS

• Advanced prostate cancer (PC) • Uterine fibroid (UF)-related heavy menstrual bleeding

CLINICAL OVERVIEW

Relugolix is a gonadotropin-releasing hormone (GnRH) receptor antagonist. It reduces the production of testosterone and estradiol, hormones that stimulate growth of prostate cancer and uterine fibroids, respectively. Prostate cancer The 48-week, open-label, phase 3 HERO trial compared the safety and efficacy of relugolix with leuprolide in 930 men with androgen-sensitive advanced PC. Patients were randomized 2:1 to oral relugolix 360 mg on day 1, followed by 120 mg once daily, or SC leuprolide depot 22.5 mg every 3 months. After 48 weeks of therapy, relugolix was superior to leuprolide in maintaining testosterone suppression to castration levels (< 50 ng/dL) (96.7% versus 88.8%, respectively; p<0.001 for superiority). The study also reported similar rates of castration resistance-free survival between the groups (74% versus 75%, respectively). Greater testosterone recovery was seen with relugolix compared to leuprolide (testosterone level, 270.76 ng/dL versus 12.26 ng/dL, respectively) 90 days after therapy discontinuation. In addition, the risk of MACE was lower with relugolix versus leuprolide (2.9% versus 6.2%, respectively), including in men with a history of MACE (3.6% versus 17.8%, respectively). The overall safety profile was similar between the groups. In a phase 2 trial, the rate of sustained castration (testosterone < 50 ng/dL) was 93% with relugolix and 85% with the GnRH receptor antagonist degarelix with 24 weeks of therapy. Testosterone recovery was also more rapid with relugolix compared to degarelix (257 ng/dL versus 30 ng/dL, respectively) 12 weeks after therapy discontinuation. Uterine fibroids The 24-week, double-blind, phase 3 LIBERTY-1 and LIBERTY-2 trials assessed the safety and efficacy of a once-daily oral combination of relugolix 40 mg/estradiol 1 mg/norethindrone 0.5 mg for use in 770 premenopausal women with heavy menstrual bleeding related to UF. Response to therapy was defined as menstrual blood loss (MBL) < 80 mL and a ≥ 50% MBL reduction from baseline (primary endpoint). Both trials demonstrated that relugolix combination therapy led to a significantly greater response rate compared to placebo (LIBERTY-1, 73.4% versus 18.9%, respectively; LIBERTY-2, 71.2% versus 14.7%, respectively; p<0.0001 for both). In both trials, combination therapy resulted in a 84.3% mean reduction in MBL from baseline (p<0.0001). Secondary endpoints significantly favored relugolix combination therapy, including reduction in UF pain, improved anemia, change in uterine volume, and improved QOL; however, changes in UF volume and bone density did not differ significantly between the groups. Relugolix combination therapy was generally well tolerated.

PLACE IN THERAPY Prostate cancer

Prostate cancer is the second most common cancer in men in the US, with an anticipated 191,930 new cases and 33,330 deaths attributed to the disease in 2020. Treatment decisions should be individualized, based on disease stage/grade, and health, life expectancy, and patient preference. The majority of prostate cancers are hormonally dependent. Due to the hormone responsiveness of the tumor, androgen deprivation therapy (ADT) is a cornerstone of PC treatment, particularly for advanced or metastatic disease. ADT suppresses serum testosterone concentrations to castration levels (< 50 ng/dL) and can be accomplished with either bilateral orchiectomy or with the administration of a GnRH agonist (e.g., leuprolide [Lupron®, Eligard®], goserelin [Zoladex®], histrelin [Vantas®], triptorelin [Trelstar®]) or a GnRH antagonist (e.g., degarelix [Firmagon®]). ADT may be combined with radiation therapy for locally advanced PC.

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relugolix cont. PLACE IN THERAPY cont.

Relugolix is a GnRH antagonist, and unlike GnRH agonists, is not associated with testosterone flare due to the rise and fall of testosterone levels in patients with PC. Notably, relugolix's once-daily oral formulation may provide treatment flexibility and may be preferred over the injectable (IM, SC) formulations for the GnRH agonists administered every 1, 3, 4, or 6 months (depending on formulation). Goserelin is available as a 12-month SC implant. The GnRH antagonist degarelix is HCP-administered SC once monthly. Uterine fibroids Uterine fibroids (UFs) are the most common benign gynecologic tumors. They affect approximately 50% to 60% of premenopausal women and are more common among Black or African American women (80%). UFs are caused by overgrowth of connective tissue and smooth muscle in the uterus. While most cases are asymptomatic, UFs can lead to heavy menstrual bleeding and pain and can cause infertility. Treatment approaches include surgery (myomectomy, hysterectomy, uterine artery embolization) and medical therapies, which include oral contraceptives and progestin-releasing intrauterine devices (IUDs) to reduce heavy menstrual bleeding, NSAIDs to reduce pain, and mifepristone and GnRH agonists to shrink the fibroids. GnRH agonists are associated with bone loss and vasomotor symptoms that may be mitigated with low-dose “add-back” therapy using a combination of estrogen and progestin. In addition, tranexamic acid (Lysteda®) is approved for the treatment of cyclic heavy menstrual bleeding; it is dosed 3 times daily, and given for a maximum of 5 days during monthly menstruation. If approved for UF, relugolix will be the second oral GnRH antagonist combined with estradiol and norethindrone, following elagolix/estradiol/norethindrone (Oriahnn™), for the management of heavy menstrual bleeding associated with UFs in premenopausal women.

FDA APPROVAL TIMELINE

Prostate cancer – December 20, 2020  Priority Review Uterine fibroids – June 1, 2021

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$0

$17

$91

$181

$273

The forecast is a projection of total US sales per year.

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ONCOLOGY

trilaciclib IV G1 Therapeutics/Boehringer Ingelheim PROPOSED INDICATION

Myelopreservation in small cell lung cancer (SCLC) patients being treated with chemotherapy

CLINICAL OVERVIEW

Trilaciclib is a cyclin-dependent kinase 4 and 6 (CDK4/6) inhibitor that protects hematopoietic stem and progenitor cells by preventing proliferation during chemotherapy administration. Three double-blind, phase 2 trials evaluated the safety and efficacy of trilaciclib in patients with extensive stage SCLC. Patients were randomized to trilaciclib (n=123) or placebo (n=119) as add-on to standard chemotherapy that comprised etoposide/carboplatin ± atezolizumab, or topotecan. Pooled data from the 3 trials revealed trilaciclib resulted in a significant (p<0.05) decrease in myelosuppression as evident by incidence of the following (versus placebo, respectively): mean duration of severe neutropenia in cycle 1 (0 versus 4 days), severe neutropenia (11.4% versus 52.9%), G-CSF administration (28.5% versus 56.3%), and grade 3/4 thrombocytopenia (19.5% versus 36.1%). The overall survival and PFS were similar between the groups. Trilaciclib was administered IV prior to chemotherapy.

PLACE IN THERAPY

It is estimated that 228,820 new cases of lung cancer will be diagnosed in the US in 2020, of which approximately 13% will be SCLC. Nearly all cases of SCLC are due to cigarette smoking. SCLC is characterized by rapid growth and widespread metastases. Limited-stage SCLC indicates that only 1 side of the chest is affected and can likely be cured with chemotherapy and radiation therapy. SCLC is considered extensivestage when it has spread throughout the lung, to both sides of the chest, or to other parts of the body, and chemotherapy is used to control rather than cure the disease. First-line treatment for extensive-stage SCLC is chemotherapy (etoposide plus a platinum agent) with or without PD-L1 immunotherapy (atezolizumab [Tecentriq®] or durvalumab [Infinzi®]). While SCLC typically responds to initial therapy, long-term survival is rare. Moreover, the standard chemotherapy regimen is associated with myelosuppression, particularly in elderly patients. Neutropenia can be managed using colony stimulating growth factors (e.g., GM-CSF, G-CSF), but cumulative thrombocytopenia is a dose-limiting factor; in addition, routine use of growth factors is not recommended during initial therapy. If approved, trilaciclib will be the first CDK4/6 inhibitor to mitigate chemotherapy-induced myelosuppression. It has the potential to improve QOL and optimize the standard chemotherapy regimen as well as reduce the need for rescue interventions (growth factors, blood or platelet transfusions). While safety data for trilaciclib have not been announced, the CDK4/6 inhibitors as a class are generally well tolerated. Other agents in this class include abemaciclib (Verzenio®), palbociclib (Ibrance®), and ribociclib (Kisqali®). None of the 3 agents are approved for myelopreservation; rather all are indicated for the treatment of select patients with advanced/metastatic breast cancer. Trilaciclib is in phase 2 trials for the treatment of metastatic triple-negative breast cancer; final overall survival data are expected in Q4 2020.

FDA APPROVAL TIMELINE February 15, 2021

 Breakthrough Therapy

 Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$0

$16

$62

$146

$312

The forecast is a projection of total US sales per year.

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CARDIOLOGY

vericiguat oral Merck/Bayer PROPOSED INDICATION Chronic heart failure (HF)

CLINICAL OVERVIEW

Vericiguat is a soluble guanylate cyclase (sGC) stimulator that increases the level of cyclic guanosine monophosphate (cGMP), an important messenger in several signaling pathways in the CV system. The randomized, double-blind, placebo-controlled, phase 3 VICTORIA trial evaluated the efficacy of vericiguat administered in combination with available HF therapies in 5,050 patients with worsening chronic HF (New York Heart Association [NYHA] II-IV) with reduced left ventricular ejection fraction (LVEF < 45%) and elevated natriuretic peptide levels (within the previous 30 days). Patients were at high risk of hospitalization and CV death following a recent HF decompensation. During a median of 10.8 months, vericiguat reduced the risk for HF hospitalization or CV death (composite primary endpoint) compared to placebo (35.5% versus 38.5%, respectively; HR, 0.9; 95% CI, 0.82 to 0.98; p=0.019). This difference was driven by a decrease in hospitalization for HF (HR, 0.9; 95% CI, 0.81 to 1; p=0.048); there was no significant difference in CV death between the groups (HR, 0.93; 95% CI, 0.81 to 1.06; p=0.269). Overall, the safety profile for vericiguat was similar to placebo. Vericiguat was studied at a dosage of up to 10 mg orally once daily.

PLACE IN THERAPY

An estimated 6.2 million adults in the US have HF. It is a chronic, progressive condition in which the heart does not pump enough blood to meet the body's demand and leads to a high degree of morbidity and mortality. Approximately 66% of HF cases have reduced LVEF (HFrEF). Risk factors for HF include coronary artery disease, diabetes, hypertension, obesity, valvular heart disease, smoking, and sedentary lifestyle. While there is no cure for HF, treatment options include lifestyle changes and medications, as well as implantable devices and surgery in select cases. Improved cardiac function and life expectancy has been demonstrated with the following pharmacotherapies: ACEIs, ARBs, ARNIs (sacubitril/valsartan [Entresto®]), beta blockers, hydralazine/isosorbide dinitrate, the SGLT2 inhibitor dapagliflozin (Farxiga®), and mineralocorticoid receptor agonists. Ivabradine (Corlanor®), a hyperpolarization-activated cyclic nucleotide-gated channel blocker, is also indicated in select patients with HFrEF. If approved, vericiguat will provide another therapeutic option for patients with HFrEF. Vericiguat may compete with oral sacubitril/valsartan (Entresto) and the oral antidiabetic agent dapagliflozin (Farxiga) in the HFrEF space. Both agents have demonstrated significant reductions in both CV death and HF hospitalization and at higher rates than reported for vericiguat (vericiguat, HR=0.9; Entresto, HR=0.8; Farxiga, HR=0.74). Notably, in the clinical trial, prespecified events with vericiguat were accumulated earlier than anticipated (10.8 months, versus 24 and 27 months in Farxiga and Entresto trials, respectively); this reduced exposure time may have contributed to its lower response rate compared to Farxiga and Entresto. All 3 agents are administered orally; vericiguat and Farxiga are taken once daily, while Entresto is dosed twice a day. The SLGT2 inhibitors canagliflozin (Invokana®) and empagliflozin (Jardiance®) are also in phase 3 trials for HF. Other drugs in phase 3 trials for HF target p38 mitogen-activated protein kinase (ARRY-797), aldose reductase (AT-001), mineralocorticoid receptor (finerenone), and myosin (mavacamten, omecamtive mercarbil).

FDA APPROVAL TIMELINE January 20, 2021  Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$0

$29

$67

$121

$169

The forecast is a projection of total US sales per year.

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IMMUNOLOGY

voclosporin oral Aurinia PROPOSED INDICATION Lupus nephritis (LN)

CLINICAL OVERVIEW

Voclosporin is a next-generation calcineurin inhibitor (CNI) that blocks interleukin-2 (IL-2) expression and T-cell mediated immune responses and stabilizes podocytes in the kidneys. Safety and efficacy of voclosporin as add-on to background therapy of mycophenolate mofetil (MMF) plus taper of low-dose corticosteroids were demonstrated in the double-blind, phase 3 AURORA and phase 2b AURA-LV studies. In AURORA, 357 adults with LN were randomized to voclosporin or placebo. The primary endpoint was renal response, defined as urine protein/creatinine ratio (UPCR) ≤ 0.5 mg/mg, eGFR ≥ 60 mL/min or no confirmed decrease from baseline in eGFR of > 20%, sustained low-dose steroid use, and no use of rescue medication. The renal response rate at week 52 with voclosporin was superior compared to placebo (40.8% versus 22.5%, respectively; p<0.001); statistically significant differences between the groups were seen as early as 24 weeks. Approximately 10% of patients in each group achieved a decrease in eGFR by > 30%. Response with voclosporin was demonstrated across racial/ethnic groups (e.g., Black, Hispanic/Latino, Asian). In the AURA-LV trial, a higher rate of complete renal remission was seen with voclosporin compared to placebo at week 24 (32.6% versus 19.3%, respectively; p=0.046) and week 48 (49.4% versus 23.9%, respectively; p<0.001). Voclosporin was well tolerated. In both trials, infection was the most common adverse event reported. In AURORA, 1 death occurred in the voclosporin group and 5 in the placebo group. However, in the earlier AURA-LV study, the number of deaths was higher with voclosporin compared to placebo (10 [11.2%] versus 1 [1.1%], respectively); notably, the majority of deaths were reported at 2 study sites outside the US with compromised access to care and were not attributed to the study drug. For both clinical trials above, the data reflect oral doses of voclosporin of 23.7 mg twice daily. AURA-LV also included a dose of 39.5 mg twice daily; superiority to placebo was seen with both doses.

PLACE IN THERAPY

An estimated 1.5 million Americans have some form of lupus, with 90% being women. Moreover, it is 2 to 3 times more likely to affect women of color (e.g., Black, Hispanic/Latino, Asian, Native American) compared to Caucasians. Systemic lupus erythematosus (SLE) accounts for approximately 70% of all cases. Lupus nephritis occurs in up to 60% of patients with SLE, typically evident within 5 years of SLE diagnosis. Notably, LN progresses to ESRD in 5% to 20% of patients. Initial treatment of LN includes corticosteroids plus MMF or cyclophosphamide; corticosteroids plus azathioprine appears to be less beneficial. Complete renal response rates are 10% to 40% at 12 months with SOC therapy. Limited data suggest improved response when tacrolimus, a CNI, is added to an MMF plus glucocorticosteroid regimen. Once complete or partial response is achieved, MMF and azathioprine are the most common agents used for maintenance treatment. Voclosporin, a next-generation CNI, is an analog of cyclosporine. It may have a more predictable pharmacokinetic profile compared to legacy CNIs and may not need therapeutic monitoring. Significant improvement in renal response was observed when voclosporin was added to the SOC (MMF), regardless of race or ethnicity. If approved, voclosporin will be the first medication FDA-approved specifically to treat LN.

FDA APPROVAL TIMELINE January 22, 2021  Fast Track

 Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$0

$90

$273

$429

$654

The forecast is a projection of total US sales per year.

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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. Many controversies surround 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 FDA is still considering how to implement the nomenclature for previously approved biosimilar products. 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 will focus on providing flexibility for 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. Several states had already enacted biosimilar substitution legislation. Biosimilars are expected to receive full extrapolation for the eligible indications of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will likely need to be considered individually. Insulins were historically regulated by the FDA 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 biologics 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) improving the biosimilar development and approval process; (2) maximizing scientific and regulatory clarity for sponsors; (3) providing effective communications for patients, clinicians, and payers; and (4) reducing 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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To date, a total of 28 biosimilars have received FDA approval. Of these, only 18 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zarxio® (filgrastim-sndz)

Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer/Celltrion

April 2016

Erelzi™ (etanercept-szzs)

Sandoz

August 2016

Amjevita™ (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Samsung Bioepis/ Merck

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

December 2017

Retacrit® (epoetin alfa-epbx)

Pfizer/Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Mylan

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz™ (adalimumab-adaz)

Sandoz

October 2018

Udenyca® (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Celltrion/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Celltrion/Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Samsung Bioepis/ Merck

January 2019

Trazimera™ (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Samsung Bioepis/ Merck

April 2019

Kanjinti™ (trastuzumab-anns)

Amgen

June 2019

Zirabev™ (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™ (adalimumab-bwwd)

Samsung Bioepis/ Merck

July 2019

Ruxience™ (rituximab-pvvr)

Pfizer

July 2019

Abrilada™ (adalimumab-afzb)

Pfizer

November 2019

Ziextenzo® (pegfilgrastim-bmez)

Novartis/Sandoz

November 2019

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Commercially Available

  -

 -

 -

    -

     -

  -

 -

Originator Product (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) Humira (Abbvie) Neulasta (Amgen)


APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Avsola™ (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer/Hospira

June 2020

Hulio® (adalimumab-fkjp)

Mylan

July 2020

Commercially Available

 -

Originator Product (Manufacturer) Remicade (Janssen) Neulasta (Amgen) Neulasta (Amgen)

* 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 biosimilar agent to Sanofi’s Lantus®, and Sanofi’s Admelog® insulin lispro, approved as a biosimilar product to Eli Lilly’s Humalog®. In June 2020, the FDA approved insulin glargine (Semglee™) by Mylan/Biocon under an abbreviated 505(b)(2) New Drug Application (NDA) pathway; the reference product was Lantus. Semglee is considered a biologic under section 351(a) rather than a biosimilar. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play a role in market adoption of biosimilars. While < 2% of Americans use biologics, they account for almost 40% of all prescription drug spending. Moreover, they comprised 70% of growth in drug spending from 2010 to 2015. Not surprisingly, there is a growing body of evidence on predicted biologic spend and potential biosimilar savings. A 2020 report by IQVIA forecasts that biosimilars are on track to reduce overall US drug spend by $100 billion over the next 5 years. In fact, the next 5 years are expected to have an estimated 5-fold increase in savings relative to the past 5 years as more biosimilars launch and existing biosimilars see more utilization and reductions in price. Three recent biosimilar launches in 2019 saw substantial uptake within the first year of commercialization, these are: bevacizumab (42%), trastuzumab (38%), and rituximab (20%). In the US, it is estimated that biosimilars will cost 15% to 35% less than the originator product, although price dynamics vary. The potential cost savings, however, can vary based on the market segment where brand contracts can play a role. A 2017 report by the RAND Corporation estimates a $54 billion cost savings from biosimilars between 2017 and 2026. In July 2018, an FDA analysis reported that if Americans had access to FDA-approved biosimilars in 2017, it would have resulted in a $4.5 billion savings. A 2017 analysis by the Moran Company projects biosimilars could save the government an estimated $11.4 billion by 2027, but it would require the Centers for Medicare and Medicaid Services (CMS) to revise its reimbursement policy for biosimilars. Subsequently, in November 2017, the CMS revised its reimbursement policy. The CMS now issues a unique Healthcare Common Procedure Coding System (HCPCS) code to each individual biosimilar. Under this rule, Medicare Part B separately codes and pays for biosimilars and no longer groups them into a common payment code with originator agents. A June 2018 infliximab case study by the Pacific Research Institute forecasts annual savings of up to $465 million from increased use of biosimilars to replace a single biologic for commercial payers and Medicare. Biosimilars are paving the way for increased access to important biologic therapies that may increase survival and/ or QOL for many patients with difficult-to-treat diseases while also reducing costs.

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BIOSIMILAR OVERVIEW continued

ONCOLOGY

bevacizumab IV Aybintio and Bmab-100 are investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth (VEGF)specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, non-squamous non-small cell lung cancer (nsNSCLC), glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA APPROVAL TIMELINE

Merck/Samsung Bioepis (Aybintio) Pending Mylan/Biocon (Bmab-100) December 25, 2020

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$2,076

$1,534

$1,278

$1,098

$901

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

BLOOD MODIFIER

filgrastim IV, SC Apotex and Kashiv are seeking 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 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 who are acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).

FDA APPROVAL TIMELINE Apotex (Grastofil) Pending Kashiv Pending

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$127

$104

$89

$80

$73

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

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BIOSIMILAR OVERVIEW continued

ENDOCRINE

insulin aspart SC Mylan/Biocon Mylan/Biocon is seeking biosimilar approval to Novo Nordisk’s Novolog®, a rapid-acting insulin to improve glycemic control in patients with T1DM or T2DM.

FDA APPROVAL TIMELINE April to June 2021

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$948

$740

$643

$578

$533

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

BLOOD MODIFIER

pegfilgrastim SC Lapelga and MSB-11455 are 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).

FDA APPROVAL TIMELINE Apotex (Lapelga) Pending

Merck/Fresenius (MSB-11455) January to March 2021

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$1,980

$1,576

$1,295

$1,093

$948

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

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BIOSIMILAR OVERVIEW continued

ONCOLOGY

rituximab (ABP-798) IV Amgen/Allergan ABP-798 is an investigational biosimilar to Genentech’s Rituxan, a CD20-directed cytolytic antibody indicated for the treatment of non-Hodgkin’s lymphoma (NHL), chronic lymphocytic leukemia (CLL), rheumatoid arthritis (RA), and antineutrophil cytoplasmic antibody-associated vasculitis.

FDA APPROVAL TIMELINE December 18, 2020

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$3,514

$2,480

$1,819

$1,444

$1,127

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

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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 2024, are displayed. The financials are projected total annual US sales, reported in millions. tirzepatide

bardoxolone methyl

$652

$507

Diabetes

Renal

NVX-CoV2373 vaccine

belumosudil

$1,615

$610

Immunology

COVID-19

betibeglogene autotemcel (Zynteglo)

mRNA-1273 vaccine COVID-19

Hematology/Gene therapy

$2,349

$366

mirikizumab

bimekizumab

$552

$465

Immunology

Immunology

mavacamten

deucravacitinib

$886

$835

Cardiovascular

Immunology

lonapegsomatropin

efgartigimod

$623

$754

Endocrine

Immunology

lenadogene nolparvovec (GS010) Ophthalmology/ Gene therapy

$41

elivaldogene tavalentivec (Lenti-D) ipatasertib Oncology

Neurology/Gene therapy

$49

$530 pecialty drug names appear in ď‚Ť S magenta throughout the publication.

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PIPELINE DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2021. 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) and those that have been withdrawn from development are also noted. APPLICATION APPLICATION SUBMITTED SUBMITTED TO THE FDA

IN PHASE PHASE 33 TRIALS TRIALS

59% 41% 34% 26% 25% 7%

Specialty

65% 35% 32% 13% 8%

Traditional

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Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar


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

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

Submitted (New Drugs) loteprednol etabonate 0.25%

Kala

Dry eye disease

Ophthalmic

Submitted – 505(b)(2) NDA

10/30/2020

mannitol (dry powder)

Cheisi

CF (adults)

Inhaled

Submitted – NDA; Fast Track; Orphan Drug

10/30/2020

viloxazine

Supernus

ADHD

Oral

Submitted – NDA

11/06/2020

amphetamine sulfate IR (tamper-resistant)

Arbor

ADHD (adults, pediatrics ages ≥ 3 years)

Oral

Submitted – NDA

11/13/2020

samidorphan/olanzapine

Alkermes

Bipolar disorder; Schizophrenia

Oral

Submitted – NDA

11/13/2020

sutimlimab

Sanofi

Cold agglutnin disease

IV

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

11/13/2020

naloxone single-dose prefilled syringe

US Worldmeds

Opioid overdose

IM

Submitted – 505(b)(2) NDA

11/15/2020

lisocabtagene maraleucel

Bristol-Myers Squibb

DLBCL (relapsed/ refractory)

IV

Submitted – BLA; 11/16/2020 Breakthrough Therapy; Orphan Drug; Priority Review; RMAT

lonafarnib

Eiger

Hutchinson–Gilford Progeria syndrome

Oral

Submitted – NDA; 11/20/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

treprostinil dry powder

Liquidia

PAH

Inhaled

Submitted – 505(b)(2) NDA

setmelanotide

Rhythm

Obesity (pro-opiomelanocortin and leptin receptor deficiency)

SC

Submitted – NDA; 11/27/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

naxitamab

Y-mAbs Therapeutics

Neuroblastoma (relapsed/ IV refractory, high-risk)

Submitted – BLA; 11/30/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

inclisiran

Novartis/The Medicines Company

Dyslipidemia (for secondary prevention patients with ASCVD and familial hypercholesterolemia)

SC

Submitted – NDA; Orphan Drug

tanezumab

Pfizer

Osteoarthritis pain

IV

Submitted – BLA; Fast December Track 2020

berotralstat

Biocryst

Hereditary angioedema (attack prevention)

Oral

Submitted – NDA; Fast Track; Orphan Drug

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11/24/2020

December 2020

12/03/2020


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

lumasiran

Alnylam

Hyperoxaluria

SC

Submitted – NDA; 12/07/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

margetuximab

Macrogenics

Breast cancer (HER2+, in combination with chemotherapy)

IV

Submitted – BLA; Fast 12/18/2020 Track

rituximab (biosimilar to Genentech’s Rituxan)

Amgen/Allergan

RA; CLL/SLL; NHL (indolent); ANCAassociated vasculitis

IV

Submitted – BLA

12/18/2020

relugolix

Myovant

Prostate cancer (advanced)

Oral

Submitted – NDA; Priority Review

12/20/2020

roxadustat

AstraZeneca

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

Oral

Submitted – NDA

12/20/2020

ansofaxine

Luye

MDD

Oral

Submitted – NDA

12/25/2020

bevacizumab (biosimilar to Genentech’s Avastin)

Mylan/Biocon

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

IV

Submitted – BLA

12/25/2020

vibegron

Urovant

Overactive bladder

Oral

Submitted – NDA

12/25/2020

arbaclofen ER

Osmotica

MS-associated spasticity

Oral

Submitted – 505(b)(2) NDA

12/29/2020

furosemide wearable pump Scpharmaceuticals

Congestive heart failure

SC

Submitted – 505(b)(2) NDA

12/30/2020

tirbanibulin

Almirall

Actinic keratoses

Topical

Submitted – NDA

12/30/2020

belumosudil

Kadmon

Chronic GVHD

Oral

Submitted – NDA; Breakthrough Therapy; Orphan Drug; RTOR

Jan–Mar 2021

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Merck/Fresenius

Neutropenia/leukopenia

SC

Submitted – BLA

Jan–Mar 2021

vericiguat

Merck/Bayer

Chronic heart failure

Oral

Submitted – NDA; Priority Review

01/20/2021

voclosporin

Aurinia

Lupus nephritis

Oral

Submitted – NDA; Fast Track; Priority Review

01/22/2021

pegunigalsidase alfa

Chiesi

Fabry’s disease

IV

Submitted – BLA; seeking Accelerated Approval; Fast Track; Priority Review

01/27/2021

tepotinib

Merck

NSCLC (mesenchymalepithelial transition exon 14 [METex14] skipping)

Oral

Submitted – NDA; Breakthrough Therapy; Priority Review; RTOR

February 2021

evinacumab

Regeneron

Homozygous familial hypercholesterolemia

IV

Submitted – BLA; Breakthrough Therapy; Priority Review

02/11/2021

24 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

umbralisib

TG

Marginal zone lymphoma (≥ 1 prior anti-CD20 based regimen)

Oral

Submitted – NDA; seeking Accelerated Approval; Breakthrough Therapy; Orphan Drug; Priority Review

02/15/2021

trilaciclib

G1 Therapeutics/ Boehringer Ingelheim

SCLC

IV

Submitted – NDA; Breakthrough Therapy; Priority Review

02/15/2021

casimersen

Sarepta

DMD (amenable to exon 45 skipping)

IV

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

02/25/2021

melflufen

Oncopeptides

Multiple myeloma (triple refractory)

IV

Submitted – NDA; Orphan Drug; Priority Review

02/26/2021

paclitaxel/encequidar

Athenex

Breast cancer

Oral

Submitted – NDA; Priority Review

02/28/2021

taurolidine/heparin/citrate

Cormedix

Prevention of catheterrelated sepsis (hemodialysis patients)

IV

Submitted – NDA; Fast Track; Priority Review; QIDP

02/28/2021

ropeginterferon alfa-2b

Pharmaessentia

Polycythemia vera (in absence of symptomatic splenomegaly)

SC

Submitted – BLA; Orphan Drug

Mar–Apr 2021

serdexmethylphenidate

Kempharm

ADHD

Oral

Submitted – 505(b)(2) NDA

03/02/2021

plasminogen (human)

Liminal

Congenital plasminogen deficiency

IV

Submitted – BLA; Fast Track; Orphan Drug; Rare Pediatric Disease

03/05/2021

aducanumab

Biogen/Eisai

Alzheimer’s disease

IV

Submitted – BLA; Fast 03/07/2021 Track; Priority Review

arimoclomol

Orphazyme

Niemann-Pick disease

Oral

Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review; Rare Pediatric Disease

03/17/2021

ponesimod

Janssen

MS (relapsing)

Oral

Submitted – NDA

03/18/2021

dasiglucagon

Zealand

Hypoglycemia (diabetesrelated)

SC

Submitted – NDA; Orphan Drug

03/27/2021

idecabtagene vicleucel

Bristol-Myers Squibb/ Bluebird Bio

Multiple myeloma (≥ 3 prior therapies)

IV

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

03/27/2021

tivozanib

AVEO

RCC (relapsed/refractory)

Oral

Submitted – NDA

03/31/2021

inolimomab

Elsalys

GVHD (acute, steroidrefractory)

IM

Submitted – BLA; Orphan Drug; RTOR

April 2021

eflornithine/sulindac

Mallinckrodt

Familial adenomatous polyposis

Oral

Submitted – 505(b)(2) NDA; seeking Accelerated Approval; Fast Track; Orphan Drug

Apr–Jun 2021

25 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Mylan/Biocon

T1DM; T2DM

SC

Submitted – BLA

Apr–Jun 2021

tralokinumab

AstraZeneca

Atopic dermatitis

SC

Submitted – BLA

Apr–Jun 2021

fosdenopterin

Bridgebio

Molybdenum cofactor deficiency (MoCD)

IV

Submitted – NDA; 04/09/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

estetrol/drospirenone

Mayne

Contraception

Oral

Submitted – NDA

04/16/2021

treprostinil dry powder

United Therapeutics

PAH

Inhaled

Submitted – 505(b)(2) NDA

04/17/2021

benzoyl peroxide

Sol-gel

Rosacea

Transdermal

Submitted – 505(b)(2) NDA

04/26/2021

abrocitinib

Pfizer

Atopic dermatitis

Oral

Submitted – NDA; Breakthrough Therapy

04/30/2021

dehydrated alcohol

Eton

Methanol poisoning

SC

Submitted – NDA; Orphan Drug

05/27/2021

leuprolide mesylate readyto-use, 6-month depot

Foresee

Prostate cancer

SC

Submitted – 505(b)(2) NDA

05/27/2021

zonisamide oral suspension

Eton

Partial seizures

Oral

Submitted – 505(b)(2) NDA

05/28/2021

udenafil

Allergan

Single ventricle heart disease (ages ≥ 12 years)

Oral

Submitted – NDA; Orphan Drug

June 2020

relugolix/estradiol/ norethindrone

Myovant

Uterine fibroid-related Oral heavy menstrual bleeding

Submitted – NDA

06/01/2021

umbralisib

TG

Follicular lymphoma (≥ 2 prior systemic therapies)

Oral

Submitted – NDA; seeking Accelerated Approval; Orphan Drug

06/15/2021

lonapegsomatropin

Ascendis

Growth hormone deficiency (pediatrics)

SC

Submitted – BLA; Orphan Drug

06/25/2021

proteolytic enzymes

Mediwound

Burn injury debridement

Topical

Submitted – BLA; Orphan Drug

06/29/2021

bimekizumab

UCB

PSO

SC

Submitted – BLA

July 2021

avacopan

Chemocentryx

ANCA-associated vasculitis

Oral

Submitted – NDA; Orphan Drug

07/07/2021

apomorphine infusion pump

Supernus

Parkinson’s disease (offepisodes)

SC

Submitted – NDA

07/14/2021

topiramate oral solution

Eton

Partial seizures

Oral

Submitted – 505(b)(2) NDA

08/06/2021

vosoritide

Biomarin

Achondroplasia

SC

Submitted – NDA; Orphan Drug

08/20/2021

pegcetacoplan

Apellis

Paroxysmal nocturnal hemoglobinuria

SC

Submitted – NDA; Fast Track; Orphan Drug

09/15/2021

loncastuximab tesirine

ADC

DLBCL (relapsed/ refractory)

IV

Submitted – BLA; Orphan Drug

09/21/2021

pneumococcal multivalent vaccine

Pfizer

Invasive pneumococcal disease prevention

IM

Submitted – BLA; Breakthrough Therapy; Fast Track

October 2021

26 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

bevacizumab (biosimilar to Genentech’s Avastin)

Merck/Samsung Bioepis

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

IV

Submitted – BLA

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Kashiv

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

SC

Submitted – BLA

Pending

linaclotide acetate (Linzess®)

Ironwood

IBS (treatment of abdominal symptoms)

Oral

Submitted – sNDA

October 2020

ticagrelor (Brilinta®)

AstraZeneca

Ischemic stroke

Oral

Submitted – sNDA; Priority Review

Oct–Nov 2020

durvalumab (Imfinzi®) 4-week fixed dose regimen

AstraZeneca

Bladder cancer; NSCLC

IV

Submitted – sBLA

Oct–Dec 2020

alectinib (Alecensa®)

Genentech

NSCLC (1st-line, ALK+)

Oral

Submitted – sNDA

November 2020

estradiol/progesterone (Bijuva®) 0.5 mg/100 mg

TherapeuticsMD

Menopause (including hormone replacement therapy [HRT])

Oral

Submitted – sNDA

11/16/2020

baloxavir marboxil (Xofluza®) oral granules, tablets

Genentech

Influenza (treatment, ages 1-12 years; postexposure prophylaxis, ages 1 year to adults)

Oral

Submitted – sNDA

11/23/2020

pembrolizumab (Keytruda®)

Merck

Breast cancer (locally recurrent unresectable or metastatic, TNBC, PD-L1positive; in combination with chemotherapy)

IV

Submitted – sBLA; seeking Accelerated Approval; Breakthrough Therapy; Priority Review

11/27/2020

tasimelteon (Hetlioz®) capsule and oral liquid

Vanda

Smith-Magenis syndrome (adults and children)

Oral

Submitted – sNDA; Orphan Drug; Priority Review

12/01/2020

ocrelizumab (Ocrevus®) 2-hour twice-yearly infusion

Genentech

MS (relapsing and primary IV progressive)

Submitted – sBLA

12/14/2020

elexacaftor/tezacaftor/ ivacaftor and ivacaftor (Trikafta®)

Vertex

CF (with additional rare CFTR mutations)

Oral

Submitted – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug

12/30/2020

ivacaftor (Kalydeco®)

Vertex

CF (with additional rare CFTR mutations)

Oral

Submitted – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug

12/30/2020

tezacaftor/ivacaftor and ivacaftor (Symdeko®)

Vertex

CF (with additional rare CFTR mutations)

Oral

Submitted – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug

12/30/2020

crizotinib (Xalkori®)

Pfizer

Anaplastic large cell lymphoma (relapsed/ refractory, pediatric)

Oral

Submitted – sNDA; Breakthrough Therapy

January 2021

Submitted (Supplementals)

27 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

lacosamide (Vimpat®)

UCB

Primary generalized tonic-clonic seizures (adjunctive treatment, ages ≥ 4 years)

IV, Oral

Submitted – sNDA

January 2021

omalizumab (Xolair®) self-administered

Genentech

Asthma; Urticaria

SC

Submitted – sBLA

Jan–Mar 2021

peginterferon beta-1a (Plegridy®)

Biogen

MS (relasping/remitting, active secondary progressive)

IM

Submitted – sBLA; Fast Track

February 2021

baricitinib (Olumiant®)

Eli Lilly

Atopic dermatitis

Oral

Submitted – sNDA

Feb–Apr 2021

cabozantinib (Cabometyx®)

Exelixis

RCC (advanced, 1st-line, in combination with nivoluab)

Oral

Submitted – sNDA; Priority Review

02/20/2021

nivolumab (Opdivo®)

Bristol-Myers Squibb

RCC (advanced, in combination with cabozantinib)

IV

Submitted – sBLA; Breakthrough Therapy; Fast Track; Priority Review

02/20/2021

sacubitril/valsartan (Entresto)

Novartis

Heart failure with preserved ejection fraction (HFpEF)

Oral

Submitted – sNDA

02/26/2021

pralsetinib (Gavreto™)

Blueprint Medicines

Thyroid cancer (RETfusion+)

Oral

Submitted – sNDA; Breakthrough Therapy; Priority Review; RTOR

02/28/2021

belimumab (Benlysta®)

GlaxoSmithKline

Lupus nephritis

IV

Submitted – sBLA; Breakthrough Therapy

Mar–May 2021

rimegepant (Nurtec™ ODT)

Biohaven

Migraine prevention

Oral

Submitted – sNDA

May–Jun 2021

selinexor (Xpovio )

Karyopharm

Multiple myeloma (after ≥ Oral 1 prior line of therapy)

Submitted – sNDA; Orphan Drug

03/19/2021

bupivacaine liposome suspension (Exparel®)

Pacira

Postsurgical pain (ages ≥ 6 years)

IM

Submitted – sNDA

03/22/2021

pembrolizumab (Keytruda)

Merck

Breast cancer (highrisk, early-stage, TNBC, neoadjuvant, in combination with chemotherapy); Breast cancer (high-risk, earlystage, TNBC, adjuvant, monotherapy)

IV

Submitted – sBLA; seeking Accelerated Approval; Breakthrough Therapy; Priority Review

03/29/2021

upadacitinib (Rinvoq™)

Abbvie

PsA

Oral

Submitted – sNDA

04/01/2021

pimavanserin (Nuplazid )

Acadia

Dementia-related hallucinations and delusions

Oral

Submitted – sNDA; Breakthrough Therapy

04/03/2021

alirocumab (Praluent®)

Regeneron

Homozygous familial hypercholesterolemia

SC

Submitted – sBLA; Orphan Drug

04/04/2021

treprostinil (Tyvaso®)

United Therapeutics

Interstitial lung diseaseassociated pulmonary hypertension

Inhaled

Submitted – sNDA; Priority Review

04/14/2021

ibrutinib (Imbruvica®)

Abbvie

Waldenstrom macroglobulinemia (in combination with rituximab)

Oral

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

04/23/2021

®

®

28 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

tenapanor (Ibsrela®)

Ardelyx

Hyperphosphatemia (in CKD dialysis-dependent patients)

Oral

Submitted – sNDA

04/29/2021

omadacycline (Nuzyra®)

Paratek

CABP (oral-only dosing)

IV, Oral

Submitted – sNDA; Fast Track; QIDP

05/31/2021

upadacitinib (Rinvoq)

Abbvie

Axial spondyloarthritis

Oral

Submitted – sNDA

06/25/2021

axicabtagene ciloleucel (Yescarta®)

Gilead

Follicular lymphoma, Marginal zone lymphoma (relapsed/refractory, after ≥ 2 lines of therapy for both)

IV

Submitted – sBLA; Breakthrough Therapy; Orphan Drug

July 2021

daratumumab/ hyaluronidase-fihj (Darzalex Faspro™)

Janssen

Amyloidosis (light-chain)

SC

Submitted – sBLA; RTOR

July 2021

levonorgestrel (Mirena®)

Bayer

Contraception (up to 7 years duration)

Intrauterine

Submitted – sNDA

July 2021

selexipag (Uptravi®)

Janssen

PAH (WHO FC II-III, temporarily unable to take oral formulation)

IV

Submitted – sNDA; Orphan Drug

07/30/2021

upadacitinib (Rinvoq)

Abbvie

Atopic dermatitis (adults, adolescents)

Oral

Submitted – sNDA; Breakthrough Therapy

08/21/2021

abaloparatide-TD

Radius Health

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

Ad26COVS1 vaccine

Janssen

COVID-19

IM

Phase 3 – BLA

TBD

Ad5-nCoV vaccine

Cansino/Beijing Institute of Biotechnology

COVID-19

IM

Phase 3 – BLA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Coherus

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

SC

Phase 3 – BLA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Fresenius

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

SC

Phase 3 – BLA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Momenta

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

SC

Phase 3 – BLA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Mylan/Biocon

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

SC

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea®)

Mylan/Biocon

Diabetic macular edema

Intraocular

Phase 3 – BLA

TBD

AG10

Eidos

Transthyretin amyloid cardiomyopathy (ATTRCM)

Oral

Phase 3 – NDA

TBD

alicaforsen

Atlantic Healthcare

Pouchitis

Rectal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

alpha 1 proteinase inhibitor

Kamada

Alpha-1 antitrypsin deficiency

Inhaled

Phase 3 – BLA; Orphan Drug

TBD

amivantamab

Janssen

NSCLC

IV

Phase 3 – BLA; Breakthrough Therapy

TBD

andolast

Mylan

Asthma

Inhaled

Phase 3 – NDA

TBD

anifrolumab

AstraZeneca

SLE

IV

Phase 3 – BLA; Fast Track

TBD

anthrax vaccine adsorbed

Emergent

Anthrax infection

IM

Phase 3 – BLA; Fast Track

TBD

Phase 3 (New Drugs)

29 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

antolimab

Allakos

Gastroenteritis (eosinophilic esophagitis)

IV

Phase 3 – BLA; Orphan Drug

TBD

ARO-AAT

Arrowhead

Liver disease (alpha-1 antitrypsin deficiency)

SC

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

asciminib

Novartis

Chronic myelogenous leukemia (CML)

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ataluren

PTC

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

AT-GAA

Amicus

Pompe disease

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

atogepant

Allergan

Migraine prevention

Oral

Phase 3 – NDA

TBD

autologous genetically modified human dermal fibroblasts

Castle Creek

Epidermolysis bullosa

Intradermal

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

TBD

avalglucosidase alfa

Sanofi

Pompe disease

IV

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

AZD-1222 vaccine (formerly ChAdOx1-S)

AstraZeneca/University of Oxford

COVID-19

IM

Phase 3 – BLA

TBD

baclofen/naltrexone/ sorbitol

Pharnext

Charcot-Marie-Tooth disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

balixafortide

Polyphor

Breast cancer

IV

Phase 3 – NDA; Fast Track

TBD

balstilimab

Agenus

Cervical cancer

IV

Phase 3 – BLA; Fast Track

TBD

bamlanivimab

Eli Lilly

COVID-19

IV

Phase 3 – BLA

TBD

bardoxolone methyl

Reata

Alport syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

beremagene geperpavec

Krystal

Epidermolysis bullosa

Topical

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

TBD

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

Thalassemia; Sickle cell disease

IV

Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug. RMAT

TBD

bevacizumab

Outlook

Wet AMD

Intraocular

Phase 3 – BLA

TBD

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera Solutions

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

IV

Phase 3 – BLA

TBD

bevacizumab (biosimilar to Genentech’s Avastin)

Boehringer Ingelheim

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

IV

Phase 3 – BLA

TBD

bevacizumab (biosimilar to Genentech’s Avastin)

Kyowa Kirin/AstraZeneca

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

IV

Phase 3 – BLA

TBD

bexagliflozin

Theracos

T2DM

Oral

Phase 3 – NDA

TBD

bimekizumab

UCB

Axial spondyloarthritis; PsA

SC

Phase 3 – BLA

TBD

bintrafusp alfa

Merck

Biliary tract cancer

IV

Phase 3 – BLA; Orphan Drug

TBD

30 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

BIVV 001

Sanofi

Hemophilia A

IV

Phase 3 – BLA; Orphan Drug

TBD

BNT162 vaccine (multiple variants)

Biontech/Pfizer

COVID-19

IM

Phase 3 – BLA; Fast Track

TBD

budenoside

Calliditas

Immunoglobulin A (IgA) nephropathy (Berger’s disease)

Oral

Phase 3 – 505(b)(2) NDA; Orphan Drug

TBD

cabotegravir long-acting

Viiv

HIV-1 infection preexposure prevention (PrEP)

IM

Phase 3 – NDA

TBD

calmangafodipir

Pledpharma

Chemotherapy-induced peripheral neuropathy

IV

Phase 3 – NDA

TBD

cannabidiol gel

Zynerba

Fragile X syndrome

Transdermal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

capivasertib

AstraZeneca

Breast cancer

Oral

Phase 3 – NDA

TBD

capsaicin

Centrexion

Osteoarthritis

Intraarticular

Phase 3 – NDA; Fast Track

TBD

carglumic acid

Recordati

Hyperammonaemia (autosomal disorderrelated)

Oral

Phase 3 – NDA; Orphan Drug

TBD

CD24Fc

OncoImmune

COVID-19

IV

Phase 3 – BLA

TBD

ceftobiprole medocaril

Basilea

ABSSSI

IV

Phase 3 – NDA; Fast Track; QIDP

TBD

cenicriviroc mesylate

Allergan

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

ciltacabtagene autoleucel

Janssen

Multiple myeloma

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

clindamycin phosphate gel

Daré

Bacterial vaginosis

Intravaginal

Phase 3 – NDA; QIDP

TBD

CM-AT (pancreatic enzyme)

Curemark

Autism spectrum disorders

Oral

Phase 3 – BLA; Fast Track

TBD

crisantaspase

Jazz

ALL

IM, IV

Phase 3 – BLA; Fast Track

TBD

CSL112

CSL

Atherosclerosis

IV

Phase 3 – BLA

TBD

dactolisib

Restorbio

COVID-19

Oral

Phase 3 – NDA

TBD

dalcetrapib

Dalcor

Dyslipidemia/ hypercholesterolemia

Oral

Phase 3 – NDA

TBD

daprodustat

GlaxoSmithKline

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

Oral

Phase 3 – NDA

TBD

daridorexant

Idorsia

Insomnia

Oral

Phase 3 – NDA

TBD

decuprate

Alexion

Wilson’s disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

dehydrated human Mimedx amnion-chorion membrane

Achilles tendonitis; Plantar fasciitis

IV

Phase 3 – BLA

TBD

denosumab (biosimilar to Amgen’s Prolia®)

Novartis

Osteoporosis/osteopenia

SC

Phase 3 – BLA

TBD

deramanido

Otsuka

Tuberculosis

Oral

Phase 3 – NDA

TBD

deucravacitinib

Bristol-Myers Squibb

PSO

Oral

Phase 3 – NDA

TBD

dexamethasone SR

Otonomy

Meniere’s disease

Intratympanic

Phase 3 – 505(b)(2) NDA; Fast Track

TBD

31 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

dexmedetomidine

Bioxcel

Schizophrenia

SL

Phase 3 – NDA; Fast Track

TBD

dextromethorphan/ bupropion

Axsome

MDD

Oral

Phase 3 – 505(b)(2) NDA; Breakthrough Therapy; Fast Track

TBD

difelikefalin

Enteris

Pruritus (hemodialysisrelated)

IV

Phase 3 – NDA; Breakthrough Therapy

TBD

dociparstat

Chimerix

COVID-19

IV

Phase 3 – NDA

TBD

docosahexaenoic acid

Micelle

Sickle cell disease

Oral

Phase 3 – NDA; Orphan Drug

TBD

dovitinib

Oncology Venture

RCC

Oral

Phase 3 – NDA

TBD

dusquetide

Soligenix

Mucositis

IV

Phase 3 – NDA; Fast Track

TBD

dust mite immunotherapy

Stallergenes

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

edasalonexent

Catabasis

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

efgartigimod

Argenx

Myasthenia gravis

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

elamipretide

Stealth Bio

Barth syndrome

SC

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

elivaldogene tavalentivec (Lenti-D)

Bluebird Bio

Adrenomyeloneuropathy (adrenoleukodystrophy)

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

IV

Phase 3 – NDA; Fast Track; QIDP

TBD

EP-2101 therapeutic vaccine

OSE Immunotherapeutics

NSCLC

SC

Phase 3 – NDA; Orphan Drug

TBD

eprenetapopt

Aprea

Myelodysplastic syndrome (tumor protein p53 mutation)

IV

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

TBD

erdosteine

Alitair

COPD

Oral

Phase 3 – NDA

TBD

estetrol

Mithra

Menopause vasomotor symptoms

Oral

Phase 3 – NDA

TBD

etanercept (biosimilar to Amgen’s Enbrel)

Coherus

RA; PSO

SC

Phase 3 – BLA

TBD

etranacogene dezaparvovec

Uniqure

Hemophilia B

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

etrasimod

Arena

UC

Oral

Phase 3 – NDA

TBD

etrolizumab

Genentech

CD

SC

Phase 3 – BLA

TBD

faricimab

Genentech

Diabetic macular edema; Wet AMD

Intraocular

Phase 3 – BLA

TBD

fasinumab

Regeneron

Osteoarthritis

SC

Phase 3 – BLA

TBD

32 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

fexapotide triflutate

Nymox

Benign prostatic hyperplasia

Intratumoral

Phase 3 – NDA

TBD

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Phase 3 – NDA

TBD

fidanacogene elaparvovec

Pfizer

Hemophilia B

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

PsA; UC

Oral

Phase 3 – NDA

TBD

filsuvez

Amryt

Epidermolysis bullosa

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

finerenone

Bayer

Diabetic nephropathy

Oral

Phase 3 – NDA

TBD

fitusiran

Sanofi

Hemophilia A and B (with and without inhibitors)

SC

Phase 3 – NDA; Orphan Drug

TBD

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

Allergan

Female reproductive disorder

SC

Phase 3 – BLA

TBD

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

Finox

Female reproductive disorder

SC

Phase 3 – BLA

TBD

follitropin delta

Ferring

Female reproductive disorder

IV

Phase 3 – BLA

TBD

ganaxolone

Marinus

Seizure disorders

IV, Oral

Phase 3 – NDA; Orphan Drug

TBD

gefapixant

Merck

Chronic cough

Oral

Phase 3 – NDA

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3 – NDA; QIDP

TBD

givinostat

Italfarmaco

DMD

Oral

Phase 3 – NDA

TBD

glatiramer acetate depot

Mylan

MS

IM

Phase 3 – 505(b)(2) NDA

TBD

glepaglutide

Zealand

Short bowel syndrome

SC

Phase 3 – NDA; Orphan Drug

TBD

glycopyrrolate MDI

AstraZeneca

COPD

Inhaled

Phase 3 – NDA

TBD

hydrocortisone granules

Diurnal

Congenital adrenal hyperplasia

Oral

Phase 3 – 505(b)(2) NDA; Orphan Drug

TBD

hypericin

Soligenix

Cutaneous T-cell lymphoma (CTCL)

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ibrexafungerp

Scynexis

Vulvovaginal candidiasis

Oral

Phase 3 – NDA; Fast Track; Orphan Drug; QIDP

TBD

idursulfase

Takeda

Mucopolysaccharidosis II Intrathecal (MPS II; Hunter syndrome)

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

infigratinib

Bridgebio

Biliary tract cancer

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; PsA; CD; UC

IV

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

iodine I-131 monoclonal antibody

Actinium

Myeloablation prior to allogeneic HSCT to treat AML

IV

Phase 3 – BLA; Orphan Drug

TBD

ipatasertib

Genentech

Prostate cancer

Oral

Phase 3 – NDA

TBD

ipatasertib

Genentech

Breast cancer

Oral

Phase 3 – NDA

TBD

33 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

JNJ-78436735

Janssen

COVID-19

N/A

Phase 3 – BLA

TBD

KSI-301

Kodiak

Diabetic macular edema; Macualar edema related to retinal vein occlusion

Intraocular

Phase 3 – BLA

TBD

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3 – BLA; Orphan Drug

TBD

L-citrulline

Asklepion

Acute lung injury

IV

Phase 3 – NDA; Orphan Drug

TBD

lebrikizumab

Dermira

Atopic dermatitis

SC

Phase 3 – BLA; Fast Track

TBD

lenacapavir

Gilead

HIV-1 infection (heavily treatment-experienced)

Oral (lead-in dose), SC

Phase 3 – NDA; Breakthrough Therapy

TBD

lenadogene nolparvovec (GS010)

Gensight

Leber’s hereditary optic neuropathy

Intraocular

Phase 3 – BLA; Orphan Drug

TBD

lenzilumab

Humanigen

COVID-19

IV

Phase 3 – BLA

TBD

leriglitazone

Minoryx

Adrenomyeloneuropathy (adrenoleukodystrophy)

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

leronlimab

Cytodyn

COVID-19

SC

Phase 3 – BLA

TBD

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

SC

Phase 3 – 505(b)(2) NDA

TBD

levoketoconazole

Strongbridge

Cushing’s syndrome

Oral

Phase 3 – 505(b)(2) NDA; Orphan Drug

TBD

ligelizumab

Novartis

Urticaria

SC

Phase 3 – BLA

TBD

linzagolix

ObsEva

Endometriosis; Uterine fibroids

Oral

Phase 3 – NDA

TBD

L-lactic acid/citric acid/ potassium bitartrate

Evofem

Urinary tract and reproductive tract infections (antibacterial)

Intravaginal

Phase 3 – NDA; Fast Track; QIDP

TBD

lonafarnib

Eiger

Hepatitis D infection

Oral

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

TBD

lorecivivint

Samumed

Osteoarthritis (knee)

Intraarticular

Phase 3 – NDA

TBD

lumateperone

Intra-Cellular Therapies

Bipolar disorder

Oral

Phase 3 – NDA

TBD

lutetium 177Lu-PSMA-617

Novartis

Prostate cancer

IV

Phase 3 – NDA

TBD

LYS-SAF302

Sarepta

Mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo A syndrome)

Intracerebral

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

maribavir

Takeda

Cytomegalovirus infection treatment

Oral

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

TBD

masitinib

AB Science

Asthma

Oral

Phase 3 – NDA

TBD

mavacamten

Myokardia

Obstructive hypertrophic cardiomyopathy

Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

MBG453

Novartis

Myelodysplastic syndrome

IV

Phase 3 – NDA

TBD

34 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

IV

Phase 3 – BLA; Orphan Drug

TBD

microbiota suspension

Ferring

Clostridium difficile infection (recurrent)

Rectal

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

TBD

micropine

Eyenovia

Pharmacologic mydriasis

Topical

Phase 3 – NDA

TBD

minocycline

Citius

Catheter-related bacteremia

IV

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

TBD

mirikizumab

Eli Lilly

PSO; UC

IV, SC

Phase 3 – BLA

TBD

mirvetuximab soravtansine

Immunogen

Ovarian cancer

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

mitapivat

Agios

Pyruvate kinase deficiency

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

mobocertinib

Takeda

NSCLC

Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

mRNA-1273 vaccine

Moderna/NIAID

COVID-19

IM

Phase 3 – BLA; Fast Track

TBD

MT-7117

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

nabiximols

GW

MS-related spasticity

Oral transmucosal

Phase 3 – NDA

TBD

nadofaragene firadenovec

Trizell

Mesothelioma

Percutaneous injection

Phase 3 – BLA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 – NDA

TBD

napabucasin

Sumitomo Dainippon

CRC

Oral

Phase 3 – NDA

TBD

narsoplimab

Omeros

HSCT-Associated thrombotic microangiopathy

IV, SC

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

natalizumab (biosimilar to Biogen’s Tysabri®)

Novartis

MS

IV

Phase 3 – BLA

TBD

NBI-74788

Neurocrine Biosciences

Congenital adrenal hyperplasia

Oral

Phase 3 – NDA

TBD

nedosiran

Dicerna

Hyperoxaluria

SC

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

nemolizumab

Galderma

Atopic dermatitis

SC

Phase 3 – BLA

TBD

nemorexant

Idorsia

Insomnia

Oral

Phase 3 – NDA

TBD

nirsevimab

AstraZeneca

RSV infection prevention

IM

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

NVX-CoV2373 vaccine

Novavax

COVID-19

IM

Phase 3 – BLA

TBD

odevixibat

Albireo

Progressive familial intrahepatic cholestasis

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

35 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

olipudase alfa

Sanofi

Niemann-Pick disease

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

omidubicel

Gamida Cell

Bone marrow transplant and stem cell transplant

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

ondansetron ER once-daily

Redhill

Gastroenteritis

Oral

Phase 3 – 505(b)(2) NDA

TBD

oportuzumab monatox

Sesen Bio

Bladder cancer (BCGunresponsive, nonmuscle invasive)

Intravesical

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

TBD

pacritinib

CTI Bio

Myelofibrosis

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

paliperidone palmitate 6-month injectable

Janssen

Schizophrenia

IM

Phase 3 – NDA

TBD

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

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

TBD

pamrevlumab

Fibrogen

Idiopathic pulmonary fibrosis

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

pegzilarginase

Aeglea

Arginase 1 deficiency

IV

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

TBD

pevonedistat

Takeda

Myelodysplastic syndrome

IV

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

plinabulin

Beyondspring

NSCLC

IV

Phase 3 – NDA

TBD

plinabulin

Beyondspring

Neutropenia/leukopenia

IV

Phase 3 – NDA; Breakthrough Therapy

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

SC

Phase 3 – BLA

TBD

PTI-NC-733

Proteostasis Therapeutics CF

Oral

Phase 3 – NDA; Fast Track

TBD

ranibizumab (biosimilar to Genentech’s Lucentis®)

Biogen/Samsung Bioepis

Wet AMD

Intraocular

Phase 3 – BLA

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Coherus/Santo

Wet AMD

Intraocular

Phase 3 – BLA

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

STADA Arzneimittel

Wet AMD

Intraocular

Phase 3 – BLA

TBD

REGN-COV2

Regeneron

COVID-19

SC

Phase 3 – BLA

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

reltecimod

Atox

Necrotizing soft tissue infection

IV

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

36 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

relugolix/estradiol/ norethindrone

Myovant

Endometriosis

Oral

Phase 3 – NDA

TBD

reproxalap

Aldeyra

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

resmetirom

Madrigal

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

rilzabrutinib

Principia Bio

Pemphigus vulgaris

Oral

Phase 3 – NDA; Orphan Drug

TBD

risperidone long-acting in situ microparticle

Laboratorios Farmacéuticos

Schizophrenia

IM

Phase 3 – 505(b)(2) NDA

TBD

ritlecitinib

Pfizer

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

rituximab (biosimilar to Genentech’s Rituxan)

Archigen

RA; CLL/SLL; NHL (indolent); ANCAassociated vasculitis

IV

Phase 3 – BLA

TBD

rivoceranib

LSK Biopartners

Gastric cancer

Oral

Phase 3 – NDA; Orphan Drug

TBD

roflumilast cream

Arcutis

PSO

Topical

Phase 3 – NDA

TBD

RSV nanoparticle vaccine

Novavax

RSV prevention

IM

Phase 3 – BLA; Fast Track

TBD

SAR408701

Sanofi

NSCLC

IV

Phase 3 – BLA

TBD

SARS-CoV-2 inactivated vaccine

Sinovac

COVID-19

IM

Phase 3 – BLA

TBD

SARS-CoV-2 inactivated vaccine (Vero cell)

Sinopharm/Beijing

COVID-19

IM

Phase 3 – BLA

TBD

SARS-CoV-2 inactivated vaccine (Vero cell)

Sinopharm/Wuhan

COVID-19

IM

Phase 3 – BLA

TBD

SARS-CoV-2 vaccine (adeno-based)

Gamaleya Research Institute

COVID-19

IM

Phase 3 – BLA

TBD

scCeftriaxone

scPharmaceuticals

Antibacterial (general)

SC

Phase 3 – NDA

TBD

seladelpar

Cymabay

Primary biliary cholangitis/hepatic fibrosis

Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

sepofarsen

Proqr

Leber’s congenital amaurosis

Intraocular

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

SER-109

Seres

C. difficile infection (recurrent)

Oral

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

sodium hyaluronate/ triamcinolone hexacetonide

Anika

Osteoarthritis (knee)

Intraarticular

Phase 3 – NDA

TBD

sodium oxybate oncenightly dosing

Avadel

Narcolepsy

Oral

Phase 3 – 505(b)(2) NDA; Orphan Drug

TBD

sofpironium

Brickell

Axillary hyperhidrosis

Topical

Phase 3 – NDA

TBD

somatrogon

Opko

Growth hormone deficiency (pediatric)

SC

Phase 3 – BLA; Orphan Drug

TBD

sparsentan

Retrophin

Focal segmental glomerulosclerosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

37 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

spartalizumab

Novartis

Melanoma

IV

Phase 3 – BLA

TBD

sulopenem etzadroxil

Iterum

UTI (uncomplicated, quinolone-resistant)

IV, Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

tabelecleucel

Atara

Epstein-Barr virus-associated post-transplant lymphoproliferative disease

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

tapinarof

Roivant

PSO

Topical

Phase 3 – NDA

TBD

tebipenem pivoxil

Spero

Urinary tract and reproductive tract infections (antibacterial)

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 – NDA

TBD

teplizumab

Provention Bio

T1DM (prevention or delay)

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

tesetaxel

Odonate

Breast cancer

Oral

Phase 3 – NDA

TBD

tezepelumab

AstraZeneca

Asthma (severe, uncontrolled)

SC

Phase 3 – BLA; Breakthrough Therapy

TBD

timbetasin

Regenerx

Dry eye syndrome

Ophthalmic

Phase 3 – BLA

TBD

tirzepatide

Eli Lilly

T1DM

SC

Phase 3 – NDA

TBD

tofersen

Biogen

Amyotrophic lateral sclerosis

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tominersen

Genentech

Huntington’s disease

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tonogenchoncel-L

Kolon Tissuegene

Osteoarthritis

Intraarticular

Phase 3 – BLA

TBD

tradipitant

Vanda

Gastroparesis; Emesis; COVID-19

Oral

Phase 3 – NDA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Novartis

Breast cancer; Gastric/ gastroesophageal cancer

IV

Phase 3 – BLA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Tanvex

Breast cancer; Gastric/ gastroesophageal cancer

IV

Phase 3 – BLA

TBD

trastuzumab duocarmazine

Synthon Bio

Breast cancer

IV

Phase 3 – BLA; Fast Track

TBD

triamcinolone acetonide

Clearside

Uveitis

Intraocular

Phase 3 – 505(b)(2) NDA

TBD

tripotassium citrate monohydrate/potassium hydrogen carbonate microtablet

Advicenne

Renal tubular acidosis

Oral

Phase 3 – NDA

TBD

trivalent hepatitis B vaccine

VBI Vaccines

Hepatitis B infection prevention

IM

Phase 3 – BLA

TBD

trofinetide

Acadia

Rett syndrome

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ublituximab

TG

MS

IV

Phase 3 – BLA

TBD

ublituximab + umbralisib

TG

CLL/SLL

IV + Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

umbralisib

TG

Mantle cell lymphoma

Oral

Phase 3 – NDA

TBD

vadadustat

Akebia

Anemia due to CKD (dialysis-dependent)

Oral

Phase 3 – NDA

TBD

38 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

vazegepant

Biohaven

Migraine treatment; COVID-19

Intranasal

Phase 3 – NDA

TBD

veliparib

Abbvie

Breast cancer; Ovarian cancer

Oral

Phase 3 – NDA

TBD

venglustat

Sanofi

Polycystic kidney disease

Oral

Phase 3 – NDA

TBD

VGX-3100 therapeutic vaccine

Inovio

Cervical dysplasia

IM

Phase 3 – BLA

TBD

vilanterol trifenatate

GlaxoSmithKline

Asthma; COPD

Inhaled

Phase 3 – NDA

TBD

visomitin

Mitotech

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

vonoprazan fumarate

Phathom

Esophagitis; H. pylori infection

Oral

Phase 3 – NDA; QIDP

TBD

VT-1161

Mycovia

Vulvovaginal candidiasis

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

vutrisiran

Alnylam

Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary)

SC

Phase 3 – NDA; Orphan Drug

TBD

ziritaxestat

Galapos

Idiopathic pulmonary fibrosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

zoliflodacin

Entasis

Gonorrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

albuterol (ProAir® RespiClick®)

Teva

COPD

Inhaled

Phase 3 – sNDA

TBD

anakinra (Kineret®)

Swedish Orphan Biovitrum

COVID-19

SC

Phase 3 – sBLA

TBD

bacille Calmette-Guérin (BCG) (TICE® strain) vaccine

Merck

COVID-19

Intradermal

Phase 3 ─ sBLA

TBD

baricitinib (Olumiant®)

Eli Lilly

COVID-19

Oral

Phase 3 – sNDA

TBD

benralizumab (Fasenra®)

AstraZeneca

Nasal polyposis

SC

Phase 3 – sBLA

TBD

brexpiprazole (Rexulti )

Otsuka

Alzheimer’s diseaserelated agitation; PTSD

Oral

Phase 3 – sNDA; Fast Track

TBD

canakinumab (Ilaris®)

Novartis

COVID-19

IV

Phase 3 – BLA

TBD

dapagliflozin (Farxiga)

AstraZeneca

CKD; Diabetic nephropathy; COVID-19

Oral

Phase 3 – sNDA; Breakthrough Therapy; Fast Track

TBD

dupilumab (Dupixent®)

Sanofi

Esophagitis; Pruritus; Urticaria

SC

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

empagliflozin (Jardiance)

Boehringer Ingelheim

Chronic heart failure; CKD; Diabetic nephropathy

Oral

Phase 3 – sNDA; Fast Track

TBD

fenfluramine (Fintepla®)

Zogenix

Lennox-Gastaut syndrome

Oral

Phase 3 – sNDA; Orphan Drug

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 – sNDA

TBD

immunoglobulin 5% (Octagam®)

Octapharma

COVID-19

IV

Phase 3 – sBLA

TBD

mepolizumab (Nucala®)

GlaxoSmithKline

Nasal polyposis

SC

Phase 3 – sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

IV

Phase 3 – sNDA; QIDP TBD

Phase 3 (Supplementals)

®

39 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

nitazoxanide (Alinia®)

Lupin

Influenza treatment; COVID-19

Oral

Phase 3 – sNDA

TBD

omalizumab (Xolair®) autoinjector

Genentech

Food allergies

SC

Phase 3 – sBLA; Breakthrough Therapy

TBD

ozanimod (Zeposia®)

Celgene

UC

Oral

Phase 3 – sNDA

TBD

polatuzumab vedotin-piiq (Polivy®)

Genentech

DLBLC (1st-line)

IV

Phase 3 – sBLA; Orphan Drug

TBD

prasterone (Intrarosa®)

AM

Female sexual arousal disorder

Intravaginal

Phase 3 – sNDA

TBD

ravulizumab-cwvz (Ultomiris®)

Alexion

COVID-19

IV

Phase 3 – sBLA

TBD

rilonacept (Arcalyst®)

Regeneron

Recurrent pericarditis

SC

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

risankizumab-rzaa (Skyrizi®)

Abbvie

PsA; CD; UC

IV, SC

Phase 3 – sBLA; Orphan Drug

TBD

ruxolitinib (Jakafi®)

Incyte

COVID-19

Oral

Phase 3 – sNDA

TBD

sacituzumab govitecanhziy (Trodelvy™)

Immunomedic

Bladder cancer

IV

Phase 3 – sBLA; Fast Track

TBD

sacubitril/valsartan (Entresto)

Novartis

Post-acute myocardial infarction

Oral

Phase 3 – sNDA; Fast Track

TBD

secnidazole (Solosec®)

Lupin

Trichomoniasis

Oral

Phase 3 – sNDA

TBD

selinexor (Xpovio)

Karyopharm

Liposarcoma

Oral

Phase 3 – sNDA; Orphan Drug

TBD

semaglutide (Ozempic®)

Novo Nordisk

Obesity

SC

Phase 3 – sNDA

TBD

teriflunomide (Aubagio )

Sanofi

MS (pediatrics)

Oral

Phase 3 – sNDA

TBD

ticagrelor (Brilinta )

AstraZeneca

Sickle cell disease

Oral

Phase 3 – sNDA

TBD

tisagenlecleucel-t (Kymriah™)

Novartis

DLBCL (relapsed/ refractory in 1st relapse)

IV

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

tocilizumab (Actemra®)

Genentech

COVID-19

IV

Phase 3 – sBLA

TBD

tofacitinib (Xeljanz / Xeljanz XR®)

Pfizer

JIA

Oral

Phase 3 – sNDA

TBD

®

®

®

Complete Response Letter (CRL)/Withdrawn Drugs NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

dantrolene

Eagle

Heat stroke (exertional)

IV

CRL

TBD

diazepam film

Aquestive

Seizure clusters

Oral transmucosal

CRL

TBD

filgotinib (Jyseleca®)

Gilead

RA

Oral

CRL

TBD

sodium thiosulfate

Fennec

Cisplatin-induced ototoxicity prevention

IV

CRL

TBD

terlipressin

Mallinckrodt

Hepatorenal syndrome type 1

IV

CRL

TBD

tramadol

Fortress

Pain (moderate to severe, medically supervised setting)

IV

CRL

TBD

40 | magellanrx.com


PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

valoctocogene roxaparvovec

Biomarin

Hemophilia A

IV

CRL

TBD

veverimer

Tricida

CKD-related metabolic acidosis

Oral

CRL

TBD

viaskin peanut immunotherapy

DBV

Peanut allergy (ages 4 to 11 years)

Transdermal

CRL

TBD

zolmitriptan micro-needle patch

Zosano

Migraine treatment

Transdermal

CRL

TBD

41 | magellanrx.com


GLOSSARY 6MWT 6 Minute Walking Test

CD Crohn's Disease

ABSSSI Acute Bacterial Skin and Skin Structure Infection

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 ACR70 American College of Rheumatology 70% Improvement ADHD Attention Deficit Hyperactivity Disorder ADL Activities of Daily Living AED Anti-Epileptic Drug ALK Anaplastic Lymphoma Kinase ALL Acute Lymphoblastic Leukemia ALT Alanine Transaminase AMD Age-Related Macular Degeneration AML Acute Myeloid Leukemia ANCA Antineutrophil Cytoplasmic Antibody ANDA Abbreviated New Drug Application ARB Angiotensin II Receptor Blocker ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor ART Antiretroviral Therapy ARV Antiretroviral AS Ankylosing Spondylitis ASCVD Atherosclerotic Cardiovascular Disease AST Aspartate Aminotransferase BCVA Best Corrected Visual Acuity BLA Biologics License Application BsUFA Biosimilar User Fee Act BMI Body Mass Index CABP Community Acquired Bacterial Pneumonia CAP Community Acquired Pneumonia 42 | magellanrx.com

CF Cystic Fibrosis CHF Congestive Heart Failure CI Confidence Interval CKD Chronic Kidney Disease CLL Chronic Lymphocytic Leukemia CNS Central Nervous System COPD Chronic Obstructive Pulmonary Disease COVID-19 Coronavirus Disease 2019 CRC Colorectal Cancer CRL Complete Response Letter CSF Colony Stimulating Factor CV Cardiovascular CVD Cardiovascular Disease DAS28-CRP Disease Activity Score-28 with C Reactive Protein DEA Drug Enforcement Administration DLBCL Diffuse Large B Cell Lymphoma DMD Duchenne Muscular Dystrophy DMARD Disease Modifying Antirheumatic Drug DNA Deoxyribonucleic Acid DOR Duration of Response DPP-4 Dipeptidyl Peptidase 4 DR Delayed-Release ECOG Eastern Cooperative Oncology Group EDSS Expanded Disability Status Scale EGFR Epidermal Growth Factor Receptor ER Extended-Release FDA Food and Drug Administration FH Familial Hypercholesterolemia FLT3 FMS-Like Tyrosine Kinase-3


GLOSSARY continued G-CSF Granulocyte Colony Stimulating Factor

LDL-C Low-Density Lipoprotein Cholesterol

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

mAb Monoclonal Antibody

GI Gastrointestinal GIST Gastrointestinal Stromal Tumor GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist GVHD Graft Versus Host Disease H Half HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HCC Hepatocellular Carcinoma HCP Healthcare Professional HCV Hepatitis C Virus HER Human Epidermal Growth Factor Receptor HER2 Human Epidermal Growth Factor Receptor 2 HFA Hydrofluoroalkane HIT Heparin Induced Thrombocytopenia HIV Human Immunodeficiency Virus HIV-1 Human Immunodeficiency Virus-1 HR Hazard Ratio HSCT Hematopoietic Stem Cell Transplant HTN Hypertension IBS Irritable Bowel Syndrome IBS-C Irritable Bowel Syndrome, Constipation Predominant

MACE Major Adverse Cardiovascular Events MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler MRI Magnetic Resonance Imaging MRSA Methicillin-Resistant Staphylococcus Aureus MS Multiple Sclerosis N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NDA New Drug Application NHL Non-Hodgkin Lymphoma NIAID National Institute of Allergy and Infectious Diseases NSAID Non-Steroidal Anti-Inflammatory Drug NSCLC Non-Small Cell Lung Cancer ODT Orally Disintegrating Tablet OR Odds Ratio ORR Overall/Objective Response Rate OS Overall Survival PAH Pulmonary Arterial Hypertension PARP Poly (ADP-ribose) Polymerase PASI 50 Psoriasis Area and Severity Index ≥ 50% PASI 70 Psoriasis Area and Severity Index ≥ 70% PASI 90 Psoriasis Area and Severity Index ≥ 90%

IM Intramuscular

PCI Percutaneous Coronary Intervention

ITP Immune Thrombocytopenic Purpura

PD-1 Programmed Death Protein 1

ITT Intent-To-Treat

PD-L1 Programmed Death-Ligand 1

IV Intravenous

PDUFA Prescription Drug User Fee Application

JIA Juvenile Idiopathic Arthritis

PFS Progression-Free Survival

43 | magellanrx.com


GLOSSARY continued PGA Physician Global Assessment

sPGA Static Physician Global Assessment

PsA Psoriatic Arthritis

SNRI Serotonin and Norepinephrine Reuptake Inhibitor

PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty

SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor

PTSD Post-Traumatic Stress Disorder

SSSI Skin and Skin Structure Infection

Q Quarter

T1DM Type 1 Diabetes Mellitus

QIDP Qualified Infectious Diseases Product

T2DM Type 2 Diabetes Mellitus

QOL Quality of Life

TBD To Be Determined

R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone

TEAE Treatment-Emergent Adverse Events

RA Rheumatoid Arthritis RBC Red Blood Cell RCC Renal Cell Carcinoma REMS Risk Evaluation and Mitigation Strategy RMAT Regenerative Medicine Advanced Therapy RNA Ribonucleic Acid RRR Relative Risk Reduction RSV Respiratory Syncytial Virus RTOR Real-Time Oncology Review sBLA supplemental Biologics License Application SC Subcutaneous SCCHN Squamous Cell Cancer of the Head and Neck SCLC Small Cell Lung Cancer SCT Stem Cell Transplant SGLT2 Sodium-Glucose Co-Transporter-2 SL Sublingual SLE Systemic Lupus Erythematosus SLL Small Lymphocytic Lymphoma sNDA supplemental New Drug Application SOC Standard of Care

44 | magellanrx.com

TNBC Triple Negative Breast Cancer TNF Tumor Necrosis Factor TNFα Tumor Necrosis Factor-alpha UA Unstable Angina 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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