MRx Pipeline - April 2021 by Prime/Magellan Rx

MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS APRIL 2021

EDITORIAL STAFF Maryam Tabatabai, PharmD Editor-in-Chief Vice President, Clinical Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist Consultant Panel Michelle Booth, PharmD Director, Medical Pharmacy Strategy Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist Katie Lockhart Manager, Forecasting and Pharmacoeconomics

Table of CONTENTS

Brian MacDonald, PharmD Senior Manager, Specialty Clinical Programs

EDITOR-IN-CHIEF'S MESSAGE

PIPELINE DEEP DIVE

KEEP ON YOUR RADAR

PIPELINE DRUG LIST

GLOSSARY

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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.

Editor-in-Chief's MESSAGE Welcome to the MRx Pipeline. This quarterly publication offers clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline. MRx Pipeline, our universal forecast, addresses trends applicable across market segments. Traditional and specialty drugs as well as agents under the pharmacy and medical benefits are featured. Also profiled in the report are new molecular entities, pertinent new and expanded indications for existing medications, and biosimilars. Clinical analyses, financial outlook, and pre-regulatory status are considered. The products housed in the MRx Pipeline have been researched in detail. They have been developed in consultation with our internal team of clinical and analytics experts.

METHODOLOGY

Emerging therapeutics continue to grow and influence the clinical and financial landscape. Therefore, Magellan Rx Management has developed a systematic approach to determine the products with significant clinical impact. For the in-depth clinical evaluations, the products’ potential to meet an underserved need in the market by becoming the new standard of care, and the ability to replace existing therapies were investigated. The extent to which the pipeline drugs could shift market share on a formulary and their impact on disease prevalence were also important considerations. In order to assist payers with assessing the potential impact of these pipeline drugs, where available, a financial forecast has been included for select products. Primarily complemented by data from EvaluateTM, this pipeline report looks ahead at the 5-year projected annual US sales through the year 2025. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, including the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business.

LOOKING BACK

Despite the pandemic's unprecedented challenges, in 2020, the US FDA approved 53 novel drugs, making it the second highest number of approvals in 10 years. Thus far in 2021, the agency has approved 18 novel drugs putting it at par with last year’s numbers. Oral options for lupus nephritis, MS, oncology, and heart failure as well as a oncemonthly injectable for HIV are among these approvals. Notably, 83% of agents approved so far in 2021 use at least 1 of the FDA’s expedited approval methods and 53% are designated as Orphan Drugs. Furthermore, a single-dose COVID-19 vaccine received Emergency Use Authorization (EUA) later followed by an 11-day temporary pause to review a safety signal, after which its use was resumed by the CDC and FDA. While numbers do not tell the entire story, they do represent incredible advances in patient care and hope for the American public.

ON THE HORIZON

As we look ahead, there is a continued trend toward the approval of specialty medications and drugs for rare and ultra rare diseases, with 63% and 34% of approvals expected, respectively, for agents with applications submitted to the FDA. There are 6 agents seeking FDA’s Accelerated Approval, which allows for earlier drug approval for serious conditions that fill an unmet need based on a surrogate endpoint. The growth of biosimilars including biosimilar insulin and new treatment modalities using gene therapy are expected. This will be a pivotal year in combatting COVID-19 with a public health arsenal of COVID-19 therapeutics and next-generation vaccines to target an evolving virus. Other noteworthy pipeline trends to watch include the development of complex therapies, oncology, immunology, immunodermatology, therapeutic options for rare hereditary diseases, and a new therapy for Alzheimer’s disease, with 2025 US sales forecast exceeding $2 billion. Moreover, sprouting products for hematology, ophthalmology, and women’s health await on the horizon. The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Innovative agents that show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm. Maryam Tabatabai, PharmD Editor-in-Chief, MRx Pipeline

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

SPECIALTY

PRIORITY REVIEW

BREAKTHROUGH THERAPY

92%

35%

15%

BIOSIMILAR

ORPHAN DRUG

54%

27%

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

IMMUNOLOGY

anifrolumab IV AstraZeneca/Bristol-Myers Squibb PROPOSED INDICATIONS

Systemic lupus erythematosus (SLE)

CLINICAL OVERVIEW

Anifrolumab is a human immunoglobulin G1 kappa (IgG1K ) monoclonal antibody directed against the type 1 interferon receptor subunit 1 involved in inflammatory responses. Two multinational, randomized, double-blind, placebo-controlled, phase 3 clinical trials, TULIP 1 and TULIP 2, evaluated the efficacy and safety of anifrolumab 300 mg IV every 4 weeks for 48 weeks in patients 18 to 70 years of age with moderately to severely active autoantibody-positive SLE who were receiving stable SOC therapy (e.g., oral corticosteroids [OCS], azathioprine, mizoribine [not approved in the US], mycophenolate, methotrexate). In TULIP-1 (n=457), anifrolumab did not meet its primary endpoint of reduction in disease activity measured by the SLE Responder Index 4 (SRI4) as compared to placebo (p=0.412). However, TULIP-2 (n=365) met its primary endpoint of British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA), and reported a significantly greater overall response with anifrolumab than placebo (47.8% versus 31.5%, respectively; p=0.001); similar responses were seen in patients with high or low interferon gene signature. Notably, TULIP-1 also reported a significant reduction in disease activity based on BICLA as a secondary endpoint. A key difference between the 2 endpoint measures is SRI4 requires complete improvement in at least 1 organ, whereas BICLA requires partial improvement across all organs; both measures require no new flares. In addition, both trials demonstrated similar reductions in OCS use (target, ≤ 7.5 mg/day) and improvement in severity of SLE cutaneous manifestations (≥ 50% reduction). Herpes zoster infection was reported in patients who received anifrolumab (TULIP 1, 5.6%; TULIP 2, 8.3%). While serious adverse events were reported less often with anifrolumab (8.3%) than with placebo (17%); 1 death due to pneumonia occurred in the anifrolumab arm.

PLACE IN THERAPY

SLE is an autoimmune inflammatory disorder that can involve multiple organ systems. It is estimated to affect 1.5 million people in the US. It is diagnosed predominantly in women of child-bearing age, particularly in women of color (e.g., Black, Hispanic/Latino, Asian, Native American). Disease severity ranges from mild to life-threatening, and symptom flares and remission can occur. It is estimated that 60% to 80% of adults with SLE have an increased type I interferon gene signature, which correlates with disease activity. There is no cure for SLE. Pharmacological therapy includes OCSs, immunosuppressive agents, hydroxychloroquine, and the B-lymphocyte stimulator (BLysS)-specific inhibitor belimumab (Benlysta®). If approved, anifrolumab will provide an HCP-administered option with a novel mechanism of action. Both anifrolumab and belimumab are administered IV every 4 weeks in adults. Belimumab IV is also approved for SLE in pediatrics ≥ 5 years of age and a SC formulation is also approved for use in adults. Notably, approval of belimumab was based on significant improvement in SRI4, an endpoint that was not met by anifrolumab in phase 3 trials. Several other products with varying mechanisms of action are also in phase 3 trials for SLE. In addition, anifrolumab is in phase 2 studies for lupus nephritis, an indication held by belimumab and the recently approved oral voclosporin (Lupkynis™). A SC formulation of anifrolumab for SLE is in phase 2 trials.

FDA APPROVAL TIMELINE July to December 2021  Fast Track

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$15

$82

$158

$226

$282

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

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IMMUNOLOGY

avapritinib (Ayvakit™) oral Blueprint Medicines PROPOSED INDICATIONS Systemic mastocytosis (SM)

Avapritinib (Ayvakit) is already approved for the treatment of unresectable or metastatic gastrointestinal stromal tumor (GIST) with a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

CLINICAL OVERVIEW

Avapritinib is a tyrosine kinase inhibitor that targets PDGFRA and PDGFRA D842 mutants as well as multiple KIT exon 11, 11/17, and 17 mutants. The double-blind, phase 2 PIONEER trial assessed avapritinib in adults (n=39) with indolent SM with moderate to severe SM symptoms and ECOG Performance Status 0 to 2. The KIT D816V mutation was detected in 95% of patients. Response was defined as ≥ 30% reduction in the patient-reported Indolent Systemic Mastocytosis-Symptom Assessment Form Total Symptom Score (ISM-SAF TSS), which records symptom scores from 0 (none) to 10 (worst), including abdominal and bone pain, diarrhea, nausea, cutaneous symptoms, neurocognitive symptoms, and fatigue. In part 1 of the study (dose-finding), at 24 weeks, the response rate in patients randomized to avapritinib 25 mg orally once daily was 60% versus 0% in patients who were given placebo. Based on all avapritinib doses evaluated (25 mg, 50 mg, 100 mg; n=10 for each), skin lesions lightened by 71% with avapritinib versus 25% with placebo. Moreover, there was a median 46% decrease in mast cell infiltration with avapritinib (all doses) compared to a 51% increase with placebo. Fatigue, headache, dizziness, nausea, and edema were the most commonly reported TEAEs; no grade ≥ 3 TEAEs were reported with avapritinib 25 mg, which is the dose selected for part 2 of the study (efficacy phase).

PLACE IN THERAPY

SM is most commonly caused by alterations in D816V in the KIT gene which leads to overproduction of mast cells that ultimately accumulate in organs including the liver, spleen, bone, and small intestines. Onset of symptoms occur in children and adults, and range from GI disturbances (diarrhea, nausea), cutaneous symptoms (urticaria pigmentosa), hepatomegaly, splenomegaly, lymphadenopathy, anemia, and anaphylactoid reaction. Symptoms and their severity vary based on type of SM and organ involvement. Indolent SM is the most common type of SM and progresses slowly with a low mast cell burden. The other types include systemic smoldering mastocytosis, SM with an associated hematologic non-mast cell lineage (SM-AHN; associated with myelodysplastic conditions), aggressive SM (ASM; may lead to organ impairment/failure due to aggressive mast cell infiltration), mast cell leukemia (MCL; rare), and mast cell sarcoma. There is currently no cure for SM. Treatment consists of antihistamines, proton pump inhibitors, epinephrine, steroids, mast cell stabilizers (ketotifen), leukotriene modifiers, cromolyn sodium, bisphosphonates, and psoralen plus ultraviolet A radiation. An interferon, immune modulator, or chemotherapy may be required for aggressive forms of the disease. Currently, there are 2 kinase inhibitors FDA-approved to treat SM; these include midostaurin (Rydapt®), indicated for ASM, SM-AHN, and MCL (in combination with chemotherapy), and imatinib (Gleevec®, generics), indicated in select adults with ASM. If approved, avapritinib could compete with midostaurin, which has a similar response rate (63%) to avapritinib in patients with the KIT D816V mutation.

FDA APPROVAL TIMELINE June 16, 2021

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$10

$67

$164

$312

$440

The forecast is a projection of total US sales per year. 5 | MAGELLANRX.COM

NEUROLOGIC

difelikefalin IV Cara PROPOSED INDICATIONS

Chronic kidney disease-associated pruritus (CKD-aP)

CLINICAL OVERVIEW

Difelikefalin is a first-in-class potent and highly selective kappa opioid receptor agonist that targets the peripheral nervous system and select immune cells. In the US-based, double-blind, phase 3 KALM-1 trial, 378 patients undergoing hemodialysis with moderate to severe pruritus were randomized to difelikefalin or placebo. After 12 weeks of therapy, 51.9% of patients given difelikefalin experienced a ≥ 3-point decrease from baseline in the weekly mean 24-hour Worst Itching Intensity Numerical Rating Scale (WI-NRS; primary endpoint), which ranges from 0 (best) to 10 (worst) compared to 30.9% in those who received placebo (p<0.001). In addition, the proportion of patients who experienced a ≥ 4-point decrease from baseline in 24-hour WI-NRS (secondary outcome) was 37.1% with difelikefalin and 17.9% with placebo. More patients in the difelikefalin group also reported a significant improvement in itch-related QOL compared to the placebo group as measured by the 5-D Itch and the Skinex-10 scales. The most common adverse effects reported with difelikefalin were diarrhea, dizziness, and vomiting, which were generally mild to moderate in severity. No evidence of abuse or physical dependence to difelikefalin was observed. There were also no reports of hallucination or dysphoria. The global KALM-2 trial (n=473) reported similar findings. In both trials, the difelikefalin dosage was 0.5 mcg/kg body weight administered after each dialysis session as an IV bolus into the venous port of the dialysis circuit.

PLACE IN THERAPY

CKD-aP, also known as uremic pruritus, is defined as itching that is associated with kidney disease when no other comorbid condition (e.g., liver disease, skin condition) can be determined to be the cause. The persistent itching of CKD-aP can lead to poor QOL, impaired sleep, and depression. CKD-aP affects approximately 40% of patients with ESRD and over 60% of those undergoing hemodialysis. CKD-aP is typically bilateral in nature and may lessen over time in patients on dialysis. While the precise cause of CKD-aP is unknown, several factors may contribute, including systemic inflammation, altered nociceptive sensory pathways, opioid receptor dysfunction, and skin alterations. Emollients are the preferred topical treatment of CKD-aP. Topical analgesics may also provide relief of CKD-aP. With the exception of gabapentin, off-label use of systemic therapies (e.g., antihistamines, mast cell stabilizers) has not proven beneficial. Although gabapentinoids may provide relief of itching, there are concerns for abuse/misuse and the risk of falls with their use; these concerns have not been described with difelikefalin. If approved, IV difelikefalin will be the first opioid agonist that targets the kappa receptor to treat CDK-aP. An oral formulation of the agent is in development. Difelikefalin is also in phase 3 trials for post-surgical pain. Notably, the oral opioid agonist/antagonist nalbuphine ER is in phase 3 trials for CKD-aP.

FDA APPROVAL TIMELINE August 23, 2021  Breakthrough

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$14

$94

$163

$215

$265

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

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DIABETES

donislecel IV Celltrans PROPOSED INDICATIONS

Treatment of brittle type 1 diabetes mellitus (T1DM)

CLINICAL OVERVIEW

Doniselcel comprises purified allogeneic islets of Langerhans derived from the pancrease of deceased donors. Islets contain insulin-secreting beta cells that are important in glucose metabolism. An open-label, phase 3 trial enrolled 21 patients with T1DM to evaluate donislecel. Patients received up to 3 transplants with donislecel according to the University of Illinois at Chicago protocol, which includes the use of basiliximab, tacrolimus, sirolimus, etanercept, and exenatide to promote patient safety and graft survival. Median age was 47 years (range, 21 to 67 years). Among the 19 patients who completed the trial, a total of 10 and 12 patients were free from exogenous insulin use 365 days after the first and last transplants, respectively. Among 11 patients analyzed for hypoglycemia, there was a 66.1% and 90.1% reduction in hypoglycemic severity at 365 days after the first and last transplants, respectively, as measured by the patientreported Ryan composite hypoglycemic score. All patients were followed for safety for 1 year. The most common TEAEs (≥ 20%) were anemia, vomiting, and hyponatremia. Pneumonia was reported in 2 patients. Serious events that were reported in 1 patient each included anemia, pancytopenia, myocardial ischemia, hypoglycemia, intra-abdominal hemorrhage, cholecystitis, legionella pneumonia, and uterine leiomyoma.

PLACE IN THERAPY

It is estimated that nearly 1.6 million people in the US have T1DM, including 187,000 children and adolescents. T1DM is due to an autoimmune response that destroys the beta cells of the pancreas over months or years. Risk factors for T1DM are uncertain, but genetic predisposition and environmental factors (e.g., viral infection) may play a role. In a small proportion of patients with T1DM, the condition is considered “brittle” if the patient experiences frequent, hard-to-control blood glucose fluctuations between hyper-and hypo-glycemia. There is currently no cure for T1DM. Strategies to manage the disease include use of exogenous insulin and the amylin analog pramlintide (Symlin®). Despite pharmacologic treatment, hypoglycemic unawareness and failure to achieve glycemic targets remain challenges in this population. Advancing technologies for glucose monitoring and insulin delivery, including continuous glucose monitoring (CGM), sensor-augmented insulin pumps with automatic low glucose insulin dose suspension, and the MiniMed™ 670G “artificial pancreas,” are intended to help control glucose levels and mitigate hypoglycemia. In a small phase 3 trial, islet cell transplantation with donislecel resulted in insulin independence, restored near normal glycemic control, and eliminated severe hypoglycemia in T1DM patients. If approved, donislecel will be the first islet transplant product for T1DM. Its use may be limited by the need for several donors and the need for life-long immunosuppression. Moreover, for unknown reasons, islet graft function may diminish over time; some data suggest the use of etanercept may contribute.

FDA APPROVAL TIMELINE

While the approval timeline for the FDA decision has not been announced, the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee reviewed donislecel on April 15, 2021. The committee voted 12 to 4 (and 1 abstention) in favor of approval of the product. The panel also noted that the target population for donislecel will be small, given the availability of the advanced technologies mentioned above.  Orphan Drug

FINANCIAL FORECAST (reported in millions)

The financial forecast for donislecel is not currently available.

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INFECTIOUS DISEASE

ibrexafungerp oral Scynexis PROPOSED INDICATIONS

Vulvovaginal candidiasis (VVC)

CLINICAL OVERVIEW

Ibrexafungerp is a glucan synthase inhibitor that disrupts the synthesis of polymer β-(1,3) D-glucan in the fungal cell wall. Ibrexafungerp has demonstrated activity against multidrug-resistant pathogens, including azole- and echinocandin-resistant strains. The randomized, double-blind, phase 3 VANISH-303 (n=376) and VANISH-306 (n=455) trials demonstrated superiority of oral ibrexafungerp over placebo for the treatment of postmenarchal females ≥ 12 years of age with acute VVC due to Candida species confirmed via culture. In the 2 trials, ibrexafungerp demonstrated a clinical cure rate at day 10 (primary endpoint) of 50.5% and 63.3%, respectively. Rates achieved in the secondary endpoint of mycological eradication at day 10 in each study were 49.5% and 58.5%, respectively. In the phase 2b DOVE trial in women with moderate to severe acute VVC (n=153), ibrexafungerp demonstrated similar response rates at day 10 as oral fluconazole (clinical cure, 52% [14 of 27] versus 58% [14 of 24], respectively; mycological eradication, 63% for both). In addition, rates of clinical cure at day 25 were higher with ibrexafungerp than with fluconazole (70% versus 50%, respectively). Ibrexafungerp was generally well tolerated. Mild GI events (diarrhea, nausea) were reported. In the clinical trials, patients received 2 oral doses of ibrexafungerp 300 mg taken 12 hours apart.

PLACE IN THERAPY

VVC is the second leading cause of vaginitis and results in approximately 1.4 million outpatient visits each year in the US. Increased risk factors for VVC include pregnancy, diabetes, immunosuppression, and medications (e.g., hormonal contraceptives, antibiotics). The majority of cases are uncomplicated and caused by Candida albicans. Approximately 10% of cases are considered to be complicated, comprising severe or recurrent disease and infection due to other Candida species. Treatment options for VVC include OTC topical antifungals and prescription oral fluconazole (available as generic). Notably, resistance to azole antifungals is rare with C. albicans but has been reported with other Candida species. However, the longer duration needed for recurrent VVC can lead to azole resistance of C. albicans. In phase 3 trials, oral ibrexafungerp was effective in treating VVC, and the phase 2b DOVE study suggests similar efficacy and more persistent antifungal activity compared to fluconazole. No study data announced to date have addressed its use in pregnant women. If approved, ibrexafungerp will be the first oral glucan synthase inhibitor to treat VVC. Other agents that inhibit glucan synthase, the echinocandin antifungals, are administered IV and are not approved for VVC. Oral ibrexafungerp is also being evaluated for prevention of recurrent VVC, an area with no FDA-approved therapies; the trial investigating this indication enrolled women with VVC who failed fluconazole. Oral ibrexafungerp is also in phase 3 trials in adults for systemic Candida spp. infections, including emergency use and for cases refractory to standard therapy.

FDA APPROVAL TIMELINE June 1, 2021  Fast Track

 Orphan Drug

 Priority Review

 QIDP

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$35

$99

$162

$224

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

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HEMATOLOGY

narsoplimab IV Omeros PROPOSED INDICATIONS

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA)

CLINICAL OVERVIEW

Narsoplimab is a human monoclonal antibody that inhibits mannan-binding lectin-associated serine protease-2 (MASP-2), an effector enzyme of the lectin pathway of complement. Notably, narsoplimab is not expected to interfere with the antibody-dependent classical complement activation pathway that is essential in the immune response to infection. A pivotal, single-arm, phase 3 trial evaluated narsoplimab in 28 adults with HSCT-TMA. At baseline, a large majority of patients enrolled had multiple comorbidities (e.g., GVHD, significant infections, multiorgan dysfunction). The primary endpoint of complete response required clinical improvement in TMA markers (e.g., platelet count, lactate dehydrogenase), and in organ function (renal, pulmonary, GI, or neurological) or freedom from transfusion. A complete response was reported in 61% and 74% of patients who received ≥ 1 dose (n=28) and ≥ 4 doses (n=23) of narsoplimab, respectively (p<0.0001 compared to the 15% threshold for both). The secondary endpoint of 100-day survival was reported in 68% and 83% in patients who received ≥ 1 dose and ≥ 4 doses of narsoplimab, respectively, and 94% (14/15) in complete responders. In addition, the median overall survival (OS) was 274 days and 361 days among those who received ≥ 1 dose and ≥ 4 doses, respectively; OS was not estimable in complete responders. No safety signals were identified. Adverse events included fever, diarrhea, vomiting, nausea, and neutropenia, which are characteristic of the post-HSCT population. Six deaths were reported due to causes consistent with HSCT. Narsoplimab was administered IV once weekly for up to 8 weeks with a 6-week follow-up period. The specific narsoplimab dose used in the phase 3 study has not been published.

PLACE IN THERAPY

HSCT-TMA is a post-transplant complication that can be severe or life-threatening and occurs within 100 days of transplant. It involves systemic vascular endothelial injury which leads to intravascular platelet activation, thrombi formation, and vessel wall damage. HSCT-TMA may be triggered by multiple mechanisms during the HSCT process, and contributing factors may include calcineurin inhibitor (CNI) use, GVHD, and select viral infections. Initial therapy calls for treatment of comorbid conditions (e.g., infection, GVHD) as well as discontinuation of instigating medications (e.g., CNIs, sirolimus, tacrolimus, cyclosporine). If approved, narsoplimab will be the first agent FDA-approved to treat HSCT-TMA. Narsoplimab (≥ 4 doses) produced a much longer overall survival compared to historical data for this condition (361 versus 21 days, respectively). Other agents in phase 3 development for HSCT-TMA are the complement inhibitors ravulizumab (Ultomiris®) and investigational nomacopan. Narsoplimab and nomacopan are also being evaluated for the thrombotic microangiopathy condition, hemolytic uremic syndrome (HUS), which is an indication already held by ravulizumab. SC administration of narsoplimab is under investigation.

FDA APPROVAL TIMELINE July 16, 2021

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$18

$46

$79

$118

$191

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

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METABOLIC

odevixibat oral Albireo PROPOSED INDICATIONS

Progressive familial intrahepatic cholestasis (PFIC)-related pruritus

CLINICAL OVERVIEW

Odevixibat is a non-systemic ileal bile acid transport (IBAT) inhibitor that decreases the reabsorption of bile acids into the distal portion of the small intestine. The 24-week, double-blind, phase 3 PEDFIC-1 trial evaluated odevixibat in 62 patients ages 6 months to 18 years with genetically confirmed PFIC type 1 or 2 (PFIC1 [27%], PFIC2 [73%]), elevated serum bile acids (sBAs), and a history of significant pruritus. Patients were randomized to odevixibat 40 mcg/kg (O-40) or 120 mcg/kg (O-120) orally once daily or placebo. Both doses of odevixibat produced statistically significant improvement in the proportion of positive pruritus assessments (PPAs) (O-40, 58.3% [p=0.003]; O-120, 51.8% [p=0.033]; placebo, 30.1%) at 24 weeks (US primary endpoint). In addition, significant sBA response, defined as ≥ 70% reduction from baseline or sBAs ≤ 70 μmol/L at 24 weeks (European primary endpoint), was reported with O-40 (43.5%; p=0.0006) and O-120 (21.1%, p=0.035) compared to placebo (0%). The mean reduction in sBAs was 114.6 μmol/L (38%) with odevixibat (doses combined) versus a mean increase by 13.1 μmol/L with placebo. Odevixibat was well tolerated. The most common TEAE was diarrhea/frequent bowel movement (9.5% versus 5% with placebo). In addition, the open-label PEDFIC-2 extension trial demonstrated efficacy and safety for an additional 24 weeks with O-120, the planned commercial formulation. After 48 weeks of therapy, improvements in height (p=0.02) and weight (p=0.03) were observed.

PLACE IN THERAPY

PFIC affects approximately 1 in 50,000 to 100,000 births. The ultra-rare group of autosomal recessive diseases is caused by mutations in genes that produce proteins involved in biliary epithelial transport. When these proteins are deficient, high levels of sBAs accumulate in the liver and blood and lead to severe itching (the hallmark feature of PFIC), nutritional imbalances, and eventual cirrhosis. Onset of signs and symptoms of PFIC typically occurs during infancy. Dietary treatment is required to correct nutritional imbalances and associated complications (e.g., fat and fat-soluble vitamin malabsorption, failure to thrive, osteopenia, visual changes, muscle/neurological complications, blood clotting difficulty). Ursodeoxycholic acid may slow disease progression, prevent liver damage, and relieve pruritus in some patients, and surgical procedures (e.g., biliary diversion, nasobiliary drainage) may be required to prevent sBA accumulation in the liver. Ultimately, most patients will need a liver transplant, which may not resolve the non-hepatic symptoms (e.g., diarrhea), and the original disease may recur. The severe and debilitating itching with PFIC is typically refractory to the usual medications used for pruritus (e.g., antihistamines). If approved, odevixibat will be the first agent approved to treat PFIC-related pruritus. It is also in phase 3 trials for Alagille syndrome and biliary atresia. The IBAT inhibitor maralixibat is also in phase 3 trials for PFIC and has been submitted for FDA approval for Alagille syndrome.

FDA APPROVAL TIMELINE July 20, 2021  Fast Track

 Orphan Drug

 Priority Review

 Rare Pediatric Disease

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$47

$81

$123

$176

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

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IMMUNODERMATOLOGY

ruxolitinib topical Incyte PROPOSED INDICATIONS

Mild to moderate atopic dermatitis (AD)

CLINICAL OVERVIEW

Multiple proinflammatory cytokines are dependent on the Janus kinase (JAK)-signal transducer and activator of transcription (STAT) signaling pathway to mediate their effects. Ruxolitinib cream is a selective JAK1/JAK2 inhibitor designed for topical administration. Two 8-week, double-blind, vehicle-controlled, phase 3 trials, TRuE-AD1 and TRuE-AD2, evaluated ruxolitinib cream in a total of 1,208 patients ≥ 12 years of age with mild to moderate AD. Two strengths of ruxolitinib were assessed, 0.75% and 1.5%, which were applied topically twice daily. Pooled data revealed that significantly more patients treated with ruxolitinib 0.75% and 1.5% achieved the primary endpoint of IGA score of 0 (clear) or 1 (almost clear) with at least a 2-point improvement from baseline to week 8 compared to vehicle (44.7%, 52.6%, and 11.5%, respectively). Significantly more patients treated with ruxolitinib cream 0.75% and 1.5% also achieved EASI-75 compared to vehicle (53.8%, 62%, and 19.7%, respectively). In addition, ruxolitinib cream resulted in significantly greater improvement in itch and sleep measurements; relief from itching was observed within 12 hours of topical application. Adverse events reported were similar between the treatment and control arms. In addition, in a phase 2 trial, ruxolitinib 1.5% cream (n=51) was found to be at least as effective as triamcinolone 0.1% cream (n=51) based on EASI-75 and was not generally associated with application-site burning and stinging as experienced with TCSs.

PLACE IN THERAPY

AD, also known as eczema, affects approximately 31.6 million people in the US, including 9.6 million children and adolescents. It is characterized by dry, itchy, eczematous skin lesions. This chronic inflammatory skin disease is commonly associated with food allergy, allergic rhinitis, and asthma. Further, there is a link between AD and stress, anxiety, and depression, which underscores the importance of mental healthcare and a multidisciplinary approach in AD management. Emollients and TCSs are the mainstay of AD treatment. Topical second-line therapy in patients ≥ 2 years of age includes the calcineurin inhibitors (CNIs) pimecrolimus (Elidel®, generic) and tacrolimus (Protopic®, generic) which are approved for mild to moderate and moderate to severe cases, respectively. In addition, the topical phosphodiesterase 4 (PDE4) inhibitor crisaborole (Eucrisa®) is approved for mild to moderate AD in ages ≥ 3 months. Long-term continuous use of TCSs and CNIs is limited by adverse effects. Oral JAK inhibitors, including oral ruxolitinib (Jakafi®), are available to treat various immune disorders. Baricitinib (Olumiant®) and upadacitinib (Rinvoq™) as well as investigational abrocitinib have been submitted to the FDA and could be the first oral JAK inhibitors approved for moderate to severe AD in patients ≥ 12 years of age. If approved, ruxolitinib cream will be the first topical JAK inhibitor for AD. Its use may avoid the toxicities associated with the oral agents, such as serious infections, malignancy, and thrombosis. Long-term safety data from the ongoing TRuE-AD trials may help differentiate ruxolitinib cream from the oral agents. Notably, in a phase 2 trial, ruxolitinib cream was at least as effective as the medium-potency TCS triamcinolone and did not cause application site irritation. Ruxolitinib cream is also in phase 3 trials for vitiligo.

FDA APPROVAL TIMELINE June 21, 2021

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$17

$90

$169

$261

$349

The forecast is a projection of total US sales per year. 11 | MAGELLANRX.COM

INFECTIOUS DISEASE

sulopenem etzadroxil/probenecid oral Iterum PROPOSED INDICATIONS

Quinolone-resistant uncomplicated urinary tract infections (uUTI)

CLINICAL OVERVIEW

Sulopenem is an orally bioavailable, broad-spectrum antibiotic for the treatment of gram-positive, gramnegative, and anaerobic pathogens. The randomized, multicenter, double-blind, phase 3 SURE-1 trial compared safety and efficacy of sulopenem etzadroxil/probenecid for 5 days and ciprofloxacin for 3 days in 1,670 adult women with uUTI. Both oral therapies were dosed twice daily. The end of treatment (EOT) visit occurred on day 5, and the test of cure (TOC) visit on day 12. In quinolone-resistant patients, sulopenem etzadroxil/probenecid was superior to ciprofloxacin regarding overall EOT response (64.6% versus 30.2%, respectively), overall TOC response (62.6% versus 36%, respectively), and clinical TOC response (83% versus 62.6%, respectively) (p<0.001 for all). However, in quinolone-susceptible patients, higher response rates were not observed in any of the 3 measures with sulopenem etzadroxil/probenecid compared to ciprofloxacin. The incidence and type of TEAEs were similar for both drugs, with diarrhea, nausea, and headache (all < 8%) as the most commonly reported TEAEs.

PLACE IN THERAPY

UTIs account for over 6 million healthcare visits, approximately 20% of which are at emergency departments. The infection occurs more often in women and in older individuals. The causal pathogen is usually Escherichia coli (75%), but other pathogens including Klebsiella pneumoniae, Proteus mirabilis, and Staphylococcus saprophyticus have been detected. First-line treatments for uUTI in women include nitrofurantoin, trimethoprim-sulfamethoxazole (TMP-SMX), and fosfomycin. Fluoroquinolones or β-lactams may be selected as second-line or alternate therapies due to resistance patterns and/or allergy considerations, and empiric treatment may vary based on the regional antimicrobial resistance patterns. In general, E. coli isolates show high in vitro susceptibility to nitrofurantoin and fosfomycin. Resistance to TMP-SMX, oral cephalosporins, amoxicillin-clavulanate, and fluoroquinolones is generally low (< 10%); however, resistance, particularly to fluoroquinolones, is increasing. If approved, sulopenem will be the first oral penem antibiotic available in the US. In a pivotal study, it was generally well tolerated and was superior to ciprofloxacin in women with quinolone-resistant uUTI. In this space, sulopenem etzadroxil/probenecid could compete with ciprofloxacin, which has been associated with microbial resistance and serious adverse effects (e.g., tendon complications, CNS effects, peripheral neuropathy). IV-administered sulopenem, followed by the oral formulation, is currently in phase 3 trials for complicated UTI (cUTI) (SURE-2) and complicated intra-abdominal infections (SURE-3).

FDA APPROVAL TIMELINE July 23, 2021  Fast Track

 Priority Review

 QIDP

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$39

$90

$150

$222

The forecast is a projection of total US sales per year for the treatment of UTIs and could include complicated and uncomplicated cases.

12 | MAGELLANRX.COM

DIABETES

teplizumab IV Provention Bio PROPOSED INDICATIONS

Type 1 diabetes mellitus (T1DM) delay/prevention in at-risk individuals

CLINICAL OVERVIEW

Teplizumab is an Fc receptor–nonbinding anti-CD3 monoclonal antibody that modifies CD8+ T lymphocytes that are thought to be involved in destruction of pancreatic beta cells. By inhibiting this activity, teplizumab preserves beta cell function. The pivotal double-blind, phase 2 At-Risk TN-10 study evaluated teplizumab in 76 patients 8 to 49 years of age who were presymptomatic or at-risk for T1DM. At-risk was defined as having ≥ 2 T1DM-related autoantibodies and dysglycemia. At a median follow-up of 2.5 years, more than twice as many patients in the teplizumab group were free of clinical T1DM compared to patients in the placebo group (50% versus 22%, respectively; HR, 0.457; p=0.01). Teplizumab delayed the median time to T1DM diagnosis (primary endpoint) compared to placebo (59.6 versus 27.1 months, respectively; HR, 0.457; p=0.01). The study also revealed teplizumab was associated with an increased insulin secretory capacity suggesting improved beta cell function. Transient lymphopenia and rash were reported with teplizumab. Teplizumab was administered IV once daily as a single course of therapy in an outpatient setting. The dose was 51 mcg/m2, 103 mcg/m2, 207 mcg/m2, and 413 mcg/m2 on days 1, 2, 3, and 4, respectively, followed by 826 mcg/m2 once daily on days 5 through 14. Notably, in the earlier phase 2/3 PROTÉGÉ trial, teplizumab (across 3 dosing regimens) did not meet its primary composite outcome of the percentage of patients with insulin use < 0.5 U/kg/day and HbA1C < 6.5% at 1 year.

PLACE IN THERAPY

It is estimated that nearly 1.6 million people in the US have T1DM, including 187,000 children and adolescents. T1DM is due to an autoimmune response that destroys the beta cells of the pancreas over months or years. Risk factors for T1DM are uncertain, but genetic predisposition and environmental factors (e.g., viral infection) may play a role. In predisposed patients, onset of T1DM is preceded by an asymptomatic stage that includes the appearance of autoantibodies (stage 1) followed by dysglycemia (stage 2). Data reveal that during stage 2, metabolic responses to a glucose load are impaired, but HbA1c is normal. Researchers suggest that these immunologic and metabolic features can identify people at high risk for T1DM. There is no cure for T1DM. Current strategies to manage the disease include use of exogenous insulins and the amylin analog pramlintide (Symlin). Despite pharmacologic treatment, failure to achieve glycemic targets and risk of diabetic complications are still prevalent. In the At-Risk TN-10 trial, teplizumab demonstrated a delay in T1DM onset in pediatrics and adults during stage 2 of presymptomatic progression. If approved, teplizumab will be the first DMT to prevent or delay onset of T1DM. However, it will likely face challenges in uptake, including reimbursement hurdles due to an anticipated high cost and the current lack of a screening protocol for T1DM.

FDA APPROVAL TIMELINE

July 2, 2021 (The FDA's Endocrinologic and Metabolic Drugs Advisory Committee will review teplizumab on May 27, 2021)  Breakthrough Therapy

 Orphan Drug

FINANCIAL FORECAST (reported in millions)

 Priority Review

The financial forecast for teplizumab is not currently available.

13 | MAGELLANRX.COM

IMMUNOLOGY

tralokinumab SC AstraZeneca PROPOSED INDICATIONS

Moderate to severe atopic dermatitis (AD)

CLINICAL OVERVIEW

Tralokinumab is a human monoclonal antibody that inhibits interleukin-13 (IL-13), a cytokine which plays a key role in AD inflammation. Three 52-week, randomized, double-blind, placebo-controlled, phase 3 studies evaluated the safety and efficacy of tralokinumab administered SC every 2 weeks in adults (total n=1,976) with moderate to severe AD. ECZTRA-1 and ECZTRA-2 studied tralokinumab as monotherapy, and ECZTRA-3 assessed it in combination with a topical corticosteroid (TCS). In all 3 trials, at week 16, significantly more patients who received tralokinumab achieved the primary endpoint of IGA of clear (0) or almost clear (1) (reported as tralokinumab versus placebo incidence, respectively) (ECZTRA-1: 15.8% versus 7.1% [p=0.002]; ECZTRA-2: 22.2% versus 10.9% [p<0.001]; ECZTRA-3: 38.9% versus 26.2% [p=0.015]). Among the 3 trials, tralokinumab also led to significantly more patients achieving the primary endpoint of EASI-75 at week 16 (reported as tralokinumab versus placebo incidence, respectively) (ECZTRA-1: 25% versus 12.7%; ECZTRA-2: 33.2% versus 11.4%; ECZTRA-3: 56% versus 35.7%; p<0.001 for all). At week 16, the patients who responded to tralokinumab in each trial were rerandomized to tralokinumab administered every 2 weeks (Q2W) or every 4 weeks (Q4W) or to placebo. In the ECZTRA-1 and -2 monotherapy trials, 51% and 59%, respectively, of patients who continued tralokinumab Q2W maintained IGA response at week 52 without any use of TCSs. In addition, 39% and 45%, respectively, of patients who changed to tralokinumab Q4W maintained IGA response at week 52. In ECZTRA-3, 89.6% and 77.6% of those treated with tralokinumab Q2W and Q4W, respectively, maintained an IGA response at week 52. Safety profiles for tralokinumab and placebo were similar, with upper respiratory tract infections and conjunctivitis reported more often with tralokinumab. In the studies, tralokinumab was administered SC as a 600 mg loading dose, followed by 300 mg Q2W until week 16, then every Q2W or Q4W for an additional 36 weeks.

PLACE IN THERAPY

AD affects an estimated 31.6 million people in the US, including 9.6 million children and adolescents. Approximately 30% and 40% of cases in pediatrics and adults, respectively, are moderate to severe. While several systemic DMTs are available for moderate and severe AD, topical emollients, steroids, and immunomodulators are still essential in AD treatment; however, TEAEs of TCSs and immunomodulators may limit their long-term continuous use. If approved, tralokinumab, administered with or without a TCS, will be the first biologic for AD that solely targets IL-13. It has the potential to compete with dupilumab (Dupixent®) in biologic-naïve patients with moderate to severe disease. While tralokinumab targets IL-13, dupilumab inhibits both IL-13 and interleukin-4 (IL-4). It remains to be seen if this differentiates tralokinumab in safety and efficacy, since evidence suggests that IL-13 may have a greater impact on skin barrier function and local immune response due to its higher expression in AD skin lesions than IL-4. Notably, in the tralokinumab monotherapy trials, a proportion of tralokinumab responders (21% to 47%) who were re-randomized to placebo maintained their IGA responses at week 52; however, it is unknown if tralokinumab has the potential to induce remission of AD. The IL-13 inhibitor lebrikizumab is in phase 3 trials for moderate to severe AD.

FDA APPROVAL TIMELINE April to June 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$43

$145

$403

$788

$1,187

The forecast is a projection of total US sales per year. 14 | MAGELLANRX.COM

ENDOCRINE

vosoritide SC Biomarin PROPOSED INDICATIONS Achondroplasia

CLINICAL OVERVIEW

Vosoritide is an analog of C-type natriuretic peptide (CNP), a positive regulator of bone growth. Vosoritide also inhibits fibroblast growth factor receptor 3 (FGFR3), which has a negative effect on bone growth. Vosoritide has a longer half-life than its endogenous form. A randomized, multinational, double-blind, placebo-controlled, 52-week clinical trial evaluated vosoritide in 121 patients 5 to 14 years of age with achondroplasia and open growth plates. The study demonstrated that vosoritide 15 mcg/kg SC once daily led to a significantly greater mean annualized growth velocity (AGV; primary endpoint) compared to placebo (adjusted mean difference, 1.57 cm/year; p<0.0001). The change in height Z-score from baseline was also greater in the vosoritide group than the placebo group (difference, +0.28; p<0.0001). After 52 weeks, 119 patients entered an ongoing, phase extension trial in which all patients receive vosoritide until their final adult height is reached. In addition, in an open-label phase 2 trial, patients who received vosoritide (n=10) achieved a statistically significant cumulative additional mean height gain over 54 months of 9 cm compared to that reported in a natural history achondroplasia dataset (n=619) that was matched for age and gender. Across the studies, vosoritide was generally well tolerated, with transient injection site reactions and hypotension as the most common TEAEs.

PLACE IN THERAPY

Achondroplasia is the most common type of short-limbed dwarfism, with a global incidence of 1 in 15,000 to 40,000 newborns. Most cases are not inherited, but rather result from new mutations (80%) in the FGFR3 gene causing abnormal formation of cartilage and bone and ultimately shorter bones; however, if both parents have achondroplasia, then there is a 50% chance that their children will be affected. Among children without achondroplasia, the average increase in height from age 4 years to puberty is 7.7 cm (2.5 in) per year. However, in those with achondroplasia, the yearly vertical growth is much lower, leading to an adult height < 137 cm (54 in). This abnormal growth leads to skeletal malformations (e.g., curvature of the spine, spinal stenosis, bowed legs) as well as challenges in performing daily activities. Surgical treatments are used to relieve pain and other manifestations of the condition. Use of growth hormone is not recommended in this population as it has the potential to worsen the skeletal disproportion seen in these patients. If approved, self- or caregiver-administered vosoritide will be the first DMT for achondroplasia to increase height in children as young as 5 years with open growth plates. If the mean increase in AGV reported in the studies, 1.57 cm, continues during each year of vosoritide use, it could result in an increase in final height by 15.7 cm (6.2 in) if used over 10 years. Vosoritide has not been associated with any safety signals. Evaluation is ongoing regarding the effects of early use (ages 0 to < 5 years) as well as long-term effects on body proportionality, growth, and medical complications of the disease (e.g., foramen magnum stenosis with brainstem compression).

FDA APPROVAL TIMELINE August 20, 2021

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$50

$123

$190

$288

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

15 | MAGELLANRX.COM

Biosimilar Overview CLINICAL OVERVIEW

Biosimilars are very different from generic drugs in that they are not exact duplicates of their reference biologic product. The FDA approval process for biosimilars is designed to ensure that the biosimilar product is highly similar to the reference product without having any meaningful clinical differences. 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. Most states have enacted biosimilar substitution laws. 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 biologic pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.

PLACE IN THERAPY

The patents of several biologic drugs are set to expire in the next few years, opening the US market for biosimilar entry; however, patent litigation has resulted in significant launch delays of FDA-approved biosimilars. In June 2017, the US Supreme Court issued 2 landmark rulings: (1) allowing a biosimilar manufacturer to provide launch notice of commercial marketing to the originator manufacturer before or after FDA approval of the biosimilar product and (2) eliminating any federal requirement for disclosure, also known as the “patent dance.” Some states, however, mandate disclosure. These decisions may bring biosimilars to the market sooner and potentially create price competition in the marketplace. In July 2018, the FDA unveiled its Biosimilar Action Plan (BAP), a series of 11 steps to encourage biosimilar market competition, some of which were previously announced or underway. The BAP contains 4 key strategies: (1) 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.

16 | MAGELLANRX.COM

To date, a total of 29 biosimilars have received FDA approval. Of these, only 20 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zarxio® (filgrastim-sndz)

Novartis/Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer/Celltrion

April 2016

Erelzi™ (etanercept-szzs)

Novartis/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)

Novartis/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

17 | MAGELLANRX.COM

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

June 2020

Hulio® (adalimumab-fkjp)

Mylan

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Commercially Available

  -



Originator Product (Manufacturer) Remicade (Janssen) Neulasta (Amgen) Humira (Abbvie) Rituxan (Genentech)

* 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. Specialty medications, which include biologics, make up approximately 36% of global medication spend. Global specialty spending is projected to reach 40% by 2024, and reach 52% in developed markets. While < 2% of Americans use biologics, they account for 26% of all national prescription drug spending. 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 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 approximately 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 FDAapproved 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. 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.

18 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

IMMUNOLOGY

adalimumab SC AVT-02 and CHS-1420 are biosimilars to Abbvie’s Humira, tumor necrosis factor alpha (TNF-α) blocker indicated for the treatment of autoimmune disorders, including rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), ankylosing spondylitis (AS), plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD) in adults and children, ulcerative colitis (UC), hidradenitis suppurativa (HS), and non-infectious uveitis.

FDA APPROVAL TIMELINE Alvotech (AVT-02) September 2021

Coherus (CHS-1420) December 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$17,369

$17,697

$9,564

$6,636

$4,983

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

ONCOLOGY

bevacizumab IV Aybintio, Bmab-100, BAT1706, and FKB238 are investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth factor (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 Centus/AstraZeneca (FKB238) Pending

Bio-Thera Solutions (BAT1706) November 27, 2021 Samsung Bioepis/Merck (Aybintio) Pending Viatris (Mylan)/Biocon (Bmab-100) Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,142

$878

$750

$652

$577

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

19 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

filgrastim IV, SC Apotex, Amneal, and Tanvex 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 in patients who are undergoing myeloablative chemotherapy followed by bone marrow transplantation; to mobilize autologous hematopoietic progenitor cells for collection by leukapheresis; with symptomatic congenital neutropenia‚ cyclic neutropenia‚ or idiopathic neutropenia; and in patients who are acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).

FDA APPROVAL TIMELINE Apotex (Grastofil) Pending Amneal Pending Tanvex May 21, 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$104

$89

$79

$70

$62

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

ENDOCRINE

insulin aspart SC Viatris (Mylan)/Biocon Viatris (Mylan)/Biocon (MYL-1601D) 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) 2021

2022

2023

2024

2025

$795

$678

$593

$546

$510

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

20 | MAGELLANRX.COM

BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

pegfilgrastim SC Lapelga, MSB-11455, and TPI-120 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) Pending Amneal (TPI-120) Pending

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,563

$1,284

$1,078

$930

$799

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

IMMUNOLOGY

ranibizumab intravitreal Samsung Bioepis/Biogen Samsung Bioepis/Biogen are seeking biosimilar approval to Genentech’s Lucentis®, a vascular endothelial growth factor (VEGF) inhibitor indicated to treat wet age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy, and myopic choroidal neovascularization (mCNV).

FDA APPROVAL TIMELINE September to October 2021

FINANCIAL FORECAST (reported in millions) 2021

2022

2023

2024

2025

$1,629

$1,541

$1,371

$1,212

$1,061

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

21 | MAGELLANRX.COM

Keep on Your RADAR Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the MRx Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2025, are displayed. The financials are projected total annual US sales, reported in millions. VIR-7831

abrocitinib

$650

$661

COVID-19

Dermatology

tisotumab vedotin

adagrasib Oncology

$959

Oncology

aducanumab

$552

Neurology

$2,043

tirzepatide Diabetes

$1,893

bardoxolone methyl Renal

$1,106

tezepelumab Respiratory

$916

deucravacitinib Immunology

sotorasib

$1,301

$1,024

efgartigimod

Oncology

Immunology

$1,105

somatrogon Endocrine

elivaldogene tavalentivec (Lenti-D)

$56 oportuzumab monatox

Neurology/Gene therapy

Oncology

$35

$55 NVX-CoV2373 vaccine COVID-19

$2,111

mavacamten Cardiovascular

$926

lenadogene nolparvovec (GS010)

Ophthalmology/Gene therapy

$55

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

Pipeline DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2022. 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 SUBMITTED APPLICATION TO THE FDA SUBMITTED

IN PHASE 3 PHASE 3 TRIALS TRIALS

63%

66%

37%

34%

36%

30%

Specialty

18%

14%

10%

Traditional

Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar

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

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

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

Submitted (New Drugs) pegunigalsidase alfa

Chiesi

Fabry’s disease

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

April 2021

budesonide oral suspension

Takeda

Eosinophilic esophagitis

Oral

Submitted – 505(b)(2) NDA; Breakthrough Therapy; Orphan Drug; Priority Review

Apr-Jun 2021

eflornithine/sulindac

Mallinckrodt

Familial adenomatous polyposis

Oral

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

Apr-Jun 2021

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Viatris (Mylan)/Biocon

T1DM; T2DM

Submitted – BLA

Apr-Jun 2021

tanezumab

Pfizer

Osteoarthritis pain

Submitted – BLA; Fast Apr-Jun 2021 Track

tralokinumab

AstraZeneca

Atopic dermatitis (moderate-severe)

Submitted – BLA

Apr-Jun 2021

inolimomab

Elsalys

GVHD (acute, steroidrefractory)

Submitted – BLA; Orphan Drug; RTOR

Apr-Jul 2021

eflapegrastim

Spectrum

Neutropenia/leukopenia

Submitted – BLA

May 2021

bupivacaine/meloxicam

Heron

Postsurgical pain

Instillation

Submitted – NDA; Breakthrough Therapy; Fast Track

05/13/2021

pegcetacoplan

Apellis

Paroxysmal nocturnal hemoglobinuria

Submitted – NDA; Fast Track; Orphan Drug; Priority Review

05/14/2021

avalglucosidase alfa

Sanofi

Pompe disease

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

05/18/2021

filgrastim (biosimilar to Amgen’s Neupogen)

Tanvex

Neutropenia/leukopenia

Submitted – BLA

05/21/2021

dehydrated alcohol

Eton

Methanol poisoning

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

05/27/2021

leuprolide mesylate readyto-use, 6-month depot

Intas

Prostate cancer

Submitted – 505(b)(2) NDA

05/27/2021

zonisamide oral suspension

Azurity

Partial seizures

Oral

Submitted – 505(b)(2) NDA

05/30/2021

20-valent pneumococcal conjugate vaccine

Pfizer

Streptococcus pneumoniae invasive disease and pneumnia prevention

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

June 2021

infigratinib

Bridgebio

Biliary tract cancer

Oral

Submitted – NDA; June 2021 Fast Track; Orphan Drug; Priority Review; RTOR

24 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ibrexafungerp

Scynexis

Vulvovaginal candidiasis

Oral

Submitted – NDA; 06/01/2021 Fast Track; Orphan Drug; Priority Review; QIDP

relugolix/estradiol/ norethindrone

Myovant

Uterine fibroids

Oral

Submitted – NDA

06/01/2021

samidorphan/olanzapine

Alkermes

Bipolar disorder; Schizophrenia

Oral

Submitted – NDA

06/01/2021

plasminogen (human)

Liminal

Congenital plasminogen deficiency

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

06/05/2021

aducanumab

Biogen

Alzheimer’s disease

Submitted – BLA; Fast 06/07/2021 Track; Priority Review

arimoclomol

Orphazyme

Niemann-Pick disease

Oral

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

06/17/2021

ruxolitinib cream

Incyte

Atopic dermatitis (mildmoderate)

Topical

Submitted – NDA; Priority Review

06/21/2021

cantharidin

Verrica

Molluscum contagiosum

Topical

Submitted – NDA

06/23/2021

lonapegsomatropin

Ascendis

Growth hormone deficiency (pediatrics)

Submitted – BLA; Orphan Drug

06/25/2021

cyclosporine

Santen

Allergic conjunctivitis

Ophthalmic

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

06/26/2021

pineapple proteolytic enzymes extract

Vericel

Burn Injury

Topical

Submitted – BLA; Orphan Drug

06/29/2021

bimekizumab

UCB

PSO

Submitted – BLA

July 2021

abrocitinib

Pfizer

Atopic dermatitis (moderate-severe)

Oral

Submitted – NDA; Breakthrough Therapy; Priority Review

Jul-Aug 2021

atogepant

Allergan

Migraine prevention

Oral

Submitted – NDA

Jul-Sep 2021

anifrolumab

AstraZeneca/BristolMyers Squibb

SLE

Submitted – BLA; Fast Jul-Dec 2021 Track

roxadustat

AstraZeneca

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

Oral

Submitted – NDA

Jul-Dec 2021

teplizumab

Provention Bio

T1DM (delay/prevention)

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

07/02/2021

avacopan

Chemocentryx

ANCA-associated vasculitis

Oral

Submitted – NDA; Orphan Drug

07/07/2021

brincidofovir

Chimerix

Smallpox

Oral

Submitted – NDA; Fast Track; Orphan Drug; Priority Review

07/07/2021

finerenone

Bayer

Diabetic nephropathy

Oral

Submitted – NDA; Priority Review

07/09/2021

25 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

15-valent pneumococcal conjugate vaccine

Merck

Invasive pneumococcal disease prevention

Submitted – BLA; Breakthrough Therapy; Priority Review

07/16/2021

narsoplimab

Omeros

HSCT-associated thrombotic microangiopathy

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

07/16/2021

odevixibat

Albireo

Progressive familial intrahepatic cholestasisrelated pruritus

Oral

Submitted – NDA; 07/20/2021 Fast Track; Orphan Drug; Priority Review; Rare Pediatric Disease

retifanlimab

Incyte

Anal cancer (squamous cell, locally advanced/ metastatic, failed on/ intolerant to platinumbased chemotherapy)

Submitted – BLA; Orphan Drug; Priority Review

07/23/2021

sulopenem etzadroxil/ probenecid

Iterum

Uncomplicated UTI (quinolone-resistant)

Oral

Submitted – NDA; Fast Track; Priority Review; QIDP

07/23/2021

tick-borne encephalitis vaccine

Pfizer

Tick-borne encephalitis

Submitted – BLA; Priority Review; Rare Pediatric Disease

August 2021

treosulfan

Medac

Allogenic-HSCT conditioning

IV, Oral

Submitted – NDA; Orphan Drug

August 2021

tretinoin/benzoyl peroxide

Sol-Gel

Acne vulgaris

Topical

Submitted – 505(b)(2) NDA

08/01/2021

topiramate oral solution

Azurity

Partial seizures

Oral

Submitted – 505(b)(2) NDA

08/06/2021

sotorasib

Amgen

NSCLC (KRAS G12C mutation, locally advanced/metastatic, ≥ 1 prior systemic therapy)

Oral

Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review; RTOR

08/16/2021

oportuzumab monatox

Sesen

Bladder cancer (BCGunresponsive, nonmuscle invasive)

Intravesical

Submitted – BLA; Fast 08/18/2021 Track; Priority Review

vosoritide

Biomarin

Achondroplasia

Submitted – NDA; Orphan Drug; Priority Review

08/20/2021

dextromethorphan/ bupropion

Axsome

MDD

Oral

Submitted – 505(b)(2) NDA; Breakthrough Therapy; Fast Track; Priority Review

08/22/2021

difelikefalin

Cara

Chronic kidney diseaseassociated pruritus

Submitted – NDA; Breakthrough Therapy; Priority Review

08/23/2021

belumosudil

Kadmon

GVHD (chronic)

Oral

Submitted – NDA; 08/30/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Project Orbis; RTOR

26 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

adalimumab (biosimilar to Abbvie’s Humira)

Alvotech

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

Submitted – BLA

September 2021

ranibizumab (biosimilar to Genentech’s Lucentis)

Samsung Bioepis/Biogen

Wet AMD

Intravitreal

Submitted – BLA

Sep-Oct 2021

paliperidone palmitate 6-month injectable

Janssen

Schizophrenia

Submitted – NDA

09/02/2021

dihydroergotamine mesylate

Impel Neuropharma

Migraine treatment

Intranasal

Submitted – 505(b)(2) NDA

09/06/2021

belzutifan

Merck

RCC

Oral

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

09/15/2021

risperidone long-acting in situ microparticle

Rovi

Schizophrenia

Submitted – 505(b)(2) NDA

09/24/2021

udenafil

Dong-A Socio

Single ventricle heart disease (post Fontan operation)

Oral

Submitted – NDA; Orphan Drug

09/29/2021

reltecimod

Atox Bio

Necrotizing soft tissue infection (NSTI)-related organ dysfunction/failure (ages ≥ 12 years)

Submitted – NDA; seeking Accelerated Approval; Fast Track; Orphan Drug

09/30/2021

somatrogon

Pfizer/Opko

Growth hormone deficiency (pediatrics)

Submitted – BLA; Orphan Drug

October 2021

amivantamab

Janssen

NSCLC (EGFR exon 20 insertion mutations, progressed on or after platinum-based chemotherapy)

Submitted – BLA; Breakthrough Therapy

Oct-Dec 2021

maralixibat

Mirum

Alagille syndrome

Oral

Submitted – NDA; 10/01/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

tisotumab vedotin

Seagen

Cervical cancer (recurrent, IV metastatic)

Submitted – BLA; seeking Accelerated Approval; Priority Review

10/08/2021

sodium oxybate

Avadel

Narcolepsy-related excessive daytime sleepiness and cataplexy

Oral

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

10/15/2021

varenicline

Oyster Point

Dry eye syndrome

Intranasal

Submitted – 505(b)(2) NDA

10/18/2021

phenylephrine/ tropicamide

Eyenovia

Pharmacologic mydriasis

Ophthalmic

Submitted – 505(b)(2) NDA

10/29/2021

testosterone undecanoate

Marius

Hypogonadism

Oral

Submitted – 505(b)(2) NDA

10/29/2021

ciltacabtagene autoleucel

Janssen

Multiple myeloma (relapsed/refractory)

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

November 2021

dexmedetomidine

Bioxcel

Bipolar disorder- & schizophrenia-related agitation

Submitted – NDA; Fast Track

11/11/2021

27 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

omidenepag isopropyl

Santen

Glaucoma/ocular hypertension

Ophthalmic

Submitted – NDA

11/19/2021

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera

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

Submitted – BLA

11/27/2021

pacritinib

CTI

Myelofibrosis

Oral

Submitted – NDA; Fast Track; Orphan Drug

11/30/2021

trivalent hepatitis B vaccine

VBI Vaccines

Hepatitis B virus prevention

Submitted – BLA

11/30/2021

adalimumab (biosimilar to Abbvie’s Humira)

Coherus

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

Submitted – BLA

December 2021

HIV vaccine

Immune Response

HIV-1 infection

Submitted – BLA; Orphan Drug

December 2021

treprostinil DPI

United Therapeutics

PAH; Pulmonary hypertension-related intersitial lung disease

Inhaled

Submitted – NDA

Dec 2021 Apr 2022

efgartigimod

Argenx

Myasthenia gravis

Submitted – BLA; Fast 12/17/2021 Track; Orphan Drug

gefapixant

Merck

Chronic cough

Oral

Submitted – NDA

12/21/2021

dextroamphetamine

Hisamitsu

ADHD

Transdermal

Submitted – NDA

12/22/2021

pilocarpine 1.25%

Allergan

Presbyopia

Ophthalmic

Submitted – NDA

12/24/2021

levoketoconazole

Strongbridge

Cushing’s syndrome

Oral

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

12/31/2021

ublituximab + umbralisib

CLL

IV + Oral

Submitted – BLA; Fast Jan-Mar 2022 Track; Orphan Drug

daridorexant

Idorsia

Insomnia

Oral

Submitted – NDA

01/07/2022

budesonide (long-acting)

Calliditas

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

Oral

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

01/14/2022

mavacamten

Bristol-Myers Squibb

Obstructive hypertrophic cardiomyopathy

Oral

Submitted – NDA; Breakthrough Therapy; Orphan Drug

01/28/2022

balstilimab

Agenus

Cervical cancer (recurrent or metastatic)

Submitted – BLA; seeking Accelerated Approval; Fast Track

02/19/2022

immune globulin IV

Primary humoral immunodeficiency

Submitted – BLA

02/25/2022

vadadustat

Akebia

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

Oral

Submitted – NDA

03/30/2022

benegrastim

Evive

Neutropenia/leukopenia

Submitted – BLA

03/31/2022

plinabulin

Beyondspring

Neutropenia/leukopenia

Submitted – NDA; Breakthrough Therapy

03/31/2022

vutrisiran

Alnylam

Transthyretin amyloid polyneuropathy

Submitted – NDA; Fast Track; Orphan Drug

Apr-Jun 2022

28 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

bevacizumab (biosimilar to Genentech’s Avastin)

Centus/AstraZeneca

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Samsung Bioepis/Merck

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Viatris (Mylan)/Biocon

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

Submitted – BLA

Pending

donislecel

Celltrans

T1DM

Submitted – BLA; Orphan Drug

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Amneal

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Amneal

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Merck/Fresenius

Neutropenia/leukopenia

Submitted – BLA

Pending

tramadol

Fortress

Postsurgical pain

Submitted – NDA

Pending

Submitted (Supplementals) dapagliflozin (Farxiga )

AstraZeneca

CKD (with or without T2DM)

Oral

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

Apr-Jun 2021

eptacog beta (Sevenfact®)

Hema Biologics

Hemophilia A and B (prevention of bleeding related to surgery or invasive procedure)

Submitted – sBLA

Apr-Sep 2021

tenapanor (Ibsrela®)

Ardelyx

Hyperphosphatemia (dialysis-dependent patients)

Oral

Submitted – sNDA

04/29/2021

pirfenidone (Esbriet®)

Genentech

Idiopathic pulmonary fibrosis (unclassified)

Oral

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

May 2021

rimegepant (Nurtec™ ODT)

Biohaven

Migraine prevention

Oral

Submitted – sNDA

May-Jun 2021

zoster vaccine recombinant, adjuvanted (Shingrix)

GlaxoSmithKline

Herpes zoster prevention (ages ≥ 18 years at increased risk)

IM, SC

Submitted – sBLA

May-Jun 2021

teriflunomide (Aubagio®)

Sanofi

MS (pediatrics, relapsing)

Oral

Submitted – sNDA; Priority Review

05/02/2021

nivolumab (Opdivo®)

Bristol-Myers Squibb

Esophageal cancer

Submitted – sBLA; Orphan Drug; Priority Review

05/20/2021

29 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

nivolumab (Opdivo)

Bristol-Myers Squibb

Gastric/gastroesophageal junction cancer (advanced/metastatic, in combination with fluoropyrimidine- and platinum-containing chemotherapy)

Submitted – sBLA; Priority Review

05/25/2021

ozanimod (Zeposia®)

Bristol-Myers Squibb

Oral

Submitted – sNDA; Priority Review

05/30/2021

omadacycline (Nuzyra®)

Paratek

CAP

IV, Oral

Submitted – sNDA; Fast Track; QIDP

05/31/2021

semaglutide (Ozempic®) 2.4 mg

Novo Nordisk

Obesity/overweight (≥ 1 weight-related comorbidity)

Submitted – sNDA; Priority Review

06/04/2021

elexacaftor/tezacaftor/ ivacaftor (Trikafta®)

Vertex

CF (F508del mutation in CFTR gene or responsive CFTR gene mutation, ages 6-11 years)

Oral

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

06/08/2021

avapritinib (Ayvakit)

Blueprint Medicines

Mastocytosis (systemic)

Oral

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

06/16/2021

ruxolitinib (Jakafi)

Incyte

GVHD (chronic, steroidrefractory, ages ≥ 12 years)

Oral

Submitted – sNDA; Orphan Drug; Priority Review

06/22/2021

secukinumab (Cosentyx®)

Novartis

PSO (pediatrics)

Submitted – sBLA

06/25/2021

upadacitinib (Rinvoq)

Abbvie

Ankylosing spondylitis

Oral

Submitted – sNDA

06/25/2021

secnidazole (Solosec )

Lupin

Trichomoniasis infections

Oral

Submitted – sNDA

06/30/2021

levonorgestrel 52 mg intrauterine device (Mirena®)

Bayer

Contraception

Intrauterine

Submitted – sNDA

July 2021

baricitinib (Olumiant)

Eli Lilly

Atopic dermatitis (moderate-severe)

Oral

Submitted – sNDA

Jul-Aug 2021

mepolizumab (Nucala®)

GlaxoSmithKline

Nasal polypsis

Submitted – sBLA

Jul-Aug 2021

tofacitinib (Xeljanz / Xeljanz XR®)

Pfizer

Ankylosing spondylitis

Oral

Submitted – sNDA

Jul-Aug 2021

upadacitinib (Rinvoq)

Abbvie

Atopic dermatitis (moderate-severe, ages ≥ 12 years)

Oral

Submitted – sNDA; Breakthrough Therapy

Jul-Aug 2021

upadacitinib (Rinvoq)

Abbvie

PsA

Oral

Submitted – sNDA

07/01/2021

selexipag (Uptravi )

Janssen

PAH (as temporary alternative to oral formulation)

Submitted – sNDA; Orphan Drug

07/30/2021

sodium oxybate (Xywav™)

Jazz

Idiopathic hypersomnia

Oral

Submitted – sNDA; Fast Track; Orphan Drug; Priority Review

08/12/2021

enfortumab vedotin-ejfv (Padcev™)

Astellas

Bladder cancer (locally advanced/metastatic, after PD-1/PD-L1 inhibitor therapy, cisplatin ineligible)

Submitted – sBLA; Priority Review; Project Orbis; RTOR

08/14/2021

30 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

rivaroxaban (Xarelto®)

Janssen

Peripheral arterial disease (post recent lower-extremity revascularization)

Oral

Submitted – sNDA

08/26/2021

empagliflozin (Jardiance®)

Boehringer Ingelheim

Chronic heart failure (with Oral reduced ejection fraction, with or without T2DM)

Submitted – sNDA; Fast Track

September 2021

ivosidenib (Tibsovo®)

Les Laboratoires Servier

Biliary tract cancer (previously treated, isocitrate dehydrogenase 1 [IDH1] mutated)

Oral

Submitted – sNDA; Fast Track; Orphan Drug

Sep-Dec 2021

pembrolizumab (Keytruda®)

Merck

Squamous cell carcinoma (locally advanced)

Submitted – sBLA

09/09/2021

daratumumab/ hyaluronidase-fihj (Darzalex Faspro™)

Janssen

Multiple myeloma (relapsed/refractory, in combination with pomalidomide and dexamethasone)

Submitted – sBLA

09/10/2021

andexanet alfa (Andexxa®)

Portola

Acute intracranial IV hemorrhage while taking an oral Factor Xa inhibitor, including edoxaban and enoxaparin

Submitted – sBLA; Breakthrough Therapy; Orphan Drug

October 2021

dexamethasone insert (Dextenza®)

Ocular Therapeutix

Allergic conjunctivitis

Intraocular

Submitted – sNDA

October 2021

abemaciclib (Verzenio®)

Eli Lilly

Breast cancer (high risk HR+, HER2-, early disease)

Oral

Submitted – sNDA

Oct-Dec 2021

zanubrutinib (Brukinsa®)

Beigene

Waldenstrom macroglobulinemia

Oral

Submitted – sNDA; Fast Track; Orphan Drug; Priority Review

10/18/2021

dupilumab (Dupixent)

Sanofi

Asthma (moderate-severe, SC adjunct, ages 6-11 years)

Submitted – sBLA

10/21/2021

Amgen

PSO (mild-moderate)

Oral

Submitted – sNDA

12/22/2021

lumateperone (Caplyta )

Intra-Cellular Therapies

Bipolar disorder

Oral

Submitted – sNDA

12/22/2021

cabotegravir/rilpivirine (Cabenuva)

Viiv

HIV-1 infection (treatment, every 2-month dosing)

Submitted – sNDA

12/24/2021

brexucabtagene autoleucel Gilead (Tecartus™)

ALL

Submitted – sBLA; Breakthrough Therapy; Orphan Drug

Jan-Mar 2022

risankizumab-rzaa (Skyrizi®)

Abbvie

PsA

Submitted – sBLA

February 2022

axicabtagene ciloleucel (Yescarta®)

Gilead

Marginal zone lymphoma (after ≥ 2 prior lines of systemic therapy)

Submitted – sBLA; Breakthrough Therapy; Priority Review

Pending

infliximab-dyyb (Inflectra)

Celltrion

IBS

Submitted – sBLA

Pending

apremilast (Otezla®) ®

Phase 3 (New Drugs) abaloparatide-TD

Radius Health

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

acoramidis

Bridgebio

Transthyretin amyloid cardiomyopathy; Transthyretin amyloid polyneuropathy

Oral

Phase 3 – NDA; Orphan Drug

TBD

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

adagrasib

Mirati

NSCLC

Oral

Phase 3 – NDA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Fresenius

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

Phase 3 – BLA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Viatris (Mylan)/Momenta

Hidradenitis suppurativa; Uveitis

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea®)

Samsung Bioepis/Biogen

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Santo/Formycon

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

amcenestrant

Sanofi

Breast cancer

Oral

Phase 3 – NDA

TBD

anthrax vaccine, adsorbed

Emergent

Anthrax infection

Phase 3 – BLA; Fast Track

TBD

apolipoprotein A-I (human)

CSL

Atherosclerosis

Phase 3 – BLA

TBD

arfolitixorin hemisulfate

Isofol

CRC

Phase 3 – NDA

TBD

asciminib

Novartis

Chronic myelogenous leukemia

Oral

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

TBD

ataluren

PTC

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

autologous genetically modified human dermal fibroblasts

Castle Creek

Epidermolysis bullosa

Intradermal

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

TBD

AZD7442

AstraZeneca

COVID-19

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

Phase 3 – NDA; Fast Track

TBD

bamlanivimab + etsevimab

Eli Lilly

COVID-19

Phase 3 – BLA

TBD

bardoxolone methyl

Reata

Alport syndrome; Polycystic kidney disease

Oral

Phase 3 – NDA; Orphan Drug

TBD

bentracimab

Phasebio

Ticagrelor reversal

Phase 3 – BLA; Breakthrough Therapy

TBD

beremagene geperpavec

Krystal

Epidermolysis bullosa

Topical

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

TBD

beroctocog alfa

Hemophilia A

Phase 3 – BLA

TBD

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

Sickle cell disease; Thalassemia

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

TBD

bevacizumab-vikg

Outlook

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

bexagliflozin

Theracos

T2DM

Oral

Phase 3 – NDA

TBD

bimekizumab

UCB

AS; Hidradenitis suppurativa

Phase 3 – BLA

TBD

bintrafusp alfa

Merck

Biliary tract cancer; NSCLC

Phase 3 – BLA; Orphan Drug

TBD

brensocatib

Insmed

Bronchiectasis

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

budesonide/albuterol

AstraZeneca

Asthma

Inhaled

Phase 3 – NDA

TBD

32 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

bulevirtide

Gilead

Hepatitis D infection

Phase 3 – NDA; Breakthrough Therapy

TBD

C19VAZ vaccine (formerly AZD-1222; ChAdOx1)

AstraZeneca

COVID-19

Phase 3 – BLA

TBD

cannabidiol gel

Zynerba

Fragile X syndrome

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

capsaicin

Centrexion

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – 505(b)(2) NDA; Fast Track

TBD

casirivimab/imdevimab

Regeneron

COVID-19

IM, IV, SC

Phase 3 – BLA

TBD

CD24Fc

OncoImmune

COVID-19

Phase 3 – BLA

TBD

ceftobiprole medocaril

Basilea

ABSSSI; CAP; HAP

Phase 3 – NDA; Fast Track; QIDP

TBD

ceftriaxone wearable micropump

scPharmaceuticals

Gram+/gram- infection

Phase 3 – NDA

TBD

cipaglucosidase alfa

Amicus

Pompe disease

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

clindamycin phosphate gel

Daré

Bacterial vaginosis

Intravaginal

Phase 3 – NDA; Fast Track; QIDP

TBD

CM-AT (pancreatic enzyme)

Curemark

Autism spectrum disorders

Oral

Phase 3 – BLA; Fast Track

TBD

concizumab

Novo Nordisk

Hemophilia A and B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

crovalimab

Genentech

Paroxysmal nocturnal hemoglobinuria

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

cyclosporine A

Novaliq

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

dactolisib

Restorbio

COVID-19

Oral

Phase 3 – NDA

TBD

dalcetrapib

Dalcor

Acute coronary syndrome (ADCY9 AA genotype)

Oral

Phase 3 – NDA

TBD

daprodustat

GlaxoSmithKline

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

Oral

Phase 3 – NDA

TBD

darvadstrocel

Takeda

Phase 3 – BLA; Orphan Drug

TBD

dehydrated human Mimedx amnion-chorion membrane

Achilles tendonitis; Plantar fasciitis

Phase 3 – BLA

TBD

dengue tetravalent vaccine

Takeda

Dengue fever

Phase 3 – BLA; Fast Track

TBD

denosumab (biosimilar to Amgen’s Prolia®)

Novartis

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

dersimelagon

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

despropyl macitentan

Janssen

Hypertension

Oral

Phase 3 – NDA

TBD

deucravacitinib

Bristol-Myers Squibb

PSO

Oral

Phase 3 – NDA

TBD

diazoxide choline

Soleno

Prader-Willi syndrome

Oral

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

TBD

33 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

difluprednate

Sun Pharma Advanced Research

Ocular pain/inflammation

Ophthalmic

Phase 3 – NDA

TBD

dihydroergotamine

Satsuma

Migraine treatment

Intranasal

Phase 3 – NDA

TBD

dociparstat

Chimerix

COVID-19

Phase 3 – NDA

TBD

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers

Phase 3 – BLA

TBD

doravirine/islatravir

Merck

HIV-1 infection

Oral

Phase 3 – NDA

TBD

dovitinib lactate

Allarity

Breast cancer; RCC

Oral

Phase 3 – NDA

TBD

dust mite immunotherapy

Stallergenes Greer

Allergic rhinitis

Phase 3 – BLA

TBD

EB-101 (gene therapy)

Abeona

Epidermolysis bullosa

Surgical application

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug R; RMAT

TBD

eculizumab (biosimilar to Alexion’s Soliris®)

Amgen

Paroxysmal nocturnal hemoglobinuria

Phase 3 – BLA

TBD

efanesoctocog alfa

Sanofi

Hemophilia A

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

efgartigimod

Argenx

ITP; Myasthenia gravis; Pemphigus vulgaris

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

elamipretide

Stealth

Barth syndrome

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

elivaldogene autotemcel (Lenti-D)

Bluebird Bio

Adrenoleukodystrophy

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

Phase 3 – NDA; Fast Track; QIDP

TBD

ensifentrine

Verona

COPD

Inhaled

Phase 3 – NDA

TBD

EP-2101 therapeutic vaccine

OSE Immunotherapeutics

NSCLC

Phase 3 – NDA; Orphan Drug

TBD

epinephrine

Bryn

Anaphylaxis

Intranasal

Phase 3 – NDA; Fast Track

TBD

episalvan

Amryt

Epidermolysis bullosa

Topical

Phase 3 – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

etanercept (biosimilar to Amgen’s Enbrel)

Coherus

RA; Polyarticular JIA; AS; PSO; PsA

Phase 3 – BLA

TBD

etesevimab

Eli Lilly

COVID-19

Phase 3 – BLA

TBD

etranacogene dezaparvovec

Uniqure

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

etrasimod

Arena

CD; UC

Oral

Phase 3 – NDA

TBD

etrolizumab

Genentech

Phase 3 – BLA

TBD

faricimab

Genentech

Diabetic macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

fasinumab

Regeneron

Osteoarthritis pain

Phase 3 – BLA

TBD

favipiravir

Dr. Reddy’s

COVID-19; Influenza

Oral

Phase 3 – NDA

TBD

fexapotide triflutate

Nymox

Benign prostatic hyperplasia

Intratumoral

Phase 3 – NDA

TBD

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Phase 3 – NDA

TBD

fidanacogene elaparvovec

Pfizer

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

CD; UC

Oral

Phase 3 – NDA

TBD

firmacute eubacterial spores

Seres

Clostridium difficileassociated diarrhea

Oral

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

fitusiran

Sanofi

Hemophilia A and B

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

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

Allergan

Female reproductive disorder

Phase 3 – BLA

TBD

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

Finox

Female reproductive disorder

Phase 3 – BLA

TBD

FT-4202

Forma

Sickle cell disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ganaxolone

Marinus

Status epilepticus; CDKL5 deficiency disorderrelated seizures

IV, Oral

Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease

TBD

gantenerumab

Genentech

Alzheimer’s disease

Phase 3 – BLA

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3 – NDA; QIDP

TBD

giroctocogene fitelparvovec

Pfizer

Hemophilia A

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

TBD

givinostat

Italfarmaco

DMD

Oral

Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease

TBD

glatiramer acetate depot

Viatris (Mylan)

Phase 3 – NDA

TBD

human pentraxin-2 protein

Promedior

Idiopathic pulmonary fibrosis

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

human plasminogen

Kedrion

Ligneous conjunctivitis

Topical

Phase 3 – BLA; Orphan Drug

TBD

idursulfase

Takeda

Mucopolysaccharidosis II Intrathecal (MPS II; Hunter syndrome)

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; PsA; CD; UC

Phase 3 – BLA

TBD

ingenol disoxate

Leo

Actinic keratoses

Topical

Phase 3 – NDA

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Sanofi

T1DM; T2DM

Phase 3 – BLA

TBD

insulin glargine (biosimilar to Sanofi’s Lantus)

Gan & Lee

T1DM; T2DM

Phase 3 – BLA

TBD

insulin icodec (once weekly)

Novo Nordisk

T2DM

Phase 3 – BLA

TBD

iodine-131 apamistamab

Actinium

AML

Phase 3 – BLA; Orphan Drug

TBD

35 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ipatasertib

Genentech

Breast cancer (TNBC/HR+, 1st-line, in combination with chemotherapy); Prostate cancer

Oral

Phase 3 – NDA

TBD

Johnson & Johnson COVID-19 (Ad26.COV2-S) vaccine

Janssen

COVID-19

Phase 3 – BLA

TBD

KSI-301

Kodiak

Diabetic macular edema; Retinal vein occlusionassociated macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3 – BLA; Orphan Drug

TBD

L-asparaginase

Erytech

Pancreatic cancer

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

lazertinib

Genosco

NSCLC

Oral

Phase 3 – NDA

TBD

lebrikizumab

Eli Lilly

Atopic dermatitis (moderate-severe)

Phase 3 – BLA; Fast Track

TBD

lecanemab

Eisai

Alzheimer’s disease

Phase 3 – BLA

TBD

lenacapavir

Viiv

HIV-1 infection (heavily treatment-experienced)

Phase 3 – NDA; Breakthrough Therapy

TBD

lenadogene nolparvovec (GS010)

Gensight

Leber’s hereditary optic neuropathy

Intravitreal

Phase 3 – BLA; Orphan Drug

TBD

leniolisib

Pharming

Activated Oral phosphoinositide 3-kinase delta syndrome/ p110δ-activating mutation causing senescent T cells, lymphadenopathy, & immunodeficiency (APDS/ PASLI)

Phase 3 – NDA; Orphan Drug

TBD

lenzilumab

Humanigen

COVID-19

Phase 3 – BLA

TBD

leriglitazone

Minoryx

Adrenoleukodystrophy

Oral

Phase 3 – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

leronlimab

Cytodyn

COVID-19; HIV-1 infection treatment (in combination therapy with HAART, highly treatmentexperienced);

Phase 3 – BLA; Fast Track

TBD

lidocaine/prilocaine

Plethora Solutions

Premature ejaculation

Topical

Phase 3 – NDA

TBD

ligelizumab

Novartis

Urticaria

Phase 3 – BLA; Breakthrough Therapy

TBD

linzagolix

Obseva

Endometriosis; Uterine fibroids

Oral

Phase 3 – NDA

TBD

lorecivivint

Samumed

Osteoarthritis (knee)

Intraarticular

Phase 3 – NDA

TBD

lutetium 177Lu-PSMA-617

Novartis

Prostate cancer

Phase 3 – NDA

TBD

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

magrolimab

Forty Seven

Myelodysplastic syndrome

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

TBD

maribavir

Takeda

Cytomegalovirus infection treatment

Oral

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

TBD

marstacimab

Pfizer

Hemophilia A and B

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

marzeptacog alfa

Catalyst

Hemophilia A and B

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

masitinib

AB Science

Asthma (eosinophilic); Mastocytosis; MS

Oral

Phase 3 – NDA; Orphan Drug

TBD

melphalan

Delcath

Uveal melanoma (hepatic-dominant)

Percutaneous hepatic perfusion

Phase 3 – NDA

TBD

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

Phase 3 – BLA; Orphan Drug; Rare Pediatric Disease; RMAT

TBD

microbiota suspension

Ferring

C. difficile infection (recurrent)

Rectal

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

TBD

minocycline/edetate/ethyl alcohol

Citius

Catheter-related bloodstream infection (CRBSI)

Phase 3 – NDA; Fast Track; QIDP

TBD

mirikizumab

Eli Lilly

CD; PSO; UC

IV, SC

Phase 3 – BLA

TBD

mirvetuximab soravtansine

Immunogen

Ovarian cancer

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

Moderna COVID-19 (mRNA- Moderna 1273) vaccine

COVID-19

Phase 3 – BLA; Fast Track

TBD

momelotinib

Sierra Oncology

Myelofibrosis

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

motixafortide

Biolinerx

Stem cell mobilization

Phase 3 – NDA; Orphan Drug

TBD

nabiximols

MS-related spasticity

Oral transmucosal

Phase 3 – NDA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 – NDA

TBD

naloxone hydrochloride dihydrate

Elorac

Pruritus

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

narsoplimab

Omeros

Hemolytic uremic syndrome

IV, SC

Phase 3 – BLA; Fast Track

TBD

natalizumab (biosimilar to Biogen’s Tysabri®)

Novartis

Phase 3 – BLA

TBD

37 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

nedosiran

Dicerna

Hyperoxaluria

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug; Rare Pediatric Disease

TBD

nemolizumab

Galderma

Atopic dermatitis (moderate-severe); Pruritus

Phase 3 – BLA; Breakthrough Therapy

TBD

nipocalimab

Janssen

Autoimmune hemolytic anemia

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

nirsevimab

AstraZeneca

RSV prevention

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

nomacopan

Akari

HSCT-TMA; Hemolytic uremic syndrome; Paroxysmal nocturnal hemoglobinuria

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

NVX-CoV2373 vaccine

Novavax

COVID-19

Phase 3 – BLA; Fast Track

TBD

odevixibat

Albireo

Alagille syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

olipudase alfa

Sanofi

Niemann-Pick disease

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

ondansetron ER once-daily

Redhill

Chemotherapy-induced Oral nausea & vomiting (CINV); Gastroenteritis

Phase 3 – 505(b)(2) NDA

TBD

OPT-302

Opthea

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

oteseconazole

Mycovia

Vulvovaginal candidiasis

Oral

Phase 3 – NDA; Fast Track; QIDP

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; Rare Pediatric Disease

TBD

pacritinib

CTI

COVID-19

Oral

Phase 3 – NDA

TBD

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

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

TBD

pamrevlumab

Fibrogen

COVID-19; DMD; Idiopathic pulmonary fibrosis

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

pegylated liposomal irinotecan

Ipsen

SCLC

Phase 3 – NDA; Fast Track

TBD

perfluorohexyloctane

Bausch Health

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

pevonedistat

Takeda

Myelodysplastic syndrome

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

plinabulin

Beyondspring

NSCLC

Phase 3 – NDA

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

Phase 3 – BLA

TBD

38 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

pollinex quattro ragweed

Allergy Therapeutics

Allergic rhinitis

Phase 3 – BLA

TBD

potassium citrate/ potassium bicarbonate

Advicenne

Renal tubular acidosis

Oral

Phase 3 – NDA

TBD

pozelimab

Regeneron

Paroxysmal nocturnal hemoglobinuria; Chaple disease

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Coherus

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Stada Arzneimittel/ Bausch Health

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

ranibizumab intravitreal implant

Genentech

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

rapamycin

Timber

Tuberous sclerosis complex-associated facial angiofibromas

Topical

Phase 3 – NDA

TBD

rapamycin (high-strength)

Palvella

Pachyonychia congenita

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

refanalin

Angion

Delayed graft function

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

relacorilant

Corcept

Cushing’s syndrome; Pancreatic cancer

Oral

Phase 3 – NDA; Orphan Drug

TBD

relugolix/estradiol/ norethindrone

Myovant

Endometriosis

Oral

Phase 3 – NDA

TBD

reproxalap

Aldeyra

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

rilzabrutinib

Principia

Pemphigus vulgaris; ITP

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ritlecitinib

Pfizer

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

rituximab (biosimilar to Genentech’s Rituxan)

Amgen

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

Phase 3 – BLA

TBD

rituximab (biosimilar to Genentech’s Rituxan)

Archigen

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

Phase 3 – BLA

TBD

rivipansel

Glycomimetics

Sickle cell disease

Phase 3 – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

roflumilast cream

Arcutis

Atopic dermatitis; PSO

Topical

Phase 3 – NDA

TBD

rogaratinib

Bayer

Bladder cancer

Oral

Phase 3 – NDA

TBD

ropeginterferon alfa-2b

Pharmaessentia

Essential thrombocythemia

Phase 3 – BLA

TBD

roxadustat

AstraZeneca

Anemia due to cytotoxic chemotherapy

Oral

Phase 3 – NDA

TBD

rozanolixizumab

UCB

Myasthenia gravis

Phase 3 – BLA; Orphan Drug

TBD

RSV nanoparticle vaccine

Novavax

RSV prevention

Phase 3 – BLA; Fast Track

TBD

39 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ruxolitinib (deuterated)

Concert

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

ruxolitinib cream

Incyte

Vitiligo

Topical

Phase 3 – NDA

TBD

sabatolimab

Novartis

Myelodysplastic syndrome

Phase 3 – BLA

TBD

seladelpar

Cymabay

Primary biliary cholangitis Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

seltorexant

Janssen

MDD

Oral

Phase 3 – NDA

TBD

sepofarsen

Proqr

Leber’s congenital amaurosis

Intravitreal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

sofpironium

Brickell

Axillary hyperhidrosis

Topical

Phase 3 – NDA

TBD

sotagliflozin

Lexicon

Heart failure in patients with T2DM

Oral

Phase 3 – NDA

TBD

sotatercept

Acceleron

PAH

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

sparsentan

Travere

Focal segmental glomerulosclerosis; Immunoglobulin A (IgA) nephropathy (Berger’s disease)

Oral

Phase 3 – NDA; Orphan Drug

TBD

SPK-8011

Spark

Hemophilia A

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

tapinarof

Roivant

PSO

Topical

Phase 3 – NDA

TBD

tebentafusp

Immunocore

Uveal melanoma

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

TBD

tebipenem pivoxil

Spero

UTI (complicated); Acute pyelonephritis

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 – NDA

TBD

tetrathiomolybdate

Alexion

Wilson’s disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

tezepelumab

Amgen

Asthma (severe, uncontrolled)

Phase 3 – BLA; Breakthrough Therapy

TBD

timbetasin

Regentree

Dry eye syndrome

Ophthalmic

Phase 3 – BLA

TBD

tiragolumab

Genentech

SCLC

Phase 3 – BLA

TBD

tirzepatide

Eli Lilly

T2DM

Phase 3 – NDA

TBD

tislelizumab

Beigene

HCC

Phase 3 – BLA

TBD

tocilizumab (biosimilar to Genentech’s Actemra®)

Bio-Thera

Phase 3 – BLA

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

40 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

toripalimab

Coherus

SCCHN

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

tozinameran (Pfizer/ Biontech COVID-19 vaccine; BNT162b2) vaccine

Biontech

COVID-19

Phase 3 – BLA; Fast Track

TBD

tradipitant

Vanda

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

Oral

Phase 3 – NDA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Novartis

Breast cancer; Gastric/ gastroesophageal cancer

Phase 3 – BLA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Tanvex

Breast cancer; Gastric/ gastroesophageal cancer

Phase 3 – BLA

TBD

travoprost implant

Glaukos

Glaucoma/ocular hypertension

Intraocular

Phase 3 – NDA

TBD

trehalose

Seelos

Oculopharyngeal muscular dystrophy

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

trofinetide

Acadia

Rett syndrome

Oral

Phase 3 – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease

TBD

tusamitamab ravtansine

Sanofi

NSCLC

Phase 3 – BLA

TBD

ublituximab

MS; CLL/SLL

Phase 3 – BLA; Orphan Drug

TBD

ustekinumab (biosimilar to Janssen’s Stelara®)

Amgen

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO

IV, SC

Phase 3 – BLA

TBD

valoctocogene roxaparvovec

Biomarin

Hemophilia A

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

TBD

venglustat

Sanofi

Polycystic kidney disease; Oral GM2 gangliosidoses (TaySachs disease, Sandhoff disease, AB variant)

Phase 3 – NDA; Orphan Drug

TBD

verbrinacogene setparvovec

Freeline

Hemophilia B

Phase 3 – BLA; RMAT

TBD

veverimer

Tricida

CKD-related metabolic acidosis

Oral

Phase 3 – NDA

TBD

VGX-3100 therapeutic vaccine

Inovio

Cervical dysplasia

Phase 3 – BLA

TBD

VIR-7831

Vir

COVID-19

Phase 3 – BLA

TBD

visomitin

Mitotech

Dry eye syndrome

Ophthalmic

Phase 3 – NDA

TBD

volanesorsen

Akcea

Familial chylomicronemia syndrome

Phase 3 – NDA; Orphan Drug

TBD

vonoprazan

Phathom

Esophagitis; H. pylori infection

Oral

Phase 3 – NDA; QIDP

TBD

wilfactin

LFB Group

Von Willebrand disease

Phase 3 – BLA; Orphan Drug

TBD

41 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

zavegepant

Biohaven

COVID-19; Migraine treatment

Intranasal

Phase 3 – NDA

TBD

zilucoplan

UCB

Myasthenia gravis

Phase 3 – NDA; Orphan Drug

TBD

zolbetuximab

Astellas

Gastric cancer

Phase 3 – BLA; Orphan Drug

TBD

zoliflodacin

Entasis

Gonorrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

Phase 3 (Supplementals) anakinra (Kineret®)

Swedish Orphan Biovitrum

COVID-19

Phase 3 – sBLA

TBD

baricitinib (Olumiant)

Eli Lilly

Alopecia areata; COVID-19; JIA; SLE; Uveitis

Oral

Phase 3 – sNDA; Breakthrough Therapy; Fast Track

TBD

benralizumab (Fasenra®)

AstraZeneca

ANCA-associated vasculitis; Bullous pemphigoid; Esophagitis; Nasal polyposis

Phase 3 – sBLA; Orphan Drug

TBD

cabotegravir (Vocabria)

Viiv

HIV-1 infection prevention

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

cariprazine (Vraylar®)

Allergan

MDD

Oral

Phase 3 – sNDA

TBD

empagliflozin (Jardiance)

Boehringer Ingelheim

Diabetic nephropathy

Oral

Phase 3 – sNDA

TBD

ferric carboxymaltose (Injectafer®)

Daiichi Sankyo

Anemia due to cytotoxic chemotherapy; Anemia in heart failure

Phase 3 – sNDA

TBD

ferric derisomaltose (Monoferric®)

Pharmacosmos

Anemia in heart failure

Phase 3 – sNDA

TBD

fostamatinib (Tavalisse®)

Rigel

Autoimmune hemolytic anemia

Oral

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 – sNDA

TBD

immune globulin intravenous (human) 10% (Octagam®)

Octapharma

COVID-19; Dermatomyositis

Phase 3 – sBLA; Orphan Drug

TBD

L-lactic acid/citric acid/ potassium bitartrate (Phexxi®)

Evofem

Chlamydia trachomatis infection; Neisseria gonorrhoeae infection

Intravaginal

Phase 3 – sNDA; Fast Track

TBD

mepolizumab (Nucala)

GlaxoSmithKline

COPD

IV, SC

Phase 3 – sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

Phase 3 – sNDA; QIDP TBD

nitazoxanide (Alinia®)

Lupin

COVID-19; Influenza

Oral

Phase 3 – sNDA

TBD

obeticholic acid (Ocaliva )

Intercept

NASH

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

omalizumab (Xolair®)

Genentech

Food allergies

Phase 3 – sBLA; Breakthrough Therapy

TBD

patisiran (Onpattro®)

Alnylam

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

Phase 3 – sNDA

TBD

42 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ravulizumab-cwvz (Ultomiris)

Alexion

HSCT-TMA

Phase 3 – sBLA

TBD

risankizumab-rzaa (Skyrizi)

Abbvie

CD; UC

Phase 3 – sBLA; Orphan Drug

TBD

rivaroxaban (Xarelto)

Janssen

COVID-19

Oral

Phase 3 – sNDA

TBD

romiplostim (Nplate )

Amgen

Thrombocytopenia

Phase 3 – sBLA; Orphan Drug

TBD

secukinumab (Cosentyx)

Novartis

Hidradenitis suppurativa

Phase 3 – sBLA

TBD

sodium hyaluronate/ triamcinolone hexacetonide (Cingal®)

Anika

Osteoarthritis (knee)

Intraarticular

Phase 3 – sNDA

TBD

ticagrelor (Brilinta®)

AstraZeneca

Sickle cell disease

Oral

Phase 3 – sNDA

TBD

tisagenlecleucel-t (Kymriah®)

Novartis

DLBCL (1st relapse)

Phase 3 – sBLA

TBD

tocilizumab (Actemra)

Genentech

COVID-19

Phase 3 – sBLA

TBD

upadacitinib (Rinvoq)

Abbvie

CD; Giant cell arteritis; UC

Oral

Phase 3 – sNDA; Orphan Drug

TBD

Complete Response Letter (CRL)/Withdrawn Drugs NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

paclitaxel/encequidar

Athenex

Breast cancer

Oral

CRL

TBD

pembrolizumab (Keytruda)

Merck

Breast cancer (highrisk early-stage TNBC, as neoadjuvant in combination with chemotherapy, followed by adjuvant monotherapy)

CRL

TBD

pimavanserin (Nuplazid®)

Acadia

Dementia-related hallucinations and delusions

Oral

CRL

TBD

ropeginterferon alfa-2b

Pharmaessentia

Polycythemia vera

CRL

TBD

taurolidine/heparin/citrate

Cormedix

Prevention of IV catheter-related sepsis (hemodialysis patients)

CRL

TBD

43 | 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

CF Cystic Fibrosis CHF Congestive Heart Failure CI Confidence Interval

ACR50 American College of Rheumatology 50% Improvement

CKD Chronic Kidney Disease

ACR70 American College of Rheumatology 70% Improvement

CNS Central Nervous System

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

CLL Chronic Lymphocytic Leukemia

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

AML Acute Myeloid Leukemia

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

ANCA Antineutrophil Cytoplasmic Antibodies

DEA Drug Enforcement Administration

ANDA Abbreviated New Drug Application

DLBCL Diffuse Large B Cell Lymphoma

ARB Angiotensin II Receptor Blocker

DMARD Disease Modifying Antirheumatic Drug

ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor

DMD Duchenne Muscular Dystrophy

ART Antiretroviral Therapy ARV Antiretroviral AS Ankylosing Spondylitis ASCVD Atherosclerotic Cardiovascular Disease AST Aspartate Aminotransferase BCVA Best Corrected Visual Acuity

DMT Disease Modifying Therapy DNA Deoxyribonucleic Acid DOR Duration of Response DPI Dry Powder for Inhalation DPP-4 Dipeptidyl Peptidase 4 DR Delayed-Release

BLA Biologics License Application

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

BMI Body Mass Index

ECOG Eastern Cooperative Oncology Group

BsUFA Biosimilar User Fee Act

EDSS Expanded Disability Status Scale

CABP Community Acquired Bacterial Pneumonia

eGFR estimated Glomerular Filtration Rate

CAP Community Acquired Pneumonia

ER Extended-Release

CAR T Chimeric Antigen Receptor T Cell

ESRD End-Stage Renal Disease

44 | MAGELLANRX.COM

GLOSSARY continued FDA Food and Drug Administration

ITT Intent-To-Treat

FH Familial Hypercholesterolemia

IV Intravenous

FLT3 FMS-Like Tyrosine Kinase-3

JIA Juvenile Idiopathic Arthritis

G-CSF Granulocyte Colony Stimulating Factor

LDL Low-Density Lipoprotein

GI Gastrointestinal

LDL-C Low-Density Lipoprotein Cholesterol

GIST Gastrointestinal Stromal Tumor

mAb Monoclonal Antibody

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

MACE Major Adverse Cardiovascular Events

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

MADRS Montgomery – Åsberg Depression Rating Scale

GVHD Graft Versus Host Disease H Half HAART Highly Active Antiretroviral Therapy 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

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

HER Human Epidermal Growth Factor Receptor

NIAID National Institute of Allergy and Infectious Diseases

HER2 Human Epidermal Growth Factor Receptor 2

NSAID Non-Steroidal Anti-Inflammatory Drug

HFA Hydrofluoroalkane

NSCLC Non-Small Cell Lung Cancer

HIT Heparin Induced Thrombocytopenia

ODT Orally Disintegrating Tablet

HIV Human Immunodeficiency Virus

OR Odds Ratio

HIV-1 Human Immunodeficiency Virus-1

ORR Overall/Objective Response Rate

HR Hazard Ratio

OS Overall Survival

HSCT Hematopoietic Stem Cell Transplant

OTC Over-the-Counter

HTN Hypertension

PAH Pulmonary Arterial Hypertension

IBS Irritable Bowel Syndrome

PARP Poly(ADP-ribose) polymerase

IBS-C Irritable Bowel Syndrome, Constipation Predominant

PASI Psoriasis Area and Severity Index

IGA Investigator's Global Assessment IM Intramuscular ITP Immune Thrombocytopenic Purpura

45 | MAGELLANRX.COM

PASI 50 Psoriasis Area and Severity Index 50% PASI 70 Psoriasis Area and Severity Index 70% PASI 90 Psoriasis Area and Severity Index 90% PASI 100 Psoriasis Area and Severity Index 100%

GLOSSARY continued PCI Percutaneous Coronary Intervention

sNDA supplemental New Drug Application

PCSK9 Proprotein Convertase Subtilisin Kexin 9

SNRI Serotonin and Norepinephrine Reuptake Inhibitor

PD-1 Programmed Death Protein 1 PD-L1 Programmed Death-Ligand 1 PDUFA Prescription Drug User Fee Application PFS Progression-Free Survival PGA Physician Global Assessment PsA Psoriatic Arthritis PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty

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

PTSD Post-Traumatic Stress Disorder

TEAE Treatment-Emergent Adverse Events

Q Quarter

TNBC Triple Negative Breast Cancer

QIDP Qualified Infectious Diseases Product

TNF Tumor Necrosis Factor

QOL Quality of Life

TNFα Tumor Necrosis Factor-alpha

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

UA Unstable Angina

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 46 | MAGELLANRX.COM

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