MRx Pipeline - July 2020

July 2020

MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS

TABLE OF CONTENTS Introduction Pipeline Deep Dive

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

Keep on Your Radar

Consultant Panel

Pipeline Drug List

Michelle Booth, PharmD Director, Medical Pharmacy Strategy

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

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

Glossary

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

Nothing herein is or shall be construed as a promise or representation regarding past or future events and Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Magellan Rx Management.

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

While select investigational agents for COVID-19 are profiled, given the rapidly evolving COVID-19 landscape, MRx created an exclusive report dedicated to drugs and vaccines in development for COVID-19. As cases surge in certain parts of the country and the globe, safe and effective vaccines and drugs are crucial to combat the pandemic.

MRx Pipeline+ Bonus COVID-19 Edition centralizes relevant information on emerging COVID-19 therapeutics and vaccines.

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

Clinical analyses, financial outlook, and pre-regulatory status are considered as part of the evaluation process.

The products housed in the MRx Pipeline have been researched in detail and developed in collaboration and in consultation with our internal team of clinical and analytics experts.

Emerging therapeutics continue to grow and influence the clinical and financial landscape. Therefore, Magellan Rx Management has developed a systematic approach to determine the products with significant clinical impact. For the in-depth clinical evaluations, the products’ potential to meet an underserved need in the market by becoming the new standard of care, and the ability to replace existing therapies were investigated. The extent to which the pipeline drugs could shift market share on a formulary and their impact on disease prevalence were also important considerations.

In order to assist payers with assessing the potential impact of these pipeline drugs, where available, a financial forecast has been included for select products. Primarily complemented by data from EvaluateTM, this pipeline report looks ahead at the 5-year projected annual US sales through the year 2024. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, such as the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business.

So far in 2020, a total of 29 novel drugs have received FDA approval. Last year at approximately the same timepoint, only 17 novel drugs had been approved. For the remainder of this year, 54 relevant drugs filed with the agency are profiled, each of which has an anticipated FDA decision in 2020. The progress of these agents is being actively monitored through MRx Pipeline.

In the past few years, game changers, such as chimeric antigen receptor (CAR) T therapies, have transformed the pipeline by delivery of drug therapy directly to the site of action in the body. As we look ahead, a continued trend toward the approval of specialty medications, drugs for rare diseases, growth of biosimilars, new treatment modalities using gene therapy, and additional CAR-T therapies are expected. Noteworthy pipeline trends to watch in 2020 include therapies for COVID-19, the highly-anticipated first gene therapy for hemophilia A, and the first treatment for genetic deficiency obesity. In the upcoming quarters, development of complex therapies, therapeutic options for rare hereditary diseases, oncology, immunology, neurology, and investigational agents will be monitored on the MRx radar. Moreover, sprouting products for cardiology, ophthalmology, hematology, and women’s health await on the horizon.

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

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

SPECIALTY

PRIORITY REVIEW

81%

25% BIOSIMILAR

44%

BREAKTHROUGH THERAPY

31% ORPHAN DRUG

38%

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

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ENDOCRINE

berotralstat oral Biocryst PROPOSED INDICATIONS

Hereditary angioedema (HAE) attack prevention

CLINICAL OVERVIEW

HAE is a rare genetic condition characterized by recurrent episodes of SC or submucosal edema of the GI tract, limbs, face, and upper respiratory tract. Patients with HAE types 1 and 2 have low levels of functional C1 esterase inhibitor (C1-INH), a protein that blocks plasma kallikrein. During HAE attacks, unregulated plasma kallikrein activity results in excessive production of the vasodilator bradykinin, leading to localized swelling and inflammation. Most HAE episodes are self-limiting and resolve in 3 to 5 days. However, abdominal edema may lead to nausea, vomiting, and severe pain. Life-threatening swelling of the throat or larynx can also occur.

Frequency of attacks can vary greatly and can occur without any apparent trigger.

Berotralstat is a second-generation, oral plasma kallikrein inhibitor. It was studied in 2 placebo-controlled phase 3 trials in 79 patients ≥ 12 years of age with type 1 or 2 HAE. The APeX-2 trial reported, from baseline to week 24, a 44.2% reduction in rate of HAE attacks (primary endpoint), a 49.2% reduction in HAE attacks requiring treatment, and a 53.6% reduction in on-demand medication use associated with once-daily oral berotralstat 150 mg compared to placebo (p<0.001 for all). The most common adverse effects reported were mild to moderate GI symptoms. ApeX-2 trial data at 48-weeks and the long-term (48-week) open-label Apex-S trial confirmed that berotralstat is generally well tolerated. Patients experienced sustained reductions in HAE attack frequency and improved QOL.

PLACE IN THERAPY

HAE affects 1 person in 10,000 to 50,000 in the US. Symptoms of HAE begin in early childhood and persist throughout life. Management consists of acute treatment of attacks, short-term prophylaxis during precipitating conditions, and long-term prophylaxis for frequent attacks. Agents approved in the US employ various mechanisms to manage HAE. Products that increase levels of C1-INH include danazol or replacement with plasma-derived C1-INH (Berinert®, Cinryze®, Haegarda®) and recombinant C1-INH (Ruconest®). The kallikrein inhibitors escallantide (Kalbitor®) and lanadelumab-flyo (Takhzyro®) and the bradykinin receptor antagonist icatibant (Firazyr®) are also available. Treatment options for acute attacks include Berinert (IV), Ruconest (IV), Firazyr (SC), and Kalibitor (IV). Danazol (oral) administered 3 times a day, Cinryze (IV) and Haegarda (SC) administered every 3 or 4 days, and Takhzyro (SC) administered every 2 or 4 weeks are used for routine prophylaxis of HAE attacks. Except for Kalbitor, all approved products can be self-administered.

Berotralstat’s oral formulation and dosage frequency may be more convenient compared to the other prophylactic agents (danazol, Cinryze, Haegarda, Takhzyro). Furthermore, the safety profile for berotralstat is more favorable compared to oral danazol, which carries several contraindications and boxed warnings (e.g., thrombosis) due to androgen-like effects. Conversely, SC Takhzyro reported a much higher reduction in monthly attack rates compared to berotralstat (Takhzyro, 74% to 87%; berotralstat, 44.2%) in noncomparative trials. Notably, there are no patents pending to prevent approval of biosimilars to Cinzyre that could compete with berotralstat. While berotralstat has only been studied for prevention of HAE attacks in patients ≥ 12 years of age, over time, manufacturers of products within this class have sought approval in younger patients. In addition, a liquid formulation of berotralstat is being studied for acute HAE attacks.

FDA APPROVAL TIMELINE December 3, 2020  Fast Track

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$3

$29

$84

$142

$196

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

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ONCOLOGY

idecabtagene vicleucel IV Bristol-Myers Squibb/Bluebird Bio PROPOSED INDICATIONS

Relapsed or refractory (R/R) multiple myeloma (MM)

CLINICAL OVERVIEW

Idecabtagene vicleucel (ide-cel) is a B cell maturation antigen (BCMA)-directed autologous chimeric antigen receptor (CAR) T cell therapy. It binds to BCMA on the surface of MM cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic destruction of BCMA-expressing tumor cells.

Ide-cel demonstrated clinical efficacy in the open-label, single-arm, phase 2 KarMMa trial among 128 patients with R/R MM who had received ≥ 3 prior regimens, including treatment with an immunomodulatory agent, proteasome inhibitor, and a CD38-directed antibody. In addition, 94% of patients received previous autologous SCT. A total of 128 patients received a single dose of ide-cel. A dose-dependent, clinically meaningful response was reported across all 3 doses studied (range, 150 x 106 to 450 x 106 cells). At a median follow-up of 11.3 months, responses observed with the highest dose were: ORR, 81%; complete response (CR), 35%; median DOR, 11.3 months; and median PFS, 11.3 months. Response rates were consistent among older individuals and difficult-to-treat patients (e.g., penta-refractory, high tumor burden, extramedullary disease). Median peak CAR+ T cell expansion was detected 11 days post dose. Cytokine release syndrome (CRS) was reported in 84% of patients in the overall population, including 7 cases of grade ≥ 3 severity. Neurotoxicity (NE) was reported in 18% of patients, including 4 cases of grade 3 severity. To manage CRS and NE adverse effects, tocilizumab was administered in 52% and 2% of patients, respectively, while corticosteroids were used in 15% and 8% of patients, respectively. Neutropenia and thrombocytopenia were observed in 91% and 63% of patients (mostly grade ≥ 3), respectively. Within 8 weeks of the dose, 5 deaths occurred; 2 of these deaths were due to disease progression and 3 were due to adverse events (CRS, aspergillus pneumonia, GI hemorrhage). One death occurred within 6 months of the dose due to cytomegalovirus pneumonia.

PLACE IN THERAPY

Over 32,270 new cases of MM and 12,830 deaths due to the condition are predicted in the US in 2020. While patients typically respond to initial therapy, eventual treatment-resistant relapse usually occurs.

If approved, ide-cel will be the first CAR-T therapy for MM. Autologous CAR-T therapy reengineers T cells collected from the patient. After re-infusion back into the patient’s blood, the modified cells recognize and kill cancerous B cells. Ide-cel’s activity targets BCMA, which is found in 60% to 70% of MM patients.

Ide-cel demonstrated a significant and durable response in heavily pretreated MM patients. The initial indication is expected to be as fourth-line treatment, an area where poor response to available therapy is observed. Single-dose ide-cel will likely compete with the CD38-directed cytolytic antibody isatuximab-irfc (Sarclisa®), histone deacetylase inhibitor panobinostat (Farydak®), thalidomide analogue pomalidomide (Pomalyst®), proteasome inhibitor ixazomib (Ninlaro®) as well as the investigational BCMA-targeting antibody drug conjugate belantamab mafodotin (anticipated approval, August 2020). Several other BCMA-directed therapies are on the horizon, including CAR-T therapies by Janssen, Poseida, and Cartesian, and bispecific antibodies targeting BCMA and CD38 by Amgen and Regeneron (phase 2 for all). Ide-cel is also being studied in earlier lines of treatment.

FDA APPROVAL TIMELINE March 31, 2021

 Breakthrough Therapy

 Orphan Drug

FINANCIAL FORECAST 2020

2021

2022

2023

2024

$11

$153

$343

$520

$715

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

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ONCOLOGY

margetuximab IV Macrogenics PROPOSED INDICATIONS

Human epidermal growth factor receptor 2 (HER2)-positive breast cancer

CLINICAL OVERVIEW

Margetuximab is a monoclonal antibody that targets HER2 oncoprotein. It is engineered with a fragment crystallizable (Fc) domain to enhance the body’s immune response.

The open-label, phase 3 SOPHIA trial compared margetuximab (n=266) with trastuzumab (n=270), each given in combination with chemotherapy (capecitabine, eribulin, gemcitabine, or vinorelbine), for the treatment of HER2-positive metastatic breast cancer after anti-HER2 therapy (trastuzumab-based therapy, pertuzumab).

As of the September 2019 data cut-off, in the ITT population, margetuximab led to a slightly higher median OS compared to trastuzumab (median OS, 21.6 versus 19.8 months, respectively; p=0.326 [not significant]), and there was only a nominal increase in median PFS (5.7 versus 4.4 months, respectively; p=0.0006).

Interestingly, response to margetuximab compared to trastuzumab was more pronounced in patients with CD16A genotypes containing 158F allele (median PFS, 6.9 versus 5.1 months, respectively; p=0.005; median OS, 23.7 versus 19.4 months, respectively; p=0.087). Overall, the ORR was 25.2% versus 13.7% in the margetuximab and trastuzumab groups, respectively (p=0.0006) and clinical benefit rates were 48.1% and 35.6%, respectively (p=0.0025). In general, safety profiles were similar among the agents, with the exception of infusion-related reactions (margetuximab, 13%; trastuzumab, 3%).

The studied dose of margetuximab was 15 mg/kg via IV infusion on day 1 of each 21-day cycle, until progression or unacceptable toxicity occurred. It was administered over 120 minutes in cycle 1, then over 30 or 60 minutes during subsequent cycles.

PLACE IN THERAPY

Breast cancer is the second leading cause of cancer death in women. In 2020, an estimated 279,100 new cases of breast cancer and 42,690 related deaths will occur. Approximately 15% of cases are HER2-positive.

Trastuzumab-based therapy is the current SOC for HER2-positive breast cancer. However, only 25% to 30% of HER2-positive patients with metastatic disease will respond to SOC, leaving a large unmet need.

Margetuximab contains an Fc domain engineered to provide increased CD16A 158F allele binding and decreased CD32B binding on immune cells; 85% of the population harbors the CD16A 158F allele which is associated with a diminished response to available therapeutic antibodies, including trastuzumab. As seen in the SOPHIA trial, while margetuximab demonstrated a similar response to trastuzumab in the ITT HER2positive population, it may provide an enhanced response in patients with the CD16A 158F allele.

If approved, margetuximab in combination with chemotherapy will provide another option for the treatment of HER2-positive metastatic breast cancer. It remains to be seen if its modest clinical advantage over trastuzumab will allow it to compete with established therapies. These include trastuzumab-containing IV and SC administered products (including Herceptin® and its biosimilars, Herceptin Hylecta™, Enhertu®, and Kadcyla®), as well as oral regimens with kinase inhibitors lapatinib (Tykerb®), neratinib (Nerlynx®), and tucantinib (Tukysa™) that are used in combination with capecitabine (Xeloda®) in patients who have failed prior treatment with trastuzumab.

FDA APPROVAL TIMELINE December 18, 2020  Fast Track

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$12

$44

$83

$121

$159

The forecast is a projection of total US sales per year, including leukemia indications.

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HEMATOLOGY

roxadustat oral AstraZeneca PROPOSED INDICATIONS

Anemia of chronic kidney disease (CKD), including dialysis- and non-dialysis-dependent

CLINICAL OVERVIEW

Roxadustat is a hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI). HIF-PHIs stimulate erythropoietin production in the kidneys and liver, improve iron storage, and downregulate hepcidin–a key regulator of iron homeostasis.

Phase 3 trials compared roxadustat to placebo in non-dialysis-dependent patients with CKD (n=4,277) and compared roxadustat to epoetin alfa in CKD patients on dialysis (n=3,880). Pooled analyses demonstrated a statistically significant mean increase from baseline in Hb levels (averaged over 28 to 52 weeks) with roxadustat versus both comparators. In non-dialysis-dependent patients, the difference from placebo was 1.72 g/dL (p<0.001), and in dialysis-dependent patients, the difference from epoetin alfa was 0.23 g/dL (p<0.0001). During the first year of treatment, fewer patients treated with roxadustat required RBC transfusions compared to those treated with placebo (5.2% versus 15.4%, respectively; p<0.001). This was also true for roxadustat compared to epoetin alfa (9.5% versus 12.8%, respectively; p=0.046). Risk of MACE and all-cause mortality with roxadustat was similar to placebo in non-dialysis-dependent patients. There was no increase in the risk of MACE or all-cause mortality with roxadustat compared to epoetin alfa in dialysis-dependent patients. Response to roxadustat was demonstrated regardless of baseline iron stores.

The open-label, phase 3 DOLOMITE trial reported non-inferiority of roxadustat to darbepoetin alfa in 616 CKD patients not on dialysis. This was based on Hb response, defined as Hb ≥ 11 g/dL and an increase in Hb level by ≥ 1 g/dL in patients with baseline Hb > 8 g/dL or by ≥ 2 g/dL in those with baseline Hb ≤ 8 g/dL at week 24. Rate of response was 89.5% with roxadustat and 78% with darbepoetin alfa (p<0.05). Incidence of MACE was numerically lower with roxadustat, but the difference was not statistically significant.

Roxadustat was administered orally 3 times per week in clinical trials.

PLACE IN THERAPY

An estimated 37 million people in the US have CKD. Anemia is prevalent in patients with CKD and worsens as disease progresses. Anemia can lead to arrhythmia, cardiomegaly, and heart failure as well as fatigue and decreased QOL. Treatment with iron, vitamin B12, and folic acid supplementation, erythropoietin stimulating agents (ESAs; epoetin alfa [Epoetin®, Procrit®], darbepoetin alfa [Aranesp®]), and RBC transfusions are used to manage anemia. However, transfusions can reduce the patient’s eligibility for kidney transplant and increase risk of infections, heart failure, and allergic reactions.

Roxadustat is the first orally-administered small molecule HIF-PHI to be submitted to the FDA for treatment of CKD-related anemia. It has the potential to become the new SOC for anemia in CKD due to its convenient oral administration (ESAs are administered IV or SC), safety profile, and reduced need for iron supplements.

Other HIF-PHIs in phase 3 clinical trials for this indication in the US include oral daprodustat and vadadustat.

HIF pathways impact many biologic processes; therefore, there is potential concern about non-erythropoietic adverse effects, including increased risk of cancer, thrombosis, CVD, and diabetic retinopathy with this new therapeutic class.

FDA APPROVAL TIMELINE December 18, 2020

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$17

$190

$339

$460

$560

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

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METABOLIC

setmelanotide SC Rhythm PROPOSED INDICATIONS

Pro-opiomelanocortin deficiency obesity (POMC-DO) and leptin receptor deficiency obesity (LEPR-DO)

CLINICAL OVERVIEW

Genetic factors play a role in the obesity cascade. POMC-DO and LEPR-DO are ultra-rare autosomal recessive single-gene disorders that impact the energy and appetite regulating leptin-melanocortin pathway. Both are characterized by lifelong persistent hunger and weight gain beginning in the first year of life as well as hormone deficiencies. Patients with POMC-DO lack hypothalamic melanocyte-stimulating hormones and tend to have red hair and pale skin. These individuals also exhibit low levels of adrenocorticotropic hormone (ACTH), which if left untreated, may lead to life-threatening hypoglycemia, seizures, hyperbilirubinemia, and cholestasis. It is unknown if patients with POMC-DO are at increased risk of CVD, cancer, or T2DM. Likewise, LEPR-DO is associated with hypogonadotropic hypogonadism and patients experience delayed or absent puberty. Aggressive food-seeking behavior and development of T2DM in early adulthood are also reported.

POMC-DO and LEPR-DO involve gene abnormalities upstream of the melanocortin-4 receptor (MC4R) in the leptin-melanocortin pathway. Setmelanotide, a highly specific MC4R agonist, restores MC4R function.

Two open-label, single-arm, phase 3 trials evaluated setmelanotide for the treatment of POMC-DO (n=10) and LEPR-DO (n=11) in patients ≥ 6 years of age. Setmelanotide was self- or caregiver-administered as once daily SC injections. At 52 weeks, 80% and 45.5% of patients in the POMC-DO and LEPR-DO cohorts, respectively, achieved the primary endpoint of ≥ 10% change in body weight from baseline. The mean reductions in body weight were 25.4% and 12.5%, respectively (p<0.0001 for both). Significant reductions from baseline in Most Hunger score were also reported (27.8% and 41.9%, respectively; p≤0.0004 for both). Among patients with POMC-DO, the mean change in BMI from baseline differed by age (≥ 19 years, -22.3% [p=0.056]; < 19 years, -49.2% [p=0.007]); smaller changes in each age group occurred in patients with LEPR-DO (-10.6% [p=0.01] and -13.4% [p=0.12], respectively). An ongoing long-term extension trial (POMC-DO, n=9; LEPR-DO, n=6) reported a durable weight loss for up to 155 weeks. The most common TEAEs reported were injection site reactions, nausea, vomiting, and hyperpigmentation. No significant changes in CV events, blood pressure, or heart rate were reported.

PLACE IN THERAPY

While approximately 50 cases of POMC deficiency obesity have been reported in literature, the prevalence of LEPR deficiency obesity is unknown. Rhythm Pharmaceuticals estimates that the US incidence of each are 100 to 500 and 500 to 2,000, respectively. Diagnoses for these disorders are confirmed using genetic testing. Individuals with either condition struggle with dramatic weight gain that does not respond to lifestyle changes or available therapeutic interventions. Patients also experience endocrine abnormalities that could be life-threatening if not treated (POMC-DO) or result in infertility (LEPR-DO).

If approved, setmelanotide will be a first-in-class treatment for POMC-DO and LEPR-DO and could alter the disease course. Setmelanotide is also in clinical trials for obesity caused by other single-gene defects associated with hyperphagia and obesity, including Bardet-Biedl and Alström syndromes (phase 3) and Prader-Willi syndrome (phase 2). A once-weekly depot formulation is also in development.

FDA APPROVAL TIMELINE November 27, 2020

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$5

$56

$166

$269

$405

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

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 Rare Pediatric Disease

ONCOLOGY

sodium thiosulfate IV Fennec PROPOSED INDICATIONS

Prevention of cisplatin-induced ototoxicity

CLINICAL OVERVIEW

Sodium thiosulfate (STS) is an antioxidant that may inactivate oxygen free-radicals and electrophilic platinum species and thereby preserve hearing.

STS was studied in 2 open-label, phase 3 trials in pediatric patients who required cisplatin-based chemotherapy. The ACCL0431 trial included 104 evaluable patients 1 year to 18 years of age with various newly-diagnosed malignancies. The SIOPEL-6 trial enrolled 109 pediatric patients 1 month to 18 years of age with standard-risk localized hepatoblastoma. Both trials reported a nearly 50% lower incidence of hearing loss in patients treated with STS compared to patients who did not receive STS (observation control group) (ACCL0431: 28.6% versus 56.4%, respectively; SIOPEL-6: 33% versus 63%, respectively).

In ACCL0431, there was also a greater between-group difference in incidence of hearing loss, favoring STS, in patients < 5 years of age (difference, 51.9%) compared to those ≥ 5 years (difference, 18.6%). In ACCL0431, durability of response was observed 1 year post therapy completion and response to STS was not impacted by CNS irradiation. Both studies reported no difference in OS or event-free survival (EFS) between the overall study populations at 3 years; however, in ACCL0431, these rates were lower in the STS group compared with the control group in patients with disseminated disease (OS, 42% versus 61%, respectively; EFS, 45% versus 84%, respectively). Across the 2 studies, 1 patient developed metabolic acidosis, which resolved after STS was stopped. Grade ≥ 3 hypophosphatemia and hypokalemia were reported more often in the STS group.

STS was administered at a dose of 16 mg/m2 (SIOPEL-6) or 20 mg/m2 (ACCL0431) IV over 15 minutes 6 hours after each cisplatin dose.

PLACE IN THERAPY

Cisplatin, a standard component of chemotherapy regimens for pediatric malignancies, can cause damage to cochlear cells of the ear. Approximately 5,000 children in the US receive platinum-based chemotherapy each year, 40% to 60% of whom receive cisplatin and are at risk of irreversible bilateral hearing loss. This risk may be more pronounced by renal impairment, concomitant use of other ototoxic or renal toxic drugs, and radiation therapy. Ototoxicity typically occurs during or shortly after cisplatin treatment but can occur months to years after stopping therapy. Hearing loss emerging during childhood developmental years can have a profound effect on speech, language, and psychosocial development.

STS demonstrated a significant reduction in the risk of ototoxicity associated with cisplatin therapy, particularly in patients < 5 years of age, who are most susceptible to cisplatin-induced hearing loss. If approved, STS will be the first and only drug indicated to prevent chemotherapy-induced ototoxicity, an area with no other drugs in late stage research. While STS appears to have no impact on survival in the overall study populations, the ACCL0431 study raised a concern of potential decreased survival when disseminated disease is present.

FDA APPROVAL TIMELINE August 10, 2020

 Breakthrough Therapy

 Fast Track

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$10

$30

$55

$73

$95

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

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 Priority Review

IMMUNOLOGY

sutimlimab IV Sanofi PROPOSED INDICATIONS Cold agglutinin disease

CLINICAL OVERVIEW

Cold agglutinin disease is a rare autoimmune disorder characterized by hemolytic anemia. Autoantibodies bind to RBCs causing them to agglutinate (clump). The RBCs hemolyze leading to anemia. Cause of cold agglutinin disease may be unknown (primary) or due to conditions such as infection, other autoimmune disease, or certain cancers (secondary). Common symptoms are related to anemia and can include fatigue, weakness, shortness of breath, dizziness, headache, chest pain, and arrhythmia. Patients may also experience Raynaud’s syndrome, splenomegaly, mottled skin, or thromboembolic events. Symptoms are often triggered by cold temperatures or viral infection.

Sutimlimab is a humanized immunoglobulin G4 (IgG4) monoclonal antibody that inhibits activation of the classical complement pathway which is overactive in people with cold agglutinin disease.

The 26-week, single-arm, phase 3 CARDINAL trial evaluated sutimlimab in 24 patients with cold agglutinin disease who received a blood transfusion in the previous 6 months. Improvement in Hb levels (≥ 1 g/dL increase) was seen within 1 week of starting sutimlimab therapy. Among patients treated, 62.5% achieved a Hb ≥ 12 g/dL or an increase of ≥ 2 g/dL. A mean Hb > 11 g/dL was maintained from week 3 to study end, and 71% of patients were transfusion-free after week 5. As early as week 1, clinically meaningful improvements in fatigue and bilirubin level were reported. An increase in FACIT-fatigue score from baseline (mean, 10.9 points) suggested improved QOL. Total bilirubin level normalized by week 3 and was maintained through week 26. No serious adverse effects reported were considered related to sutimlimab.

Sutimlimab was administered as a weight-based dose (6.5 g or 7.5 g) via IV infusion on days 0 and 7, followed by once every other week through week 26.

PLACE IN THERAPY

The incidence of cold agglutinin disease is approximately 1 in 300,000. It is most often seen in middle aged or elderly adults. There are no approved treatments for the condition. Prognosis is generally good in mild to moderate cases with avoidance of cold temperature and in cases caused by viral infection. The prognosis is worse in individuals with underlying HIV infection or cancer. According to Sanofi, patients who suffer from cold agglutinin disease are at a 55% increased risk of thromboembolic events.

Blood transfusions or plasmapheresis provide temporary support for severe hemolytic anemia. Rituximab (Rituxan®, biosimilars) is effective in the majority of chronic cases (≥ 60%) with duration of response of 1 to 2 years. Unfortunately, response to rituximab may diminish with repeated use. Increased response and duration have been reported with the addition of fludarabine or bendamustine; however, adverse effects may limit the use of these agents. Off-label uses of eculizumab (Soliris®) and ibrutinib (Imbruvica®) have also been reported in the literature. If approved, sutimlimab will be the first therapeutic treatment indicated for cold agglutinin disease. While sutimlimab demonstrated efficacy in patients who received recent blood transfusion, the ongoing phase 3 CADENZA study is also assessing it in patients with cold agglutinin disease without recent history (prior 6 months) of transfusion.

FDA APPROVAL TIMELINE November 13, 2020

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$4

$35

$82

$121

$155

The forecast is a projection of total US sales per year, including cold agglutinin disease and ITP. 10 | magellanrx.com

ONCOLOGY

tafasitamab IV Morphosys PROPOSED INDICATIONS

Relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) in combination with lenalidomide

CLINICAL OVERVIEW

Tafasitamab is a humanized monoclonal antibody that contains a modified Fc domain. Tafasitamab targets CD19 expressed on B cells. The modified Fc domain enhances binding to the B cell, resulting in potent antibody-dependent cellular cytotoxicity (ADCC) and downregulation of B cell activation.

Tafasitamab was studied in the phase 2, single-arm L-MIND clinical trial in combination with lenalidomide in 81 adults with R/R DLBCL. Patients had received 1 to 3 prior systemic therapies, including ≥ 1 anti-CD20 agent (e.g., rituximab). Patients had an ECOG score of 0 to 2 and were ineligible for high-dose chemotherapy and autologous SCT. After a median follow-up of 31.8 months (95% CI, 27.2 to 35.9), the study reported an ORR of 58.8% (primary endpoint), complete response (CR) of 41.3%, and median DOR of 34.6 months (95% CI, 26.1 to 34.6). The median OS was 31.6 months (95% CI, 18.3 to not reached [NR]) and PFS was 16.2 months (95% CI, 6.3 to NR). The most common grade ≥ 3 TEAEs reported with tafasitamab plus lenalidomide therapy (≥ 10%) were neutropenia, thrombocytopenia, and febrile neutropenia. In patients with R/R DBLCL, a significantly higher ORR was reported with tafasitamab plus lenalidomide in the L-MIND trial compared with lenalidomide monotherapy in the retrospective, observational (real-world), matched control cohort RE-MIND trial (n=490; ORR, 34.2%).

In L-MIND, patients received 28-day cycles of tafasitamab and lenalidomide treatment. Tafasitamab 12 mg/kg was administered IV once weekly during cycles 1 through 3 (with a loading dose on day 4 of cycle 1), then every 2 weeks during cycles 4 through 12. Lenalidomide 25 mg was given orally on days 1 to 21 of cycles 1 through 12. After cycle 12, progression-free patients received tafasitamab every 2 weeks until disease progression.

PLACE IN THERAPY

Over 25,000 new cases of DLBCL are diagnosed annually in the US. While some aggressive forms are often curable with intensive chemotherapy, others are less responsive. An estimated 30% to 40% of cases relapse or progress after SOC therapy (R-CHOP). Few options remain for patients who are refractory to or relapse after chemotherapy or who are ineligible for SCT. Recently approved therapies target specific biomarkers on tumor cells and stimulate the body’s immune response against tumor cells.

If approved, tafasitamab will provide an important option for treating R/R DLBCL in patients who are not suitable for autologous SCT or high-dose chemotherapy. It may compete with rituximab-containing regimens (Rituxan and its biosimilars), polatuzumab vedotin-piiq (Polivy™), and CAR-T therapies (Kymriah®, Yescarta®) in this oncology space. Moreover, tafasitamab has shown strong response rates and may be appropriate for patients whose clinical status is not amenable for CAR-T therapy or polatuzumab vedotin-piiq.

The phase 3 B-MIND trial is studying tafasitamab in combination with bendamustine for R/R DLBCL; results are expected in 2022.

FDA APPROVAL TIMELINE August 28, 2020

 Breakthrough Therapy

 Fast Track

 Orphan Drug

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$39

$166

$292

$408

$531

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

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 Priority Review

NEUROLOGY

viloxazine oral Supernus PROPOSED INDICATIONS

Attention deficit hyperactivity disorder (ADHD)

CLINICAL OVERVIEW

Viloxazine is a serotonin norepinephrine modulating agent. It demonstrates increased levels of serotonin (5-HT), norepinephrine (NE), and dopamine (DA) in the prefrontal cortex of the brain.

Its use as a non-stimulant for the treatment of ADHD was evaluated across 4 double-blind, placebo-controlled, phase 3 clinical trials in children (ages 6 to 11 years; studies P301 and P303) and adolescents (ages 12 to 17 years; studies P302 and P304). A total of 902 patients received viloxazine. Dosages were 100 mg, 200 mg, and 400 mg in children, and 200 mg, 400 mg, and 600 mg in adolescents. The studies showed that viloxazine was effective in significantly reducing the symptoms of hyperactivity/impulsivity and inattention (based on the ADHD Rating Scale-5) compared to placebo at study end (weeks 6 to 8, depending on study) for all doses except 600 mg. Improvements were observed as early as week 1. Similarly, significant improvement in the Clinical Global Impression-Improvement (CGI-I) score was demonstrated with viloxazine (for all dosages except the 600 mg) at the study end. The most common TEAEs reported were somnolence, decreased appetite, headache, and fatigue. Across the trials, 3.5% of patients treated with viloxazine discontinued treatment due to TEAEs compared to 1.3% who received placebo.

Viloxazine was studied as monotherapy administered orally once daily.

PLACE IN THERAPY

ADHD is the most common neurobehavioral childhood disorder, occurring in approximately 7% to 8% of children and youth. It is a chronic condition with core symptoms of inattention, hyperactivity, and difficulty controlling behavior. Medication in combination with behavioral therapy is recommended. Stimulants (e.g., amphetamine, methylphenidate) are the most frequently prescribed pharmacologic treatment for ADHD. While non-stimulant agents (e.g., atomoxetine [Strattera®], guanfacine ER [Intuniv®], and clonidine ER [Kapvay®]) are considered less effective, there is a niche for their use. Atomoxetine is recommended if there are concerns with using a controlled substance, if stimulant-induced weight loss is problematic, or for patients with anxiety, mood, tics, or substance abuse disorders. Extended-release formulations of guanfacine or clonidine may be helpful when used concurrently with a stimulant in patients who cannot tolerate usual stimulant doses, particularly in patients who experience tics.

If approved, viloxazine will provide another non-stimulant treatment option for children and adolescents with ADHD. It could compete with the selective NE reuptake inhibitor atomoxetine (Strattera, generics) if it can differentiate itself based on its distinct mechanism of action, which affects 5-HT, NE, and DA, as well as its safety profile. Unlike atomoxetine, which is contraindicated in patients with severe CV disorders and has been associated with rare cases of liver injury, viloxazine has not demonstrated CV impairment and hepatotoxicity in clinical trials. Furthermore, viloxazine’s safety record based on its use as an antidepressant in Europe may provide physicians some confidence in prescribing the medication. Finally, clinically significant response to viloxazine was demonstrated at weeks 6 to 8 of the studies, and onset of action was seen as early as 1 week.

Effect of atomoxetine on ADHD symptoms can also be seen within 1 to 2 weeks of starting therapy, with a gradual increase in response over 24 weeks. Viloxazine is also being studied in adults with ADHD.

FDA APPROVAL TIMELINE November 6, 2020

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$13

$55

$100

$141

$142

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

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Biosimilar Overview CLINICAL OVERVIEW

Biosimilars are very different from generic drugs in that they are not exact duplicates of their reference biologic product. The FDA approval process for biosimilars is designed to ensure that the biosimilar product is highly similar to the reference product without having any meaningful clinical differences.

Many controversies surround biosimilars, and regulatory and litigation hurdles remain. The FDA has issued final and draft guidances. Select FDA biosimilar guidances are noted here. In January 2017, the agency issued final guidance on the nonproprietary naming of biologic products, which also applies to biosimilars. The biological products must bear a core name followed by a distinguishing 4-letter, lowercase, hyphenated suffix that is devoid of meaning. The FDA is still considering how to implement the nomenclature for previously approved biosimilar products. The international nonproprietary name (INN) impacts interchangeability as it affects pharmacists’ ability to substitute an interchangeable biosimilar for the reference product. The FDA withdrew the September 2017 draft industry guidance on determining similarity of a proposed biosimilar product to its reference product to allow for further consideration of the most current and relevant scientific methods in evaluating analytical data. The agency will focus on providing flexibility for efficient development of biosimilars while maintaining high scientific standards.

In July 2018, the FDA finalized its guidance on labeling biosimilars. The guidance pertains to prescribing information (PI) but does not contain specific recommendations on interchangeability in the labeling. The labeling guidance provides recommendations on how to include, identify, and differentiate the biosimilar and the reference product in various sections of the PI. The basic premise remains that the originator product’s safety and effectiveness can be relied upon for HCPs to make prescribing decisions; therefore, a biosimilar should include relevant data from the originator in its PI. In May 2019, the agency released its final guidance on interchangeability. Several states had already enacted biosimilar substitution legislation.

Biosimilars are expected to receive full extrapolation for the eligible indications of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will likely need to be considered individually.

Insulins were historically regulated by the FDA as small molecules. However, effective March 23, 2020, drugs such as insulin and growth hormone were deemed biologics and transitioned from the drug pathway to the biologics pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.

PLACE IN THERAPY

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

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

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To date, a total of 28 biosimilars have received FDA approval. Of these, only 18 have entered the market.

APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zarxio® (filgrastim-sndz)

Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer/Celltrion

April 2016

Erelzi™ (etanercept-szzs)

Sandoz

August 2016

Amjevita™ (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Samsung Bioepis/ Merck

May 2017

Cyltezo® (adalimumab-adbm)

Boehringer Ingelheim

August 2017

Mvasi™ (bevacizumab-awwb)

Amgen

September 2017

Ixifi™ (infliximab-qbtx)*

Pfizer

December 2017

Ogivri™ (trastuzumab-dkst)

Mylan

December 2017

Retacrit® (epoetin alfa-epbx)

Pfizer/Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Mylan

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz™ (adalimumab-adaz)

Sandoz

October 2018

Udenyca® (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Celltrion/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Celltrion/Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Samsung Bioepis/ Merck

January 2019

Trazimera™ (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Samsung Bioepis/ Merck

April 2019

Kanjinti™ (trastuzumab-anns)

Amgen

June 2019

Zirabev™ (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™ (adalimumab-bwwd)

Samsung Bioepis/ Merck

July 2019

Ruxience™ (rituximab-pvvr)

Pfizer

July 2019

Abrilada™ (adalimumab-afzb)

Pfizer

November 2019

Ziextenzo® (pegfilgrastim-bmez)

Novartis/Sandoz

November 2019

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Commercially Available

  -

 -

 -

    -

     -

  -

 -



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

APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Avsola™ (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer/Hospira

June 2020

Hulio® adalimumab-fkjp)

Mylan

July 2020

Commercially Available

 -

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

* Pfizer already has Inflectra on the market and has not announced plans to launch Ixifi.

Also available are Eli Lilly’s Basaglar® insulin glargine, a biosimilar agent to Sanofi’s Lantus® , and Sanofi’s Admelog® insulin lispro, approved as a biosimilar product to Eli Lilly’s Humalog®. In June 2020, the FDA approved insulin glargine (Semglee™) by Mylan/Biocon under an abbreviated 505(b)(2) New Drug Application (NDA) pathway; the reference product was Lantus. Semglee is considered a biologic under section 351(a).

A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play a role in market adoption of biosimilars.

While < 2% of Americans use biologics, they account for almost 40% of all prescription drug spending. Moreover, they comprised 70% of growth in drug spending from 2010 to 2015. Not surprisingly, there is a growing body of evidence on predicted biologic spend and potential biosimilar savings. The global biologic market is projected to exceed $390 billion by 2020. The global biosimilar market is expected to grow from $5.95 billion in 2018 to $23.63 billion in 2023. An IMS Health analysis expects biosimilars to save the US and Europe’s top 5 markets up to $110 billion by 2020. In the US, it is estimated that biosimilars will cost 15% to 35% less than the originator product.

The potential cost savings, however, can vary based on the market segment where brand contracts can play a role.

A 2017 report by the RAND Corporation estimates a $54 billion cost savings from biosimilars between 2017 and 2026. In July 2018, an FDA analysis reported that if Americans had access to FDA-approved biosimilars in 2017, it would have resulted in a $4.5 billion savings. A 2017 analysis by the Moran Company projects biosimilars could save the government an estimated $11.4 billion by 2027, but it would require the Centers for Medicare and Medicaid Services (CMS) to revise its reimbursement policy for biosimilars. Subsequently, in November 2017, the CMS revised its reimbursement policy. The CMS now issues a unique Healthcare Common Procedure Coding System (HCPCS) code to each individual biosimilar. Under this rule, Medicare Part B separately codes and pays for biosimilars and no longer groups them into a common payment code with originator agents. A June 2018 infliximab case study by the Pacific Research Institute forecasts annual savings of up to $465 million from increased use of biosimilars to replace a single biologic for commercial payers and Medicare.

Biosimilars are paving the way for increased access to important biologic therapies that may increase survival and/ or QOL for many patients with difficult-to-treat diseases while also reducing costs.

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BIOSIMILAR OVERVIEW continued

ONCOLOGY

bevacizumab IV Merck/Samsung Bioepis Aybintio and Bmab-100 are 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

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

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$2,058

$1,496

$1,251

$1,078

$886

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

BLOOD MODIFIER

filgrastim IV, SC Apotex and Kashiv are seeking biosimilars to Amgen’s Neupogen, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs; following induction or consolidation chemotherapy for acute myeloid leukemia (AML); with nonmyeloid malignancies 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 Kashiv Pending

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$152

$126

$108

$96

$88

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

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BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

pegfilgrastim SC Lapelga and MSB-11455 are biosimilars to Amgen’s Neulasta, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs and in patients acutely exposed to myelosuppressive doses of radiation (HSARS).

FDA APPROVAL TIMELINE Apotex (Lapelga) Pending

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

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$1,918

$1,532

$1,255

$1,057

$915

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

ONCOLOGY

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

FDA APPROVAL TIMELINE December 18, 2020

FINANCIAL FORECAST (reported in millions) 2020

2021

2022

2023

2024

$3,577

$2,498

$1,850

$1,481

$1,150

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

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KEEP ON YOUR RADAR Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the MRx Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2024, are displayed. The financials are projected total annual US sales, reported in millions. voclosporin

aducanumab

$649

$940

Immunology

Neurology

remdesivir COVID-19

betibeglogene autotemcel (Zynteglo) Hematology/Gene therapy

$1,190

$366

relugolix

casimersen

Women's health/ Oncology

Neurology/Gene therapy

$187

$578

efgartigimod

mRNA-1273 vaccine COVID-19

Immunosuppressants

$2,859

$727

lonapegsomatropin

elivaldogene tavalentivec (Lenti-D)

Endocrine

$589

Neurology/Gene therapy

$42

lenadogene nolparvovec (GS010) Ophthalmology/ Gene therapy

$39

inclisiran

Cardiovascular

ipatasertib

$693

Oncology

$510

pecialty drug names appear in ď&#x201A;Ť S magenta throughout the publication.

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PIPELINE DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2021. It is not intended to be a comprehensive inventory of all drugs in the pipeline; emphasis is placed on drugs in high-impact categories. Investigational drugs with a Complete Response Letter (CRL) and those that have been withdrawn from development are also noted.

APPLICATION APPLICATION SUBMITTED SUBMITTED TO THE FDA

IN PHASE PHASE 33 TRIALS TRIALS

61% 39% 39% 24% 20% 6%

Specialty

68% 32% 35 % 14% 8%

Traditional

Priority Review

Orphan Drug

Breakthrough Therapy

Biosimilar

Specialty drug names appear in ï&#x201A;« magenta throughout the publication.

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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) bevacizumab (biosimilar to Genentech’s Avastin)

Merck/Samsung Bioepis

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

IV

Submitted – BLA

viltolarsen

Nippon Shinyaku

DMD (exon 53 skipping)

IV

Submitted – NDA; Jul–Sep 2020 Fast Track; Orphan Drug; Priority Review; Rare Pediatric Disease

donepezil transdermal system

Corium

Alzheimer’s disease

Transdermal

Submitted – 505(b)(2) NDA

07/30/2020

viaskin peanut immunotherapy

DBV

Peanut allergy (ages 4 to 11 years)

Transdermal

Submitted – BLA; Breakthrough Therapy; Fast Track

08/05/2020

oliceridine

Trevena

Acute pain

IV

Submitted – NDA; Fast Track

08/07/2020

sodium thiosulfate

Fennec

Cisplatin-induced ototoxicity prevention

IV

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

08/10/2020

belantamab mafodotin

GlaxoSmithKline

Multiple myeloma (relapsed/refractory)

SC

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

08/14/2020

satralizumab

Genetech

Neuromyelitis optica (Devic’s syndrome)

SC

Submitted – BLA; Breakthrough Therapy; Orphan Drug

08/14/2020

filgotinib

Gilead

RA

Oral

Submitted – NDA; Priority Review

08/19/2020

valoctocogene roxaparvovec

Biomarin

Hemophilia A

IV

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

08/21/2020

veverimer

Tricida

CKD-related metabolic acidosis

Oral

Submitted – NDA; Accelerated Approval

08/22/2020

risdiplam

Genetech

Spinal muscular atrophy (types 1, 2, 3)

Oral

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

08/24/2020

bupivacaine collagen sponge

Innocoll

Postsurgical pain

Surgical implant

Submitted – NDA

08/26/2020

clascoterone

Cassiopea

Acne

Topical

Submitted – NDA

08/27/2020

tafasitamab

Morphosys

DLBCL (relapsed/ refractory, in combination with lenalidomide)

IV

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

08/28/2020

testosterone undecanoate

Lipocine

Hypogonadism

Oral

Submitted – 505(b)(2) NDA

08/28/2020

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Jul–Sep 2020

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

HIV therapeutic vaccine

Immune Response

HIV-1 infection treatment (pediatrics)

IM

Submitted – BLA; Orphan Drug

Sep–Dec 2020

azacitidine

Bristol-Myers Squibb

AML

Oral

Submitted – NDA; Priority Review

09/03/2020

terlipressin

Mallinckrodt

Hepatorenal syndrome type 1

IV

Submitted – NDA; Fast Track; Orphan Drug

09/12/2020

somapacitan

Novo Nordisk

Growth hormone deficiency (adults)

SC

Submitted – BLA

09/21/2020

diazepam film

Aquestive

Seizure clusters

Oral transmucosal

Submitted – 505(b)(2) NDA; Fast Track; Orphan Drug; Priority Review

09/27/2020

hydrocortisone granule

Eton

Adrenal insufficiency (ages birth to < 17 years)

Oral

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

09/29/2020

tramadol

Fortress

Pain (moderate to severe, medically supervised setting)

IV

Submitted – 505(b)(2) NDA

10/09/2020

zolmitriptan micro-needle patch

Zosano

Migraine treatment

Transdermal

Submitted – 505(b)(2) NDA

10/20/2020

eflapegrastim

Spectrum

Neutropenia/leukopenia

SC

Submitted – BLA

10/23/2020

REGN-EB3

Regeneron

Ebola virus infection treatment

IV

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

10/23/2020

loteprednol etabonate 0.25%

Kala

Dry eye disease

Ophthalmic

Submitted – 505(b)(2) NDA

10/30/2020

mannitol (dry powder)

Pharmaxis

CF (adults)

Inhaled

Submitted – NDA; Fast Track; Orphan Drug

10/30/2020

viloxazine

Supernus

ADHD

Oral

Submitted – NDA

11/06/2020

amphetamine sulfate IR (tamper resistant)

Arbor

ADHD (adults, pediatrics ages ≥ 3 years)

Oral

Submitted – NDA

11/13/2020

samidorphan/olanzapine

Alkermes

Bipolar disorder; Schizophrenia

Oral

Submitted – NDA

11/13/2020

sutimlimab

Sanofi

Cold agglutinin disease

IV

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

11/13/2020

naloxone single-dose prefilled syringe

US Worldmeds

Opioid overdose

IM

Submitted – 505(b)(2) NDA

11/15/2020

lisocabtagene maraleucel

Bristol-Myers Squibb

DLBCL (relapsed/ refractory)

IV

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

lonafarnib

Eiger

Hutchinson–Gilford progeria syndrome

Oral

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

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PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

pralsetinib

Blueprint Medicines

NSCLC (RET-fusion+)

Oral

Submitted – NDA; Orphan Drug; Priority Review

11/23/2020

treprostinil dry powder

Liquidia

PAH

Inhaled

Submitted – 505(b)(2) NDA

11/24/2020

setmelanotide

Rhythm

Obesity (POMC and LEPR deficiency)

SC

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

naxitamab

Y-mAbs

Neuroblastoma (relapsed/ IV refractory, high-risk)

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

11/30/2020

nifurtimox

Bayer

Chagas disease

Oral

Submitted – NDA; Orphan Drug

11/30/2020

inclisiran

The Medicines Company

Dyslipidemia (for secondary prevention in patients with ASCVD and FH)

SC

Submitted – NDA; Orphan Drug

December 2020

tanezumab

Pfizer

Osteoarthritis pain

IV

Submitted – BLA; Fast December Track 2020

berotralstat

Biocryst

Hereditary angioedema (attack prevention)

Oral

Submitted – NDA; Fast Track; Orphan Drug

lumasiran

Alnylam

Hyperoxaluria

SC

Submitted – NDA; 12/03/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease

margetuximab

Macrogenics

Breast cancer (HER2+, in combination with chemotherapy)

IV

Submitted – BLA; Fast 12/18/2020 Track

rituximab (biosimilar to Genentech’s Rituxan)

Amgen/Allergan

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

IV

Submitted – BLA

12/18/2020

roxadustat

AstraZeneca

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

Oral

Submitted – NDA

12/18/2020

relugolix

Myovant

Prostate cancer (advanced)

Oral

Submitted – NDA; Priority Review

12/20/2020

ansofaxine

Luye

MDD

Oral

Submitted – NDA

12/25/2020

bevacizumab (biosimilar to Genentech’s Avastin)

Mylan/Biocon

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

IV

Submitted – BLA

12/25/2020

vibegron

Urovant

Overactive bladder

Oral

Submitted – NDA

12/25/2020

arbaclofen ER

Osmotica

12/03/2020

MS-associated spasticity

Oral

Submitted – NDA

12/29/2020

furosemide wearable pump Scpharmaceuticals

Congestive heart failure

SC

Submitted – 505(b)(2) NDA

12/30/2020

tirbanibulin

Almirall

Actinic keratoses

Topical

Submitted – NDA

12/30/2020

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Merck/Fresenius

Neutropenia/leukopenia

SC

Submitted – BLA

Jan–Mar 2021

22 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

vericiguat

Merck

Chronic heart failure

Oral

Submitted – NDA; Priority Review

01/21/2021

voclosporin

Aurinia

Lupus nephritis

Oral

Submitted – NDA; Fast Track; Priority Review

01/22/2021

ropeginterferon alfa-2b

Pharmaessentia

Polycythemia vera (in absence of symptomatic splenomegaly)

SC

Submitted – BLA; Orphan Drug

Mar–Apr 2021

serdexmethylphenidate

Kempharm

ADHD

Oral

Submitted – 505(b)(2) NDA

03/02/2021

ponesimod

Janssen

MS (relapsing)

Oral

Submitted – NDA

03/18/2021

dasiglucagon

Zealand

Hypoglycemia (diabetesrelated)

SC

Submitted – NDA; Orphan Drug

03/27/2021

idecabtagene vicleucel

Bristol-Myers Squibb/ Bluebird Bio

Multiple myeloma (relapsed/refractory)

IV

Submitted – BLA; Breakthrough Therapy; Orphan Drug

03/31/2021

tivozanib

AVEO

RCC (relapsed/refractory)

Oral

Submitted – NDA

03/31/2021

aducanumab

Biogen

Alzheimer’s disease

IV

Submitted – BLA; Fast Apr–Jun 2021 Track

eflornithine/sulindac

Mallinckrodt

Familial adenomatous polyposis

Oral

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

Apr–Jun 2021

melflufen

Oncopeptides

Multiple myeloma (triple refractory)

IV

Submitted – NDA; Orphan Drug

Apr–Jun 2021

pegunigalsidase alfa

Chiesi

Fabry’s disease

IV

Submitted – BLA; Fast Apr–Jun 2021 Track

tralokinumab

AstraZeneca

Atopic dermatitis

SC

Submitted – BLA

Apr–Jun 2021

umbralisib

TG

Follicular lymphoma; Marginal zone lymphoma

Oral

Submitted – NDA; Breakthrough Therapy; Orphan Drug

Apr–Jun 2021

estetrol/drospirenone

Mayne

Contraception

Oral

Submitted – NDA

04/16/2021

relugolix/estradiol/ norethindrone

Myovant

Uterine fibroids-related Oral heavy menstrual bleeding

Submitted – NDA

06/01/2021

casimersen

Sarepta

DMD (amenable to exon 45 skipping)

IV

Submitted – NDA; Orphan Drug

06/25/2021

lonapegsomatropin

Ascendis

Growth hormone deficiency (pediatrics)

SC

Submitted – BLA; Orphan Drug

06/25/2021

pineapple proteolytic enzymes extract

Mediwound

Burn injury debridement

Topical

Submitted – BLA; Orphan Drug

06/30/2021

arimoclomol

Orphazyme

Niemann-Pick disease

Oral

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

Jul–Sep 2021

inolimomab

Elsalys

GVHD (acute, steroidrefractory)

IM

Submitted – BLA; Orphan Drug; RTOR

Jul–Sep 2021

pralsetinib

Blueprint Medicines

Thyroid cancer (RETfusion+)

Oral

Submitted – NDA; Breakthrough Therapy; RTOR

Jul–Sep 2021

taurolidine

Cormedix

Prevention of catheterrelated sepsis (hemodialysis patients)

IV

Submitted – NDA; Fast Track; QIDP

Jul–Sep 2021

23 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

avacopan

Chemocentryx

ANCA-associated vasculitis

Oral

Submitted – NDA; Orphan Drug

07/09/2021

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Kashiv

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

oxycodone ER

Intellipharmaceutics

Chronic pain

Oral

Submitted – 505(b)(2) NDA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

SC

Submitted – BLA

Pending

Submitted (Supplementals) omalizumab (Xolair )

Genentech

Nasal polyps

SC

Submitted – sBLA

Jul–Sep 2020

cannabidiol (Epidiolex )

GW

Tuberous sclerosis complex-related seizures

Oral

Submitted – sNDA; Orphan Drug; Priority Review

07/31/2020

esketamine (Spravato®)

Janssen

MDD (with suicidal ideation with intent)

Intranasal

Submitted – sNDA; Breakthrough Therapy; Fast Track

07/31/2020

fluticasone furoate/ umeclidinium bromide/ vilanterol (Trelegy® Ellipta®)

GlaxoSmithKline

Asthma (adults)

Inhaled

Submitted – sNDA

07/31/2020

ustekinumab (Stelara®)

Janssen

PSO (ages 6 to 11 years)

IV, SC

Submitted – sBLA

08/07/2020

dantrolene (Ryanodex®)

Eagle

Heat stroke (exertional)

IV

Submitted – sNDA; Fast Track; Orphan Drug

08/08/2020

dolutegravir/lamivudine (Dovato®)

GlaxoSmithKline

HIV-1 treatment (switch therapy)

Oral

Submitted – sNDA

08/16/2020

ofatumumab (Arzerra®)

Novartis

MS (relapsing)

SC

Submitted – sBLA; Priority Review

September 2020

ibrutinib (Imbruvica)

Abbvie

CLL/SLL (1st-line, ages < 70 years, in combination with rituximab)

Oral

Submitted – sNDA; Orphan Drug; RTOR

09/08/2020

cefiderocol (Fetroja®)

Shionogi

HAP

IV

Submitted – sNDA; Fast Track; Priority Review; QIDP

09/27/2020

linaclotide acetate (Linzess®)

Ironwood

IBS (treatment of abdominal symptoms)

Oral

Submitted – sNDA

October 2020

ticagrelor (Brilinta®)

AstraZeneca

Ischemic stroke

Oral

Submitted – sNDA; Priority Review

Oct–Nov 2020

ravulizumab-cwvz (Ultomiris®) at-home, weekly SC formulation

Alexion

Paroxysmal nocturnal hemoglobinuria

SC

Submitted – sBLA; Orphan Drug

10/09/2020

dolutegravir (Tivicay®) dispersible tablet

Viiv

HIV-1 treatment (pediatrics)

Oral

Submitted – sNDA

10/13/2020

pembrolizumab (Keytruda®)

Merck

Hodgkin’s lymphoma (classical, relapsed/ refractory, monotherapy)

IV

Submitted – sBLA; Breakthrough Therapy; Priority Review

10/30/2020

alectinib (Alecensa®)

Genentech

NSCLC (1st-line, ALK+)

Oral

Submitted – sNDA

November 2020

®

®

24 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

carfilzomib (Kyprolis®)

Amgen

Multiple myeloma (relapsed/refractory, in combination with daratumumab)

IV

Submitted – sBLA; Orphan Drug

11/13/2020

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

TherapeuticsMD

Menopause (including hormone replacement therapy [HRT])

Oral

Submitted – sNDA

11/16/2020

baloxavir marboxil (Xofluza®) oral granules, tablets

Genentech

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

Oral

Submitted – sNDA

11/23/2020

daratumumab (Darzalex®)

Janssen

Multiple myeloma (relapsed/refractory, in combination with carfilzomib)

IV

Submitted – sBLA; Orphan Drug

12/10/2020

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

Genentech

MS (relapsing and primary IV progressive)

Submitted – sBLA

12/14/2020

golimumab (Simponi Aria®)

Janssen

JIA; Juvenile psoriatic arthritis (jPsA)

IV

Submitted – sBLA

January 2021

mepolizumab (Nucala®)

GlaxoSmithKline

Hypereosinophilic syndrome (HES)

SC

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

January 2021

ivacaftor (Kalydeco®)

Vertex

CF (ages 4 to ≤ 6 months)

Oral

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

February 2021

peginterferon beta-1a (Plegridy®)

Biogen

MS (relapsing/remitting, active secondary progressive)

IM

Submitted – sBLA

February 2021

sacubitril/valsartan (Entresto®)

Novartis

Heart failure with preserved ejection fraction (HFpEF)

Oral

Submitted – sNDA

02/26/2021

selinexor (Xpovio®)

Karyopharm

Multiple myeloma (≥ 1 prior therapy)

Oral

Submitted – sNDA; Fast Track; Orphan Drug

03/19/2021

Abbvie

PsA

Oral

Submitted – sNDA

04/01/2021

pimavanserin (Nuplazid )

Acadia

Dementia-related hallucinations and delusions

Oral

Submitted – sNDA; Breakthrough Therapy

04/03/2021

tenapanor (Ibsrela®)

Ardelyx

Hyperphosphatemia (in CKD dialysis-dependent patients)

Oral

Submitted – sNDA

06/30/2021

upadacitinib (Rinvoq™) ®

Phase 3 (New Drugs) abaloparatide-TD

Radius

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

abametapir

Dr. Reddy’s

Head lice (aged ≥ 6 months)

Topical

Phase 3 – NDA

TBD

abrocitinib

Pfizer

Atopic dermatitis

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Coherus

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

SC

Phase 3 – BLA

TBD

25 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

adalimumab (biosimilar to Abbvie’s Humira)

Fresenius

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

SC

Phase 3 – BLA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Mylan/Biocon

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

SC

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea®)

Biogen/Samsung Bioepis

Diabetic macular edema

Intraocular

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Mylan/Biocon

Diabetic macular edema

Intraocular

Phase 3 – BLA

TBD

alicaforsen

Atlantic Healthcare

UC (pouchitis)

Rectal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

alpha 1 proteinase inhibitor

Kamada

Emphysema

Inhaled

Phase 3 – BLA; Orphan Drug

TBD

andolast

Mylan

Asthma

Inhaled

Phase 3 – NDA

TBD

anifrolumab

AstraZeneca

SLE

IV

Phase 3 – BLA; Fast Track

TBD

antolimab

Allakos

Gastroenteritis (eosinophilic esophagitis)

IV

Phase 3 – BLA; Orphan Drug

TBD

asciminib

Novartis

Chronic myelogenous leukemia (CML)

Oral

Phase 3 – NDA; 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

avalglucosidase alfa

Sanofi

Pompe disease

IV

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

AZD-1222 vaccine (formerly ChAdOx1-S)

AstraZeneca/University of Oxford

COVID-19

IM

Phase 3 – BLA

TBD

baclofen/naltrexone/ sorbitol

Pharnext

Charcot-Marie-Tooth disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

balixafortide

Polyphor

Breast cancer

IV

Phase 3 – NDA; Fast Track

TBD

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

Thalassemia

IV

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

TBD

bevacizumab

Outlook

Wet AMD

Intraocular

Phase 3 – BLA

TBD

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera

CRC; Glioblastoma; NSCLC; Ovarian cancer; RCC

IV

Phase 3 – BLA

TBD

bevacizumab (biosimilar to Genentech’s Avastin)

Boehringer Ingelheim

CRC; Glioblastoma; NSCLC; Ovarian cancer; RCC

IV

Phase 3 – BLA

TBD

bevacizumab (biosimilar to Genentech’s Avastin)

Kyowa Kirin/AstraZeneca

CRC; Glioblastoma; NSCLC; Ovarian cancer; RCC

IV

Phase 3 – BLA

TBD

bexagliflozin

Theracos

T2DM

Oral

Phase 3 – NDA

TBD

bimekizumab

UCB

Axial spondyloarthritis; Hidradenitis suppurativa; PsA; PSO

IV

Phase 3 – BLA

TBD

BNT162 vaccine (multiple variants)

Biontech/Pfizer

COVID-19

IM

Phase 3 – BLA

TBD

26 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

budenoside

Calliditas

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

Oral

Phase 3 – NDA; Orphan Drug

TBD

cabotegravir long-acting

Viiv

HIV-1 infection preexposure prevention (PrEP)

IM

Phase 3 – NDA

TBD

calmangafodipir

Pledpharma

Chemotherapy-induced peripheral neuropathy

IV

Phase 3 – NDA

TBD

cannabidiol gel

Zynerba

Fragile X syndrome

Transdermal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

capsaicin

Centrexion

Osteoarthritis

Intraarticular

Phase 3 – NDA; Fast Track

TBD

carglumic acid

Recordati

Hyperammonaemia (autosomal disorderrelated)

Oral

Phase 3 – NDA; Orphan Drug

TBD

CD24Fc

Oncoimmune

COVID-19

IV

Phase 3 – BLA

TBD

cedazuridine/decitabine

Otsuka

AML

Oral

Phase 3 – NDA

TBD

cediranib

AstraZeneca

Ovarian cancer

Oral

Phase 3 – NDA; Orphan Drug

TBD

ceftobiprole medocaril

Basilea

ABSSSI; Bacteremia; CABP; IV HAP

Phase 3 – NDA; Fast Track; QIDP

TBD

cenicriviroc mesylate

Allergan

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

ciltacabtagene autoleucel

Janssen

Multiple myeloma

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

clindamycin phosphate gel

Daré

Bacterial vaginosis

Intravaginal

Phase 3 – NDA; QIDP

TBD

CM-AT

Curemark

Autism spectrum disorder

Oral

Phase 3 – BLA; Fast Track

TBD

conbercept

Chengdu Kanghong

Wet AMD

Intraocular

Phase 3 – BLA

TBD

coronavac vaccine

Sinovac

COVID-19

IM

Phase 3 – BLA

TBD

COVID-19 inactivated vaccine (Vero cell)

Sinopharm/Beijing Institute

COVID-19

IM

Phase 3 – BLA

TBD

COVID-19 inactivated vaccine (Vero cell)

Sinopharm/Wuhan Institute

COVID-19

IM

Phase 3 – BLA

TBD

crisantaspase (recombinant)

Jazz

ALL

IM, IV

Phase 3 – BLA; Fast Track

TBD

dactolisib

Restorbio

COVID-19

Oral

Phase 3 – NDA

TBD

dalcetrapib

Dalcor

Dyslipidemia/ hypercholesterolemia

Oral

Phase 3 – NDA

TBD

daprodustat

GlaxoSmithKline

Anemia due to CKD (dialysis-independent/ dependent)

Oral

Phase 3 – NDA

TBD

daridorexant

Idorsia

Insomnia

Oral

Phase 3 – NDA

TBD

darvadstrocel

Takeda

CD

IV

Phase 3 – BLA; Orphan Drug

TBD

dehydrated human Mimedx amnion-chorion membrane

Achilles tendonitis; Plantar fasciitis

IV

Phase 3 – BLA

TBD

denosumab (biosimilar to Amgen’s Prolia®)

Novartis

Osteoporosis/osteopenia

SC

Phase 3 – BLA

TBD

deramanido

Otsuka

Tuberculosis

Oral

Phase 3 – NDA

TBD

27 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

dexmedetomidine

Bioxcel

Bipolar disorder; Schizophrenia

SL/Oral transmucosal

Phase 3 – NDA; Fast Track

TBD

dianhydrogalactitol

Delmar

Brain cancer

IV

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

difelikefalin

Enteris

Pruritus (hemodialysisrelated)

IV

Phase 3 – NDA; Breakthrough Therapy

TBD

digoxin immune Fab

AMAG

Eclampsia/pre-eclampsia

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

dociparstat sodium

Chimerix

COVID-19

IV

Phase 3 – NDA

TBD

docosahexaenoic acid

Micelle

Sickle cell disease

Oral

Phase 3 – NDA; Orphan Drug

TBD

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers; Diabetic peripheral neuropathy; Peripheral arterial disease

IM

Phase 3 – BLA; RMAT

TBD

dovitinib lactate

Oncology Venture

RCC

Oral

Phase 3 – NDA

TBD

dusquetide

Soligenix

Mucositis

IV

Phase 3 – NDA; Fast Track

TBD

dust mite immunotherapy

Stallergenes

Allergic rhinitis

SL/Oral transmucosal

Phase 3 – BLA

TBD

eculizumab (biosimilar to Alexion’s Soliris®)

Amgen

Paroxysmal nocturnal hemoglobinuria; Atypical hemolytic uremic syndrome

IV

Phase 3 – BLA

TBD

edasalonexent

Catabasis

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

efgartigimod

Argenx

ITP; Myasthenia gravis

IV, SC

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

efpeglenatide

Hanmi

T2DM

SC

Phase 3 – NDA

TBD

elafibranor

Genfit

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

elamipretide

Stealth Bio

Barth syndrome

IV, Oral, SC

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

elivaldogene tavalentivec (Lenti-D)

Bluebird Bio

Adrenomyeloneuropathy (adrenoleukodystrophy)

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

IV

Phase 3 – NDA; Fast Track; QIDP

TBD

entinostat

Syndax

Breast cancer

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

EP-2101 therapeutic vaccine

OSE Immunotherapeutics

NSCLC

SC

Phase 3 – NDA; Orphan Drug

TBD

eprenetapopt

Aprea

Myelodysplastic syndrome (tumor protein p53 mutation)

IV

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

TBD

erdosteine

Alitair

COPD

Oral

Phase 3 – NDA

TBD

estetrol

Mithra

Menopausal vasomotor symptoms

Oral

Phase 3 – NDA

TBD

28 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

etanercept (biosimilar to Amgen’s Enbrel)

Coherus

RA; JIA; AS; PSO; PsA

SC

Phase 3 – BLA

TBD

etranacogene dezaparvovec

Uniqure

Hemophilia B

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

etrasimod

Arena

UC

Oral

Phase 3 – NDA

TBD

etrolizumab

Genentech

CD; UC

SC

Phase 3 – BLA; Orphan Drug

TBD

evinacumab

Regeneron

Dyslipidemia/ hypercholesterolemia

SC

Phase 3 – BLA; Breakthrough Therapy

TBD

faricimab

Genentech

Diabetic macular edema; Wet AMD

Intraocular

Phase 3 – BLA

TBD

fasinumab

Regeneron

Osteoarthritis pain

SC

Phase 3 – BLA

TBD

fexapotide triflutate

Nymox

Benign prostatic hyperplasia

Intratumoral

Phase 3 – NDA

TBD

fezolinetant

Astellas

Menopausal vasomotor symptoms

Oral

Phase 3 – NDA

TBD

fidanacogene elaparvovec

Pfizer

Hemophilia B

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

PsA; CD; UC

Oral

Phase 3 – NDA

TBD

finerenone

Bayer

Diabetic nephropathy

Oral

Phase 3 – NDA

TBD

fitusiran

Sanofi

Hemophilia A and B (with and without inhibitors)

SC

Phase 3 – NDA; Orphan Drug

TBD

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

Allergan

Female reproductive disorder

SC

Phase 3 – BLA

TBD

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

Finox

Female reproductive disorder

SC

Phase 3 – BLA

TBD

follitropin delta

Ferring

Female infertility

IV

Phase 3 – BLA

TBD

fosdenopterin

Bridgebio

Molybdenum cofactor deficiency (MoCD)

IV

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

fusidic acid

Arrevus

ABSSSI

Oral

Phase 3 – NDA; Orphan Drug; QIDP

TBD

gefapixant

Merck

Chronic cough

Oral

Phase 3 – NDA

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3 – NDA; QIDP

TBD

givinostat

Italfarmaco

DMD

Oral

Phase 3 – NDA

TBD

glatiramer acetate depot

Mylan

MS

IM

Phase 3 – NDA

TBD

GLPG1690

Galapos

Idiopathic pulmonary fibrosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

guadecitabine

Otsuka

Myelodysplastic syndrome

SC

Phase 3 – NDA

TBD

hydrocortisone granules

Diurnal

Congenital adrenal hyperplasia

Oral

Phase 3 – NDA; Orphan Drug

TBD

hypericin

Soligenix

Cutaneous T-cell lymphoma (CTCL)

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

29 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ibrexafungerp

Scynexis

Fungal infections (systemic and nonsystemic)

IV, Oral

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

TBD

iclaprim

Motif Bio

ABSSSI; HAP

IV

Phase 3 – NDA; Fast Track; QIDP

TBD

idasanutlin

Genentech

AML

Oral

Phase 3 – NDA

TBD

idebenone

Santhera

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

idursulfase

Takeda

Mucopolysaccharidosis II Intrathecal (MPS II; Hunter syndrome)

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

immunoglobulin IV 10%

Prometic Life Sciences

Primary immunodeficiencies

IV

Phase 3 – BLA

TBD

infigratinib

Bridgebio

Biliary tract cancer

Oral

Phase 3 – NDA; Fast Track

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; PsA; CD; UC

IV

Phase 3 – BLA

TBD

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

Mylan/Biocon

T1DM; T2DM

SC

Phase 3 – BLA

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Sanofi

T1DM; T2DM

SC

Phase 3 – BLA

TBD

insulin glargine (biosimilar to Sanofi’s Lantus®)

Gan & Lee

T1DM; T2DM

SC

Phase 3 – BLA

TBD

iodine I-131 monoclonal antibody

Actinium

Myeloablation prior to allogeneic HSCT to treat AML

IV

Phase 3 – BLA; Orphan Drug

TBD

ipatasertib

Genentech

Breast cancer; Prostate cancer

Oral

Phase 3 – NDA

TBD

L-citrulline

Asklepion

Acute lung injury

IV

Phase 3 – NDA; Orphan Drug

TBD

lebrikizumab

Dermira

Atopic dermatitis

SC

Phase 3 – BLA; Fast Track

TBD

lenadogene nolparvovec (GS010)

Gensight

Leber’s hereditary optic neuropathy

Intraocular

Phase 3 – BLA; Orphan Drug

TBD

lenzilumab

Humanigen

COVID-19

IV

Phase 3 – BLA

TBD

leriglitazone

Minoryx

Adrenomyeloneuropathy (adrenoleukodystrophy)

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

leronlimab

Cytodyn

COVID-19

IV, SC

Phase 3 – BLA

TBD

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

SC

Phase 3 – NDA

TBD

levoketoconazole

Strongbridge

Cushing’s syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

ligelizumab

Novartis

Urticaria

SC

Phase 3 – BLA

TBD

linzagolix

Obseva

Endometriosis; Uterine fibroids

Oral

Phase 3 – NDA

TBD

lonafarnib

Eiger

Hepatitis D infection

Oral

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

TBD

lorecivivint

Samumed

Osteoarthritis (knee)

Intraarticular

Phase 3 – NDA

TBD

lutetium 177Lu-PSMA-617

Novartis

Prostate cancer

IV

Phase 3 – NDA

TBD

30 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

LYS-SAF302

Sarepta

Mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo A syndrome)

Intracerebral

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

maribavir

Takeda

Cytomegalovirus infection treatment

Oral

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

TBD

marstacimab

Pfizer

Hemophilia A and B

SC

Phase 3 – BLA; Orphan Drug

TBD

masitinib mesylate

AB Science

Asthma (eosinophilic); Alzheimer’s disease; Amyotrophic lateral sclerosis; Mastocytosis; MS (primary progressive, non-active secondary)

Oral

Phase 3 – NDA; Orphan Drug

TBD

mavacamten

Myokardia

Obstructive hypertrophic cardiomyopathy

Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

meloxicam/rizatriptan

Axsome

Migraine treatment

Oral

Phase 3 – NDA

TBD

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

IV

Phase 3 – BLA; Orphan Drug

TBD

microbiota suspension

Ferring

C. difficile infection (recurrent)

Rectal

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

TBD

mirikizumab

Eli Lilly

PSO; CD; UC

IV, SC

Phase 3 – BLA

TBD

mirvetuximab soravtansine

Immunogen

Ovarian cancer

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

mitapivat

Agios

Pyruvate kinase deficiency

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

molgramostim

Savara

Pulmonary alveolar proteinosis

Inhaled

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

TBD

mRNA-1273 vaccine

Moderna/NIAID

COVID-19

IM

Phase 3 – BLA; Fast Track

TBD

nadofaragene firadenovec

Trizell

Mesothelioma

Percutaneous Catheter/ Injection

Phase 3 – BLA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 – NDA

TBD

napabucasin

Sumitomo Dainippon

CRC

Oral

Phase 3 – NDA

TBD

narsoplimab

Omeros

HSCT-associated thrombotic microangiopathy

SC

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

natalizumab (biosimilar to Biogen’s Tysabri®)

Novartis

MS

IV

Phase 3 – BLA

TBD

nemolizumab

Galderma

Atopic dermatitis

SC

Phase 3 – BLA

TBD

nirsevimab

AstraZeneca

RSV infection prevention

N/A

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

31 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

olipudase alfa

Sanofi

Niemann-Pick disease

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

omega-3 phospholipid (krill oil)

Akcea

Hypertriglyceridemia (severe)

N/A

Phase 3 – NDA

TBD

ondansetron ER once-daily

Redhill

Gastroenteritis

Oral

Phase 3 – NDA

TBD

oportuzumab monatox

Sesen Bio

Bladder cancer (BCGunresponsive, nonmuscle invasive)

Intravesical

Phase 3 – BLA; Fast Track

TBD

OTL-103

Orchard

Wiskott-Aldrich syndrome IV

Phase 3 – BLA; Orphan Drug; RMAT

TBD

oxalobacter formigenes

Oxthera

Hyperoxaluria

Oral

Phase 3 – BLA; Orphan Drug

TBD

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

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

TBD

pamrevlumab

Fibrogen

Idiopathic pulmonary fibrosis

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

pegcetacoplan

Apellis

Paroxysmal nocturnal hemoglobinuria

Intraocular

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

pegzilarginase

Aeglea

Arginase 1 deficiency

IV

Phase 3 – BLA; Breakthrough Therapy

TBD

pimecrolimus

Bausch Health

Atopic dermatitis

Topical

Phase 3 – NDA

TBD

pimodivir

Janssen

Influenza treatment

Oral

Phase 3 – NDA; Fast Track

TBD

plasminogen (human)

Liminal

Hypoplasminogenemia

IV

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

plinabulin

BeyondSpring

Neutropenia/leukopenia; NSCLC

IV

Phase 3 – NDA

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

SC

Phase 3 – BLA

TBD

ranibizumab (biosimilar to Genentech’s Lucentis®)

Biogen/Samsung Bioepis

Wet AMD

Intraocular

Phase 3 – BLA

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Coherus/Santo

Wet AMD

Intraocular

Phase 3 – BLA

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

STADA Arzneimittel/ Bausch Health

Wet AMD

Intraocular

Phase 3 – BLA

TBD

REGN-COV2

Regeneron

COVID-19

N/A

Phase 3 – BLA

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

reltecimod

Atox

Necrotizing soft tissue infection

IV

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

relugolix (± estradiol/ norethindrone)

Myovant

Endometriosis

Oral

Phase 3 – NDA

TBD

remdesivir

Gilead

COVID-19

Inhaled, IV

Phase 3 – NDA

TBD

reproxalap

Aldeyra

Congenital ichthyosis

Topical

Phase 3 – NDA; Orphan Drug

TBD

resmetirom

Madrigal

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

32 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

rigosertib

Onconova

Myelodysplastic syndrome

IV

Phase 3 – NDA; Orphan Drug

TBD

rituximab (biosimilar to Genentech’s Rituxan)

Archigen

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

IV

Phase 3 – BLA

TBD

rivoceranib

LSK Biopartners

Gastric cancer

Oral

Phase 3 – NDA; Orphan Drug

TBD

rozanolixizumab

UCB

ITP; Myasthenia gravis

SC

Phase 3 – BLA

TBD

RSV nanoparticle vaccine

Novavax

RSV infection prevention

IM

Phase 3 – BLA; Fast Track

TBD

ruxolitinib cream

Incyte

Atopic dermatitis; Vitiligo

Topical

Phase 3 – sNDA

TBD

savolitinib

AstraZeneca

RCC

Oral

Phase 3 – NDA

TBD

seladelpar

Cymabay

Primary biliary cholangitis/hepatic fibrosis

Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

sepofarsen

Proqr

Leber’s congenital amaurosis

Intraocular

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

setmelanotide

Rhythm

Alström syndrome; Bardet-Biedl syndrome

SC

Phase 3 – NDA; Orphan Drug

TBD

sodium hyaluronate/ triamcinolone hexacetonide

Anika

Osteoarthritis (knee)

Intraarticular

Phase 3 – NDA

TBD

sodium oxybate oncenightly dosing

Avadel

Narcolepsy

Oral

Phase 3 – 505(b)(2) NDA; Orphan Drug

TBD

sofpironium bromide

Brickell

Axillary hyperhidrosis

Topical

Phase 3 – NDA

TBD

somatrogon

Opko

Growth hormone deficiency (pediatric)

SC

Phase 3 – BLA; Orphan Drug

TBD

sotagliflozin

Lexicon

T2DM; CVD

Oral

Phase 3 – NDA

TBD

sparsentan

Retrophin

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

Oral

Phase 3 – NDA; Orphan Drug

TBD

spartalizumab

Novartis

Melanoma

IV

Phase 3 – BLA

TBD

sulopenem etzadroxil

Iterum

Intra-abdominal infections; UTI (uncomplicated)

IV, Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

tabelecleucel

Atara

Epstein-Barr virus-associated post-transplant lymphoproliferative disease

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

tanezumab

Pfizer

Cancer pain; Chronic low back pain

SC

Phase 3 – BLA; Fast Track

TBD

tapinarof

Roivant

PSO

Topical

Phase 3 – NDA

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 – NDA

TBD

teplizumab

Provention Bio

T1DM (prevention or delay)

IV

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

33 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

teprasiran

Quark

Delayed graft function; Kidney injury prevention following cardiac surgery

IV

Phase 3 – NDA

TBD

tezepelumab

AstraZeneca

Asthma (severe, uncontrolled)

SC

Phase 3 – BLA; Breakthrough Therapy

TBD

timbetasin

Regenerx

Dry eye syndrome; Neurotrophic keratitis

Topical

Phase 3 – BLA; Orphan Drug

TBD

timrepigene emparvovec

Biogen

Choroideremia

Intraocular

Phase 3 – BLA; Orphan Drug; RMAT

TBD

tirzepatide

Eli Lilly

T1DM

SC

Phase 3 – NDA

TBD

tivanisiran

Sylentis

Dry eye syndrome

Topical

Phase 3 – NDA

TBD

tofersen

Biogen

Amyotrophic lateral sclerosis

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tominersen

Genentech

Huntington’s disease

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tonogenchoncel-L

Kolon Tissuegene

Osteoarthritis

Intraarticular

Phase 3 – BLA

TBD

trabedersen

Autotelic

Brain cancer (malignant glioma; glioblastoma)

IV, Intratumoral

Phase 3 – NDA; Orphan Drug

TBD

tradipitant

Vanda

Atopic dermatitis; COVID-19; Gastroparesis; Pruritus

Oral

Phase 3 – NDA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Novartis

Breast cancer; Gastric/ gastroesophageal cancer

IV

Phase 3 – BLA

TBD

trastuzumab (biosimilar to Genentech’s Herceptin)

Tanvex

Breast cancer; Gastric/ gastroesophageal cancer

IV

Phase 3 – BLA

TBD

trastuzumab duocarmazine

Synthon Bio

Breast cancer

IV

Phase 3 – BLA; Fast Track

TBD

tripotassium citrate monohydrate/potassium hydrogen carbonate microtablet

Advicenne

Renal tubular acidosis

Oral

Phase 3 – NDA

TBD

trivalent hepatitis B vaccine

VBI Vaccines

Hepatitis B (HBV) prevention

IM

Phase 3 – BLA

TBD

trofinetide

Acadia

Rett syndrome

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ublituximab

TG

CLL/SLL; MS

IV

Phase 3 – BLA; Orphan Drug

TBD

udenafil

Allergan

Congenital single ventricle heart disease (adolescents)

Oral

Phase 3 – NDA; Orphan Drug

TBD

umbralisib

TG

CLL/SLL; DLBCL; Mantle cell lymphoma

Oral

Phase 3 – NDA; Orphan Drug

TBD

vadadustat

Akebia

Anemia due to CKD (dialysis-independent/ dependent)

Oral

Phase 3 – NDA

TBD

vazegepant

Biohaven

Migraine treatment; COVID-19

Intranasal, Oral

Phase 3 – NDA

TBD

veliparib

Abbvie

Breast cancer; Ovarian cancer

Oral

Phase 3 – NDA

TBD

VGX-3100

Inovio

Cervical dysplasia

IM

Phase 3 – BLA

TBD

vilanterol trifenatate

GlaxoSmithKline

Asthma; COPD

Inhaled

Phase 3 – NDA

TBD

34 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

vocimagene amiretrorepvec

Tocen

Brain cancer (malignant glioma; glioblastoma)

Intratumoral

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

TBD

vonoprazan fumarate

Phathom

H. pylori infection; Esophagitis

Oral

Phase 3 – NDA; QIDP

TBD

vosoritide

Biomarin

Achondroplasia

SC

Phase 3 – NDA; Orphan Drug

TBD

vutrisiran

Alnylam

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

SC

Phase 3 – NDA; Orphan Drug

TBD

zilucoplan

Ra

Myasthenia gravis

SC

Phase 3 – NDA; Orphan Drug

TBD

zoliflodacin

Entasis

Gonorrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

Phase 3 (Supplementals) ado-trastuzumab emtansine (Kadcyla)

Genentech

Breast cancer (HER2+, adjuvant, with pertuzumab)

IV

Phase 3 – sBLA; Breakthrough Therapy

TBD

albuterol (ProAir® RespiClick®)

Teva

COPD

Inhaled

Phase 3 – sNDA

TBD

albutrepenonacog alfa (Idelvion®)

CSL

Hemophilia B (21-day dosing schedule)

IV

Phase 3 – sBLA; Orphan Drug

TBD

anakinra (Kineret®)

Swedish Orphan Biovitrum

COVID-19

SC

Phase 3 – sBLA

TBD

apalutamide (Erleada®)

Janssen

Prostate cancer (localized)

Oral

Phase 3 – sNDA

TBD

atezolizumab (Tecentriq®)

Genentech

Bladder cancer (1st-line metastatic); Bladder cancer (adjuvant, muscleinvasive); Breast cancer (TNBC, neoadjuvant, with nabpaclitaxel); Breast cancer (1st-line, TNBC, with paclitaxel); Melanoma; Ovarian cancer

IV

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

avatrombopag (Doptelet®)

Ararx

Thrombocytopenia (chemotherapy-induced)

Oral

Phase 3 – sNDA; Orphan Drug

TBD

baricitinib (Olumiant®)

Eli Lilly

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

Oral

Phase 3 – sNDA; Breakthrough Therapy; Fast Track

TBD

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

Merck

COVID-19

Intradermal

Phase 3 – sBLA

TBD

belimumab (Benlysta®)

GlaxoSmithKline

ANCA-associated IV vasculitis; Lupus nephritis

Phase 3 – sBLA; Breakthrough Therapy

TBD

benralizumab (Fasenra®)

AstraZeneca

Nasal polyps

SC

Phase 3 – sBLA

TBD

brexpiprazole (Rexulti )

Otsuka

Alzheimer’s diseaserelated agitation; Bipolar disorder; PTSD

Oral

Phase 3 – sNDA; Fast Track

TBD

®

35 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

cabozantinib (Cabometyx®)

Exelixis

RCC (1st-line, with nivoluab)

Oral

Phase 3 – sNDA

TBD

canakinumab (Ilaris®)

Novartis

COVID-19

IV

Phase 3 – sBLA

TBD

cannabidiol (Epidiolex)

GW

Rett syndrome

Oral

Phase 3 – sNDA

TBD

dapagliflozin (Farxiga®)

AstraZeneca

COVID-19; CKD; Diabetic nephropathy; Post MI

Oral

Phase 3 – sNDA; Fast Track

TBD

daratumumab-rHuPH20 (Darzalex Faspro™)

Janssen

Amyloidosis

SC

Phase 3 – sBLA

TBD

dupilumab (Dupixent®)

Sanofi

Atopic dermatitis (ages 6 months to 5 years); Bullous pemphigoid; COPD; Esophagitis; Pruritus; Urticaria

SC

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

durvalumab (Imfinzi®)

AstraZeneca

Bladder cancer (with tremelimumab); NSCLC (1st-line, with tremelimumab); NSCLC (neoadjuvant)

IV

Phase 3 – sBLA; Breakthrough Therapy; Fast Track

TBD

empagliflozin (Jardiance®)

Boehringer Ingelheim

Chronic heart failure; Diabetic nephropathy

Oral

Phase 3 – sNDA; Fast Track

TBD

fenfluramine (Fintepla®)

Zogenix

Lennox-Gastaut syndrome

Oral

Phase 3 – sNDA; Orphan Drug

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 – sNDA

TBD

immunoglobulin 5% (Octagam®)

Octapharma

COVID-19

IV

Phase 3 – sBLA

TBD

lacosamide (Vimpat®)

UCB

Primary generalized tonic-clonic seizures (adjunctive treatment)

IV, Oral

Phase 3 – sNDA

TBD

lumateperone (Caplyta®)

Intra-Cellular Therapies

Bipolar disorder

Oral

Phase 3 – sNDA

TBD

mepolizumab (Nucala)

GlaxoSmithKline

COPD; Nasal polyps

SC

Phase 3 – sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

IV

Phase 3 – sNDA; QIDP TBD

niraparib (Zejula®)

GlaxoSmithKline

Ovarian cancer (in combination with dostarlimab)

Oral

Phase 3 – sNDA; Orphan Drug

TBD

nitazoxanide (Alinia®)

Lupin

COVID-19

Oral

Phase 3 – sNDA

TBD

olaparib (Lynparza )

AstraZeneca

Breast cancer (metastatic, adjuvant treatment); Ovarian cancer (gBRCAm, 3rd-line); Ovarian cancer (2nd-line, in combination with cediranib)

Oral

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

omalizumab (Xolair)

Genentech

Food allergies

SC

Phase 3 – sBLA; Breakthrough Therapy

TBD

onasemnogene abeparvovac-xioi (Zolgensma®)

Novartis

Spinal muscular atrophy (type 2, 3)

IV, Intrathecal

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

TBD

ozanimod (Zeposia®)

Celgene

CD; UC

Oral

Phase 3 – sNDA

TBD

®

36 | magellanrx.com

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

paliperidone (Invega Sustenna®) 6-month injectable

Janssen

Schizophrenia

IM

Phase 3 – sNDA

TBD

patisiran (Onpattro®)

Alnylam

Transthyretin amyloid cardiomyopathy (wild type or hereditary)

IV

Phase 3 – sNDA

TBD

pertuzumab (Perjeta®)

Genentech

Breast cancer (HER2+, adjuvant, with adotrastuzumab)

IV

Phase 3 – sBLA

TBD

pirfenidone (Esbriet®)

Genentech

Idiopathic pulmonary fibrosis (unclassifiable interstitial lung disease [uILD])

Oral

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

TBD

polatuzumab vedotin-piiq (Polivy)

Genentech

DLBLC (1st-line)

IV

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

prasterone (Intrarosa®)

AMAG

Female sexual arousal disorder

Intravaginal

Phase 3 – sNDA

TBD

ravulizumab-cwvz (Ultomiris)

Alexion

COVID-19

IV

Phase 3 – sBLA

TBD

rilonacept (Arcalyst®)

Regeneron

Recurrent pericarditis

SC

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

risankizumab-rzaa (Skyrizi®)

Abbvie

PsA; CD; UC

SC

Phase 3 – sBLA; Orphan Drug

TBD

ruxolitinib (Jakafi®)

Incyte

COVID-19

Oral

Phase 3 – sNDA

TBD

sacubitril/valsartan (Entresto)

Novartis

Post-acute MI

Oral

Phase 3 – sNDA; Fast Track

TBD

Sanofi

COVID-19

SC

Phase 3 – sBLA

TBD

secnidazole (Solosec )

Lupin

Trichomoniasis

Oral

Phase 3 – sNDA

TBD

selinexor (Xpovio)

Karyopharm

Liposarcoma

Oral

Phase 3 – sNDA; Orphan Drug

TBD

semaglutide (Ozempic®)

Novo Nordisk

Diabetic nephropathy; Diabetic retinopathy; Obesity

SC

Phase 3 – sNDA

TBD

tecovirimat (Tpoxx®)

SIGA

Smallpox

IV

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

teriflunomide (Aubagio®)

Sanofi

MS (pediatrics)

Oral

Phase 3 – sNDA

TBD

ticagrelor (Brilinta)

AstraZeneca

Sickle cell disease

Oral

Phase 3 – sNDA

TBD

tisagenlecleucel-t (Kymriah)

Novartis

DLBCL (1st relapse)

IV

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

tocilizumab (Actemra®)

Genentech

COVID-19

IV

Phase 3 – sBLA

TBD

upadacitinib (Rinvoq)

Abbvie

Atopic dermatitis; Axial spondyloarthritis; CD; UC; Giant cell arteritis

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

Janssen

SLE

IV, SC

Phase 3 – sBLA

TBD

Beigene

Waldenstrom macroglobulinemia

Oral

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

sarilumab (Kevzara®) ®

ustekinumab (Stelara) zanubrutinib (Brukinsa ) ®

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PIPELINE DRUG LIST continued

Complete Response Letter (CRL)/Withdrawn Drugs NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

abicipar pegol

Allergan

Wet AMD

Intraocular

CRL

TDB

avapritinib (Ayvakit®)

Blueprint Medicines

GIST (4th-line)

Oral

CRL

TDB

bupivacaine/meloxicam

Heron

Postsurgical pain

Instillation

CRL

TDB

cantharidin 0.7% drugdevice

Verrica

Molluscum contagiosum

Topical

CRL

TDB

dasotraline

Sumitomo Dainippon

Binge eating disorder

Oral

Withdrawn

N/A

fosfomycin

Nabriva

UTI (complicated)

IV

CRL

TDB

lenvatinib (Lenvima )

Eisai

HCC (1st-line, unresectable, in combination with pembrolizumab)

Oral

CRL

TDB

nadofaragene firadenovec

FKD

Bladder cancer (highgrade, BCG-unresponsive, non-muscle invasive)

Intravesical

CRL

TDB

obeticholic acid (Ocaliva®)

Intercept

NASH

Oral

CRL

TDB

oxycodegol

Nektar

Chronic low back pain

Oral

Withdrawn

N/A

pembrolizumab (Keytruda)

Merck

HCC (1st-line, unresectable, in combination with lenvatinib)

IV

CRL

TDB

rimegepant tablet

Biohaven

Migraine treatment

Oral

Withdrawn

N/A

treprostinil disposible patch pump

United Therapeutics

PAH

SC

CRL

TDB

®

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GLOSSARY ABSSSI Acute Bacterial Skin and Skin Structure Infection ACR20 American College of Rheumatology 20% Improvement ACR50 American College of Rheumatology 50% Improvement ACR70 American College of Rheumatology 70% Improvement ADHD Attention Deficit Hyperactivity Disorder ADL Activities of Daily Living AED Anti-Epileptic Drug ALK Anaplastic Lymphoma Kinase ALL Acute Lymphoblastic Leukemia ALT Alanine Transaminase AMD Age-Related Macular Degeneration AML Acute Myeloid Leukemia ANCA Antineutrophil Cytoplasmic Antibodies ANDA Abbreviated New Drug Application ART Antiretroviral Therapy ARV Antiretroviral AS Ankylosing Spondylitis ASCVD Atherosclerotic Cardiovascular Disease AST Aspartate Aminotransferase BLA Biologics License Application BsUFA Biosimilar User Fee Act BMI Body Mass Index

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

BCVA Best Corrected Visual Acuity

FACIT Functional Assessment of Chronic Illness Therapy

CABP Community Acquired Bacterial Pneumonia

FDA Food and Drug Administration

CAP Community Acquired Pneumonia

FH Familial Hypercholesterolemia

CD Crohn's Disease

FLT3 FMS-Like Tyrosine Kinase-3

CDC Centers for Disease Control and Prevention

GI Gastrointestinal

CF Cystic Fibrosis

GIST Gastrointestinal Stromal Tumor

CHF Congestive Heart Failure

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

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GLOSSARY continued GVHD Graft Versus Host Disease

MRI Magnetic Resonance Imaging

H Half

MRSA Methicillin-Resistant Staphylococcus Aureus

HAM-D Hamilton Depression Rating Scale

MS Multiple Sclerosis

HAP Healthcare-Associated Pneumonia

N/A Not Applicable

Hb Hemoglobin

NASH Non-Alcoholic Steatohepatitis

HbA1c Hemoglobin A1c

NDA New Drug Application

HCC Hepatocellular Carcinoma

NHL Non-Hodgkin Lymphoma

HCP Healthcare Professional

NIAID National Institute of Allergy and Infectious Diseases

HCV Hepatitis C Virus

NSAID Non-Steroidal Anti-Inflammatory Drug

HER Human Epidermal Growth Factor Receptor

NSCLC Non-Small Cell Lung Cancer

HER2 Human Epidermal Growth Factor Receptor 2

ODT Orally Disintegrating Tablet

HFA Hydrofluoroalkane

ORR Overall/Objective Response Rate

HIT Heparin Induced Thrombocytopenia

OR Odds Ratio

HIV Human Immunodeficiency Virus

OS Overall Survival

HIV-1 Human Immunodeficiency Virus-1

PAH Pulmonary Arterial Hypertension

HR Hazard Ratio

PARP Poly (ADP-ribose) polymerase

HSCT Hematopoietic Stem Cell Transplant

PASI 50 Psoriasis Area and Severity Index ≥ 50%

HTN Hypertension

PASI 70 Psoriasis Area and Severity Index ≥ 70%

IBS Irritable Bowel Syndrome

PASI 90 Psoriasis Area and Severity Index ≥ 90%

IBS-C Irritable Bowel Syndrome, Constipation Predominant

PCI Percutaneous Coronary Intervention

IM Intramuscular ITP Immune Thrombocytopenic Purpura ITT Intent-To-Treat IV Intravenous JIA Juvenile Idiopathic Arthritis LDL-C Low-Density Lipoprotein Cholesterol MACE Major Adverse Cardiovascular Events MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler

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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 PTSD Post-Traumatic Stress Disorder Q Quarter QIDP Qualified Infectious Diseases Product

GLOSSARY continued QOL Quality of Life

TBD To Be Determined

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

TEAE Treatment-Emergent Adverse Events

RA Rheumatoid Arthritis RBC Red Blood Cell RCC Renal Cell Carcinoma REMS Risk Evaluation and Mitigation Strategy RMAT Regenerative Medicine Advanced Therapy RNA Ribonucleic Acid RRR Relative Risk Reduction RSV Respiratory Syncytial Virus RTOR Real-Time Oncology Review sBLA supplemental Biologics License Application SC Subcutaneous SCCHN Squamous Cell Cancer of the Head and Neck SCLC Small Cell Lung Cancer SCT Stem Cell Transplant SGLT Sodium-Glucose Co-Transporter SL Sublingual SLE Systemic Lupus Erythematosus SLL Small Lymphocytic Lymphoma sNDA supplemental New Drug Application SOC Standard of Care sPGA Static Physician Global Assessment SR Sustained-Release SNRI Serotonin and Norepinephrine Reuptake Inhibitor SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection T1DM Type 1 Diabetes Mellitus T2DM Type 2 Diabetes Mellitus

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TNBC Triple Negative Breast Cancer TNF Tumor Necrosis Factor TNFα Tumor Necrosis Factor-alpha UA Unstable Angina UC Ulcerative Colitis US United States UTI Urinary Tract Infection VAS Visual Analog Scale VEGF Vascular Endothelial Growth Factor VTE Venous Thromboembolism WBC White Blood Cell WHO World Health Organization XR Extended-Release

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