MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS JANUARY 2021
EDITORIAL STAFF Maryam Tabatabai, PharmD Editor-in-Chief Vice President, Clinical Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist Consultant Panel Michelle Booth, PharmD Director, Medical Pharmacy Strategy Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist Robert Greer, RPh, BCOP Senior Director, Clinical Strategy and Programs Katie Lockhart Manager, Forecasting and Pharmacoeconomics Brian MacDonald, PharmD Senior Manager, Specialty Clinical Programs
Table of CONTENTS
Troy Phelps Senior Director, COAR - Analytics
EDITOR-IN-CHIEF'S MESSAGE
2
PIPELINE DEEP DIVE
3
KEEP ON YOUR RADAR
20
PIPELINE DRUG LIST
21
GLOSSARY
41
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Nothing herein is or shall be construed as a promise or representation regarding past or future events and Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Magellan Rx Management.
Editor-in-Chief's MESSAGE Welcome to the MRx Pipeline. This quarterly publication offers clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline. MRx Pipeline, our universal forecast, addresses trends applicable across market segments. Traditional and specialty drugs as well as agents under the pharmacy and medical benefits are featured. Also profiled in the report are new molecular entities, pertinent new and expanded indications for existing medications, and biosimilars. Clinical analyses, financial outlook, and pre-regulatory status are considered. The products housed in the MRx Pipeline have been researched in detail. They have been developed in consultation with our internal team of clinical and analytics experts.
METHODOLOGY
Emerging therapeutics continue to grow and influence the clinical and financial landscape. Therefore, Magellan Rx Management has developed a systematic approach to determine the products with significant clinical impact. For the in-depth clinical evaluations, the products’ potential to meet an underserved need in the market by becoming the new standard of care, and the ability to replace existing therapies were investigated. The extent to which the pipeline drugs could shift market share on a formulary and their impact on disease prevalence were also important considerations. In order to assist payers with assessing the potential impact of these pipeline drugs, where available, a financial forecast has been included for select products. Primarily complemented by data from EvaluateTM, this pipeline report looks ahead at the 5-year projected annual US sales through the year 2025. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, including the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business.
LOOKING BACK
Despite the pandemic's unprecedented challenges, in 2020, the US FDA approved 53 novel drugs, making it the second highest number of approvals in 10 years. Notably, 58% of these approvals were for patients with Orphan conditions. Of note, the all-time record approvals was in 2018 with 59 novel drugs approved. Furthermore, the last 3 years share the distinction of having the highest number of approvals in the last 10 years. Remarkably, in late 2020, two life-saving COVID-19 vaccines received Emergency Use Authorization (EUA). While numbers do not tell the entire story, they do represent incredible advances in patient care and hope for the American public.
ON THE HORIZON
As we look ahead, there is a continued trend toward the approval of specialty medications and drugs for rare and ultra rare diseases, with 61% and 34% of approvals expected, respectively, for agents with applications submitted to the FDA. The growth of biosimilars, new treatment modalities using gene therapy, and additional CAR T therapies are expected. A public health arsenal of COVID-19 therapeutics and vaccines, including a single-dose shot, will make 2021 a pivotal year in combating this deadly infection. Other noteworthy pipeline trends to watch include the development of complex therapies, oncology, immunology, therapeutic options for rare hereditary diseases, and a possible new therapy for Alzheimer’s disease. Moreover, sprouting products for hematology, ophthalmology, and diabetes await on the horizon. The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Innovative agents that show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm. Maryam Tabatabai, PharmD Editor-in-Chief, MRx Pipeline
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Pipeline DEEP DIVE Objective evidence-based methodology was used to identify the Deep Dive drugs in the upcoming quarters. This section features a clinical overview and explores the potential place in therapy for these agents. Moreover, it addresses their FDA approval timeline and 5-year financial forecast.
SPECIALTY
PRIORITY REVIEW
BREAKTHROUGH THERAPY
90%
24%
10%
BIOSIMILAR
ORPHAN DRUG
62%
29%
pecialty drug names appear in ï‚« S magenta throughout the publication.
IMMUNOLOGY
avacopan oral Chemocentryx PROPOSED INDICATIONS
Antineutrophil cytoplasmic antibodies (ANCA)-associated vasculitis (AAV)
CLINICAL OVERVIEW
Hyperactivity of the complement pathway activates neutrophils causing damage to small blood vessels. Avacopan selectively inhibits the complement 5a receptor (C5aR) that is present on neutrophils. The double-blind, phase 3 ADVOCATE trial evaluated safety and efficacy of avacopan in 330 patients with AAV (granulomatosis with polyangiitis or microscopic polyangiitis). Patients were randomized 1:1 to avacopan 30 mg twice daily or standard prednisone therapy. Both groups also received either cyclophosphamide followed by azathioprine, or rituximab. The primary endpoint of disease remission was achieved in 72.3% and 70.1% of patients who received avacopan and prednisone, respectively, at week 26 (p<0.0001 for non-inferiority) and in 65.7% and 54.9% of patients, respectively, at week 52 (p=0.0066 for superiority). Avacopan resulted in longer remission compared to prednisone (HR, 0.46). In addition, among patients with renal impairment at baseline, greater improvement in function at week 52 was reported with avacopan compared to prednisolone (eGFR increase, 7.3 versus 4.1 mL/min/1.73 m2, respectively; p=0.029). Overall, fewer serious adverse events occurred with avacopan than with prednisone (25 versus 31 events, respectively). This included WBC decline, which was reported in 2.5% and 4.9% of patients, respectively. Conversely, liver function test increases were reported more often with avacopan than with prednisone (5.4% and 3.7%, respectively).
PLACE IN THERAPY
The estimated annual incidence of AAV in Europe and North America is 20 per million people. AAV encompasses a group of diseases including granulomatosis with polyangiitis (GPA), eosinophilic granulomatosis with polyangiitis (EGPA), and microscopic polyangiitis (MPA). The rare autoimmune disease occurs when neutrophils attack small blood vessels causing inflammation, damage, and possible necrosis. AAV affects several organs and signs and symptoms range from skin rash to fulminant multisystem disease. Staphylococcus aureus infection, select medications, and exposure to environmental toxins may trigger an AAV episode. If left untreated, AAV leads to significant morbidity and mortality, with a 75% survival rate at 5 years. Standard induction therapy for severe AAV episodes includes high-dose glucocorticoids plus immunosuppressive therapy (cyclophosphamide or rituximab). In patients with clinical decline despite ongoing induction therapy, plasma exchange may be added. For non-severe AAV cases, methotrexate, mycophenolate mofetil, or azathioprine may be used, depending on the patients’ clinical status. Overall, remission is typically maintained with azathioprine or methotrexate. In addition, the interleukin-5 (IL-5) inhibitor mepolizumab (Nucala®) is approved for SC administration in combination with oral corticosteroids in adults with EGPA. Avacopan offers a new approach to treat AAV that targets the C5aR. It demonstrated improvement in achieving and maintaining remission over oral steroid therapy when coupled with standard immunosuppressant therapy. Avocopan will compete with cyclophosphamide, rituximab (Rituxan®, biosimilars), and mepolizumab (Nucala) in the AAV space. Moreover, avacopan may decrease the need for steroid therapy and the incidence of steroidrelated adverse effects. The novel C5aR inhibitor is also in phase 2 trials for C3 glomerulopathy (C3G) and hidradenitis suppurativa (HS).
FDA APPROVAL TIMELINE July 7, 2021
Orphan Drug
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$25
$111
$253
$391
$532
The forecast is a projection of total US sales per year.
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IMMUNOLOGY
belumosudil oral Kadmon PROPOSED INDICATIONS
Chronic graft versus host disease (cGVHD)
CLINICAL OVERVIEW
Belumosudil is a Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor. ROCK2 has been shown to play a critical role in the regulation of proinflammatory cytokines, including interleukin 17 (IL-17) and interleukin 21 (IL-21). It is also involved in regulation of interferon gamma, T follicular helper and regulatory cells, and signal transducer and activator of transcription (STAT) 3 and 5 that are believed to be involved in development of cGVHD. The ongoing, open-label, phase 2 ROCKstar trial evaluated 2 doses of belumosudil in patients with cGVHD who had received ≥ 2 prior lines of systemic therapies, including prior ibrutinib or ruxolitinib. Among enrollees, 67% had severe cGVHD and 52% had involvement of > 4 organs. Patients were randomized 1:1 to receive oral belumosudil 200 mg either once or twice daily. In the 12-month interim analysis (median follow-up, 8 months), the primary endpoint of ORR was 73% with once-daily dosing and 77% with twice-daily dosing (p<0.001 for each). Median time to response was 4 weeks. Overall, secondary endpoints demonstrated a median DOR of 50 weeks, and 58% of patients were failure-free at 12 months. Improved QOL, as measured by the Lee Symptom Scale, was reported in 60% of patients. Corticosteroid use was decreased or discontinued in 64% and 21% of patients, respectively. Belumosudil was well tolerated. Adverse effects were similar to those observed with corticosteroids and other immunosuppressants used for cGVHD.
PLACE IN THERAPY
Chronic GVHD is a complication of allogenic HSCT in which the transplanted cells attack host cells resulting in inflammation and fibrosis. Chronic GVHD can be life-threatening, affecting multiple organ systems (e.g., skin, lungs, GI tract, liver, eyes, joints), and is reported in > 50% of allogenic HSCT recipients. Kadmon reports approximately 14,000 patients in the US are currently living with cGVHD. High-risk cGVHD requires systemic therapy and is associated with involvement of ≥ 3 organs, severity score > 2 in any single organ, persistent thrombocytopenia, or cGVHD that has progressed from acute GVHD. The mainstay of systemic therapy for moderate to severe cGVHD is glucocorticoids as well as supportive care to specific sites. If patients are unresponsive to glucocorticoids, the addition of a calcineurin inhibitor (e.g., cyclosporine, tacrolimus) may be considered. Ibrutinib (Imbruvica®) is also FDA-approved for cGVHD after failure of systemic therapy. Off-label use of imatinib, mycophenolate mofetil, rituximab, ruxolitinib, or sirolimus has also demonstrated benefit. Notably, patients with cGVHD are encouraged to enroll in a clinical trial. Belumosudil may inhibit key fibrotic processes associated with cGVHD. It demonstrated significant response in treating patients with cGVHD, and lacks significant safety concerns at this time. If approved, it will provide an oral option for patients with cGVHD who have failed ≥ 2 prior lines of systemic therapies. Belumosudil is also in phase 2 trials for idiopathic pulmonary fibrosis and cutaneous scleroderma. Ruxolitinib (Jakafi®) has also been submitted to the FDA for cGVHD; the agency's decision is anticipated in 2H 2021.
FDA APPROVAL TIMELINE May 30, 2021
Breakthrough Therapy
Orphan Drug
Priority Review
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$43
$137
$205
$274
$321
The forecast is a projection of total US sales per year.
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Project Orbis
RTOR
IMMUNOLOGY
bimekizumab SC UCB PROPOSED INDICATIONS
Moderate to severe chronic plaque psoriasis (PSO)
CLINICAL OVERVIEW
Bimekizumab is a monoclonal IgG1 antibody that inhibits interleukins 17A and 17F (IL-17 and IL-17F). The efficacy of bimekizumab was demonstrated in 4 pivotal randomized, double-blind, phase 3 trials in over 2,200 adults with moderate to severe PSO. Effectiveness was measured based on the primary endpoints of improvement in the PASI and IGA scores. » B E SURE (n=478): Bimekizumab was superior to adalimumab based on PASI 90 (86.2% versus 47.2%, respectively; p<0.001) and an IGA of 0 (clear) or 1 (almost clear) (85.3% versus 57.2%, respectively; p<0.001) at week 16. In addition, at week 24, an increase in skin clearance was observed after switching from adalimumab to bimekizumab. » B E RADIANT (n=743): Bimekizumab was superior to secukinumab based on PASI 100 at week 16. Superiority to secukinumab was also observed based on the secondary endpoints of PASI 75 at week 4 and PASI 100 at week 48. » B E VIVID (n=567): Superiority of bimekizumab compared to ustekinumab was demonstrated based on PASI 90 and IGA at week 16. Bimekizumab was also superior to ustekinumab based on the secondary endpoints of PASI 100 at week 52 (64.2% versus 38%, respectively; nominal p<0.001) and in achieving IGA of 0 or 1 and PASI 90 at week 52 compared with ustekinumab (IGA, 77.9% versus 60.7%, respectively; PASI 90, 81.6% versus 55.8%, respectively; p<0.001 for both). » B E READY (n=435): PASI 90 was maintained at 56 weeks in 86.8% of patients treated with continuous bimekizumab every 4 weeks and in 91% of patients who were switched to bimekizumab every 8 weeks compared to 16.2% who were switched to placebo. Across the studies, bimekizumab was generally well-tolerated. The most common TEAEs were nasopharyngitis, oral candidiasis, and upper respiratory tract infection. The overall rate of TEAEs were comparable with bimekizumab and adalimumab; however, serious TEAEs were reported more often with adalimumab than bimekizumab (3.1% versus 1.6%, respectively). No suicidal ideation/behavior, inflammatory bowel disease, or major adverse cardiac events were reported with bimekizumab (BE SURE). In BE VIVID, serious TEAEs were reported in 6.1% of patients treated with bimekizumab versus 7.4% treated with ustekinumab (at week 52).
PLACE IN THERAPY
Psoriasis is a chronic, multisystem, immune-mediated, inflammatory disease involving the skin and joints and characterized by hyperproliferation of epidermal keratinocytes. It affects an estimated 8 million people in the US. Median age at onset is 28 years. Treatment for PSO is based on severity of the condition. Targeted immunomodulators are indicated to treat moderate to severe PSO after failure of topical therapy alone when phototherapy is not available. Immunomodulators include injectable monoclonal antibodies which reduce the level of pathogenic cytokines (TNFα, IL-23, and IL-17), and the oral phosphodiesterase 4 (PDE4) inhibitor apremilast (Otezla®), which reduces the production of proinflammatory mediators. Bimekizumab is a monoclonal IgG1 antibody that inhibits IL-17A and IL-17F. In clinical trials, bimekizumab was generally well tolerated, but it lacks safety and efficacy data beyond 56 weeks. Although no safety concerns were identified with bimekizumab, safety signals have been associated with the anti-IL-17 class of medications. Brodalumab (Siliq®) carries a boxed warning for suicidal ideation, and inflammatory bowel disease exacerbations have been reported with secukinumab (Cosentyx®). If approved, bimekizumab will be
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bimekizumab cont. PLACE IN THERAPY cont.
the first monoclonal IgG1 antibody targeting IL-17A and IL-17F in the US to treat PSO. Effectiveness of every 4 week and every 8 week dosing were comparable; therefore, bimekizumab could be a self-administered option that is dosed as infrequently as every 8 weeks. Bimekizumab will compete with several biological agents that are currently available to treat PSO, particularly the TNFα inhibitor adalimumab (Humira®), IL-17A inhibitor secukinumab (Cosentyx®), and IL-12/23 inhibitor ustekinumab (Stelara®). In clinical trials, bimekizumab demonstrated superiority to all 3 of these agents. Other investigational biologics in late-stage development for moderate to severe PSO include mirikizumab (IL-23 inhibitor; Eli Lilly), spesolimab (IL-36R inhibitor; Boehringer Ingelheim), as well as biosimilars for ustekinumab. Bimekizumab is also in phase 3 studies for the treatment of psoriatic arthritis, axial spondyloarthritis, and hidradenitis suppurativa.
FDA APPROVAL TIMELINE July 2021
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$44
$205
$399
$601
$815
The forecast is a projection of total US sales per year and reflects future potential indications for bimekizumab.
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CARDIOLOGY
evinacumab IV Regeneron PROPOSED INDICATIONS
Homozygous familial hypercholesterolemia (HoFH) as adjunct to other lipid-lowering therapies
CLINICAL OVERVIEW
Evinacumab is a monoclonal antibody that inhibits the function of the angiopoietin-like 3 (ANGPTL3) protein. ANGPTL3 is expressed naturally in the liver and acts as a partial inhibitor of lipoprotein lipase and endothelial lipase. ANGPTL3 plays a key role in lipid metabolism by increasing the levels of triglycerides and other lipids. Inhibition of ANGPTL3 is associated with reduced LDL-C levels independent of the LDL receptor. The phase 3, double-blind, parallel-group ELIPSE HoFH trial is evaluating the efficacy and safety of evinacumab in 65 patients aged ≥ 12 years with HoFH who have achieved LDL-C ≥ 70 mg/dL on stable maximally tolerated lipid-lowering therapy. Interim data revealed that after 24 weeks of therapy, evinacumab resulted in a 47.1% reduction in LDL-C from baseline (primary endpoint) compared to a 1.9% increase with placebo (difference, -49%; 95% CI, -65 to -33.1; p<0.001). The LDL-C reduction with evinacumab was reported as early as week 2 of the study. Notably, LDL-C reductions were similar among those with null-null and non-null variants. LDL-reductions were also similar regardless of background anti-lipid therapies (e.g., statins, ezetimibe, lomitapide, PCSK9 inhibitors, apheresis). Serious adverse events reported in the evinacumab group were urosepsis and suicide attempt (1 case each). No deaths occurred. In the clinical study, evinacumab was administered as 15 mg/kg IV every 4 weeks.
PLACE IN THERAPY
HoFH is an autosomal dominant disorder that results in severe elevations in total cholesterol and LDL-C. In individuals with HoFH, cardiovascular disease and cerebrovascular disease as well as cutaneous xanthomas may emerge during childhood. It is estimated that HoFH occurs in 1 out of a million people in the US. Life expectancy of individuals with HoFH is approximately 30 years unless treated with a liver transplant, lipoprotein apheresis, or ileal bypass surgery. LDL-C lowering is a main objective of HoFH treatment, which consists of lifestyle modifications, pharmacotherapy with statins, and lipoprotein apheresis. Other LDL-lowering pharmacotherapies, such as ezetimibe, niacin, lomitapide, mipomersen, PCSK9 inhibitors, and bempedoic acid, may also be employed to achieve target LDL-C levels. If approved, evinacumab will be the first treatment that targets ANGPTL3 to lower LDL-C levels. Efficacy was similar regardless of genetic variants that result in virtually absent (null–null) or impaired (non-null) LDL-receptor activity. The initial application for approval in the US is for use in patients with HoFH and elevated LDL-C despite maximally tolerated lipid-lowering therapy. Evinacumab is also being studied in adults with persistent hypercholesterolemia and severe hypertriglyceridemia (both phase 2 trials). Subcutaneous administration of evinacumab is also being investigated. Evinacumab could compete with lomitapide in the HoFH space as it has a more desirable safety and tolerability profile compared to lomitapide, which is associated with hepatotoxicity. PSCK9 inhibitors are administered SC every 2 or 4 weeks and demonstrate higher LDL-C lowering (range, 55% to 59%) compared to evinacumab; therefore these agents could be preferred over evinacumab in the majority of HoFH patients who have residual LDL receptor activity.
FDA APPROVAL TIMELINE February 11, 2021
Breakthrough Therapy
Orphan Drug
Priority Review
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$17
$44
$65
$88
$154
The forecast is a projection of total US sales per year.
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ENDOCRINE
lonapegsomatropin SC Ascendis PROPOSED INDICATIONS
Pediatric growth hormone deficiency (GHD)
CLINICAL OVERVIEW
Lonapegsomatropin is long-acting prodrug of the human growth hormone (hGH) somatropin. Interim data from two phase 3 trials demonstrated safety and efficacy of lonapegsomatropin for GHD. In the randomized, open-label, HEIGHT trial, 161 treatment-naïve children 3 to 12 years of age with GHD were randomized to receive either SC injections of once-weekly lonapegsomatropin or daily somatropin (Genotropin®, 34 μg/kg/day = 0.24 mg/kg/week) for 52 weeks. Lonapegsomatropin was superior to somatropin in the primary endpoint of annualized height velocity (AHV) at 52 weeks (11.2 versus 10.3 cm/year, respectively; treatment difference, 0.86 cm/year; 95% CI, 0.22 to 1.5; p=0.0088). Rate of poor response (AHV < 8 cm/year) was lower with lonapegsomatropin compared to somatropin (4% versus 11%, respectively). The 26-week, open-label, FLIGHT trial demonstrated safety and tolerability of lonapegsomatropin in 146 participants 6 months to 17 years of age with GHD. No serious adverse events related to the study drug were observed in either trial. The ongoing ENLIGHTEN trial is evaluating use of an auto-injector for SC administration of lonapegsomatropin. Lonapegsomatropin was studied at a dosage of 0.24 mg/kg administered SC once weekly.
PLACE IN THERAPY
GHD is a rare disorder characterized by a lack of growth hormone (GH) production in the anterior pituitary gland. Causes of childhood-onset GHD may be considered congenital, acquired (e.g., brain trauma, CNS infection, tumor), or idiopathic. GHD results in growth retardation, short stature, and delays in lengthening of the bones of the extremities. Current treatment for GHD includes daily SC injections of somatropin (various brands). Lonapegsomatropin uses the proprietary TransCon platform to delay the release and clearance of somatropin in a predictable manner. If approved, lonapegsomatropin will be the first once-weekly hGH approved for use in pediatric patients with GHD. Ascendis is also developing an auto-injector to administer lonepagsomatropin that is stable at room temperature. Pfizer and Opko Health submitted an application for the long-acting hGH somatrogon for once-weekly administration for the treatment of pediatric GHD; an FDA decision is expected in October 2021. In addition, Novo Nordisk’s hGH analog somapacitan-beco (Sogroya®), which received FDA approval as a once-weekly SC injection for adult GHD in August 2020, is currently in phase 3 trials for pediatric GHD (ages 2 to 11 years). Lonapegsomatropin is in phase 2 trials for the treatment of GHD in adults.
FDA APPROVAL TIMELINE June 25, 2021
Orphan Drug
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$67
$197
$413
$583
$766
The forecast is a projection of total US sales per year.
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ONCOLOGY
loncastuximab tesirine IV ADC Therapeutics PROPOSED INDICATIONS
Relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL)
CLINICAL OVERVIEW
Loncastuximab tesirine is an antibody drug conjugate (ADC) that contains a humanized monoclonal antibody that targets the cluster of differentiation 19 (CD19) antigen found on B cells. Once loncastuximab tesirine enters the cell, it blocks DNA strand separation, ultimately resulting in cell death. The phase 2, single-arm LOTIS-2 trial evaluated loncastuximab tesirine in 145 adults with R/R DLBCL, including patients with poor prognosis (double/triple hit [10.3%], double/triple expressor [13.8%], transformed disease [20%]). Patients had received ≥ 2 prior therapies (range, 2 to 7); 24 patients received prior SCT, and 13 received prior CAR T therapy. Loncastuximab tesirine was administered IV over 30 minutes every 3 weeks as 150 mcg/kg during the first 2 cycles and 75 mcg/kg thereafter. After 1 year, doses were repeated every 12 weeks for up to 3 years. After a mean 4.3 treatment cycles (range, 1 to 15), loncastuximab tesirine demonstrated an ORR of 48.3% and a CR rate of 24.8%. Median DOR was 12.58 months, median PFS was 5.09 months, and overall survival was 9.53 months. TEAEs were considered manageable. The most common grade ≥ 3 TEAEs were neutropenia (26.2%), thrombocytopenia (17.9%), gamma-glutamyl transferase (GGT) increases (17.2%), and anemia (10.3%). The incidence of febrile neutropenia was low (3.4%). Following loncastuximab tesirine treatment, 15 patients in the trial received CD19-directed CAR T therapy with an investigator-assessed ORR of 46.7%, and 9 patients proceeded to SCT as consolidation after responding to loncastuximab tesirine. The phase 2, single-arm LOTIS-3 trial, combined loncastuximab tesirine with daily oral ibrutinib in adults with DLBCL who had failed or were intolerant to SOC therapy, including those with poor prognosis. Patients had received 1 to 6 prior therapies, including 4 patients who received prior SCT. During the 1-year trial, loncastuximab tesirine was administered IV as 60 mcg/kg every 3 weeks for 2 cycles during the first 14 weeks, after which the dose was 60 mcg/kg IV every 4 weeks for cycles 5, 6, 9, and 10. Oral ibritinib was given daily throughout the year. Among 37 evaluable patients in an interim analysis, ORR was 62.9% and CR rate was 31.4%. Grade ≥ 3 TEAEs included anemia (10.8%), neutropenia (10.8%), thrombocytopenia (5.4%), and fatigue (5.4%).
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 include medications that target specific biomarkers on tumor cells, as well as immunotherapies that stimulate the body’s immune response against malignancy. The CD19 protein expressed on B cells has become a very viable target for treating DLBCL. Current therapies for R/R DLBCL that are directed toward CD19 include the IV monoclonal antibody tafasitamab-cxix (Monjuvi®), used with lenalidomide in patients ineligible for autologous SCT, and single-dose CAR T therapies axicabtagene ciloleucel (Yescarta®) and tisagenlecleucel (Kymriah®). In general, ADC therapies produce deep and durable responses. Loncastuximab tesirine has exhibited a manageable safety profile, a significant ORR, and a durable response of up to 1 year or more in adults with R/R DBLCL, including patients at high risk. Moreover, loncastuximab tesirine has been effective when used after SCT or CAR T therapy and does not eliminate the possibility of subsequent SCT or CAR T; clinical data reports an ORR of 50% and CR rate of 43% in those who received CAR T therapy post loncastuximab tesirine. If approved, loncastuximab tesirine will be the second ADC available to treat R/R DBLCL. It may compete with the ADC polatuzumab vedotin-piiq (Polivy®), which targets CD79b. Although loncastuximab tesirine reported a lower CR rate than polatuzumab vedotin-piiq (used in combination with bendamustine and rituximab) in
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PLACE IN THERAPY cont.
non-comparative clinical trials (CR, 24% versus 40%, respectively), it has the advantage of being a chemotherapy-free option. Loncastuximab tesirine also has a potentially more desirable safety profile, as polatuzumab vedotin-piiq is associated with grade ≥ 3 neuropathy (incidence, 42%). Loncastuximab tesirine could also compete with tafasitamab-cxix, particularly in heavily pretreated patients with R/R DBLCL who cannot tolerate more intensive therapies. Use of loncastuximab tesirine in combination with rituximab as second-line therapy for R/R DLBCL is also being investigated in the phase 3 confirmatory LOTIS-5 trial. In addition, the product is in phase 2 evaluation for mantle cell lymphoma.
FDA APPROVAL TIMELINE May 21, 2021
Orphan Drug
Priority Review
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$48
$132
$210
$373
$662
The forecast is a projection of total US sales per year.
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IMMUNOLOGY
pegcetacoplan SC Apellis PROPOSED INDICATIONS
Paroxysmal nocturnal hemoglobinuria (PNH)
CLINICAL OVERVIEW
Pegcetacoplan is a complement 3 (C3) inhibitor designed to regulate hyperactivity of the complement pathway and destruction of RBCs associated with PNH. The phase 3 PEGASUS trial compared safety and efficacy of pegcetacoplan and eculizumab for the treatment of PNH. It enrolled 80 adults with PNH who were on stable eculizumab therapy and had a Hb level < 10.5 g/dL at screening. During a 4-week run-in period, all patients received pegcetacoplan in addition to their current eculizumab dose. Patients were then randomized to pegcetacoplan or their current eculizumab dose for 16 weeks, after which 77 patients entered an open-label period receiving pegcetacoplan until week 48. At week 16, pegcetacoplan resulted in a mean increase from baseline in Hb (primary endpoint) by 2.9 g/dL, which was sustained through week 48. In addition, 73% of patients in the pegcetacoplan group remained transfusion free. This was an increase from the year prior to the study, in which only 25% of the patients were transfusion free while on eculizumab. The TEAEs reported more often with pegcetacoplan than eculizumab included injection site reactions (36.6% versus 2.6%, respectively), any infection (29.3% versus 23.1%, respectively), and diarrhea (22% versus 0%, respectively). Meningitis was not reported in either group. The rate of discontinuations due to TEAEs was 7.3% with pegcetacoplan compared to 0% with eculizumab. In PEGASUS, pegcetacoplan 1,080 mg was administered SC twice weekly.
PLACE IN THERAPY
PNH is a rare blood disorder that is estimated to occur in 0.5 to 1.5 per million people worldwide. Diagnosis is typically made during the fourth decade of life. In PNH, the bone marrow produces abnormal RBCs that prematurely hemolyze, resulting in hemolytic anemia and chronic hemoglobinuria that can worsen during illness, trauma, or stress. Most patients experience fatigue and difficulty breathing. Individuals with PNH are at risk of developing potentially life-threatening blood clots. Allogeneic SCT is the only cure for PNH. Eculizumab (Soliris®) was the first drug available in the US to manage PNH. This was followed by the approval of ravulizumab-cwvz (Ultomiris®) in 2018. Both complement inhibitors target complement protein C5, while pegcetacoplan targets C3, which is upstream in the complement pathway and may improve efficacy. Pegcetacoplan and ravulizumab-cwvz have shown non-inferiority to eculizumab in treatment-experienced and treatment-naïve patients, respectively, based on the proportion of patients who remained transfusionfree in clinical trials. Pegcetacoplan also showed improvement in Hb over eculizumab. Eculizumab and ravulizumab-cwvz carry boxed warnings for reports of serious meningococcal infections. To date, the PEGASUS trial has not reported meningitis with pegcetacoplan. If approved, pegcetacoplan will provide an administration method that does not require an HCP visit. While eculizumab is dosed IV once every 2 weeks and ravulizumab-cwvz is dosed IV once every 8 weeks, pegcetacoplan can be self-administered SC twice weekly. Pegcetacoplan will compete with eculizumab and ravulizumab-cwvz, both of which also hold indications for atypical hemolytic uremic syndrome (aHUS).
FDA APPROVAL TIMELINE May 14, 2021
Fast Track
Orphan Drug
Priority Review
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$19
$76
$151
$206
$245
The forecast is a projection of total US sales per year. 12 | MAGELLANRX.COM
INFECTIOUS DISEASE
taurolidine/heparin/citrate IV Cormedix PROPOSED INDICATIONS
Prevention of catheter-related blood stream infections (CRBSI) in hemodialysis (HD) patients
CLINICAL OVERVIEW
Cormedix’s proprietary catheter lock solution contains taurolidine 1.35%, citrate 3.5%, and heparin 1,000 units/mL. Taurolidine is an amino acid derivative that denatures surface proteins and chemically alters membrane lipids. It has shown in vitro activity against gram-positive and gram-negative bacteria, including antibiotic resistant strains, and activity against mycobacteria and clinically relevant fungi, including Aspergillus. Heparin provides anticoagulation activity and citrate is a pH buffer. In the double-blind, phase 3 LOCK-IT-100 trial, patients with ESRD receiving HD ≥ 2 times per week were randomized 1:1 to taurolidine/heparin/citrate or the SOC catheter lock heparin 1,000 units/mL. Based on an interim analysis that included 653 patients, the study revealed a 72% reduction in CRBSIs (primary endpoint) with taurolidine/heparin/citrate compared to heparin alone (incidence, 6 versus 22 events per 1,000 catheter days, respectively; p=0.0034). Among the secondary endpoints, the incidence of catheter removal due to CRBSI was lower with taurolidine/heparin/citrate versus heparin (2% versus 7.3%, respectively). Loss of catheter patency was reported in 16% of patients in the taurolidine/heparin/citrate group compared to 12% in the heparin group, but the difference was not significant (p=0.12). Adverse events that were reported more often with taurolidine/heparin/citrate than with heparin included cardiac failure (3% versus 1.8%, respectively), fluid overload (3.5% versus 3%, respectively), and hyperkalemia (2.5% versus 2%, respectively). Taurolidine/heparin/citrate is instilled in the arterial and venous lumens of the HD catheter after each HD session. The catheter lock solution is not intended for systemic administration. Prior to the next use of the lumen, the catheter lock solution is aspirated from the lumen.
PLACE IN THERAPY
It is reported that over 450,000 individuals in the US are HD dependent. Based on 2008 data, an estimated 37,000 CRBSIs occurred among those who received outpatient HD. Tunneled dialysis catheters provide immediate vascular access and are often used for months or years. Proper catheter management to maintain patency and prevent infection is imperative. Currently, no pharmacological agent is FDA-approved for the prevention of CRBSIs. Heparin is the SOC to maintain catheter patency. In addition, the CDC recommends applying topical povidone iodine ointment or bacitracin/gramicidin/polymyxin B ointment at the catheter exit site after each HD session, if compatible with the catheter material. In general, risk factors for developing CRBSI include duration of catheterization, catheter material, insertion conditions and skill of inserter, catheter site care, increased manipulation of catheter, and catheter thrombosis. Indwelling catheters in particular, including those used for HD, are at risk for CRBSI. In clinical studies, taurolidine/heparin/citrate resulted in significantly fewer cases of CRBSI and displayed a similar safety profile when compared to heparin alone in HD patients. If approved, it will be the first antibacterial and antifungal catheter lock solution in the US to prevent catheter-related infections in this patient population. Minocycline/edetate/ethyl alcohol catheter lock solution, by Citius Pharmaceuticals, is also in phase 3 investigation for the treatment of catheter-related or central line associated bloodstream infection (CRBSI/CLABSI), including in hemodialysis patients.
FDA APPROVAL TIMELINE February 28, 2021
Fast Track
QIDP
Priority Review
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$18
$50
$95
$140
$185
The forecast is a projection of total US sales per year. 13 | MAGELLANRX.COM
Biosimilar Overview CLINICAL OVERVIEW
Biosimilars are very different from generic drugs in that they are not exact duplicates of their reference biologic product. The FDA approval process for biosimilars is designed to ensure that the biosimilar product is highly similar to the reference product without having any meaningful clinical differences. Many controversies surround biosimilars, and regulatory and litigation hurdles remain. The FDA has issued final and draft guidances. Select FDA biosimilar guidances are noted here. In January 2017, the agency issued final guidance on the nonproprietary naming of biologic products, which also applies to biosimilars. The biological products must bear a core name followed by a distinguishing 4-letter, lowercase, hyphenated suffix that is devoid of meaning. The FDA is still considering how to implement the nomenclature for previously approved biosimilar products. The international nonproprietary name (INN) impacts interchangeability as it affects pharmacists’ ability to substitute an interchangeable biosimilar for the reference product. The FDA withdrew the September 2017 draft industry guidance on determining similarity of a proposed biosimilar product to its reference product to allow for further consideration of the most current and relevant scientific methods in evaluating analytical data. The agency will focus on providing flexibility for efficient development of biosimilars while maintaining high scientific standards. In July 2018, the FDA finalized its guidance on labeling biosimilars. The guidance pertains to prescribing information (PI) but does not contain specific recommendations on interchangeability in the labeling. The labeling guidance provides recommendations on how to include, identify, and differentiate the biosimilar and the reference product in various sections of the PI. The basic premise remains that the originator product’s safety and effectiveness can be relied upon for HCPs to make prescribing decisions; therefore, a biosimilar should include relevant data from the originator in its PI. In May 2019, the agency released its final guidance on interchangeability. Several states had already enacted biosimilar substitution legislation. Biosimilars are expected to receive full extrapolation for the eligible indications of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will likely need to be considered individually. Insulins were historically regulated by the FDA as small molecules. However, effective March 23, 2020, drugs such as insulin and growth hormone were deemed biologics and transitioned from the drug pathway to the biologics pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.
PLACE IN THERAPY
The patents of several biologic drugs are set to expire in the next few years, opening the US market for biosimilar entry; however, patent litigation has resulted in significant launch delays of FDA-approved biosimilars. In June 2017, the US Supreme Court issued 2 landmark rulings: (1) allowing a biosimilar manufacturer to provide launch notice of commercial marketing to the originator manufacturer before or after FDA approval of the biosimilar product and (2) eliminating any federal requirement for disclosure, also known as the “patent dance.” Some states, however, mandate disclosure. These decisions may bring biosimilars to the market sooner and potentially create price competition in the marketplace. In July 2018, the FDA unveiled its Biosimilar Action Plan (BAP), a series of 11 steps to encourage biosimilar market competition, some of which were previously announced or underway. The BAP contains 4 key strategies: (1) improving the biosimilar development and approval process; (2) maximizing scientific and regulatory clarity for sponsors; (3) providing effective communications for patients, clinicians, and payers; and (4) reducing unfair tactics that may delay market approval and entry. The BAP strives to promote access to biosimilar products and reduce healthcare costs.
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To date, a total of 29 biosimilars have received FDA approval. Of these, only 19 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)
Manufacturer
Approval Date
Zarxio® (filgrastim-sndz)
Novartis/Sandoz
March 2015
Inflectra® (infliximab-dyyb)
Pfizer/Celltrion
April 2016
Erelzi™ (etanercept-szzs)
Novartis/Sandoz
August 2016
Amjevita™ (adalimumab-atto)
Amgen
September 2016
Renflexis® (infliximab-abda)
Samsung Bioepis/ Merck
May 2017
Cyltezo® (adalimumab-adbm)
Boehringer Ingelheim
August 2017
Mvasi™ (bevacizumab-awwb)
Amgen
September 2017
Ixifi™ (infliximab-qbtx)*
Pfizer
December 2017
Ogivri™ (trastuzumab-dkst)
Mylan
December 2017
Retacrit (epoetin alfa-epbx)
Pfizer/Hospira
May 2018
Fulphila® (pegfilgrastim-jmdb)
Mylan
June 2018
Nivestym® (filgrastim-aafi)
Pfizer
July 2018
Hyrimoz™ (adalimumab-adaz)
Novartis/Sandoz
October 2018
Udenyca® (pegfilgrastim-cbqv)
Coherus
November 2018
Truxima® (rituximab-abbs)
Celltrion/Teva
November 2018
Herzuma® (trastuzumab-pkrb)
Celltrion/Teva
December 2018
Ontruzant® (trastuzumab-dttb)
Samsung Bioepis/ Merck
January 2019
Trazimera™ (trastuzumab-qyyp)
Pfizer
March 2019
Eticovo™ (etanercept-ykro)
Samsung Bioepis/ Merck
April 2019
Kanjinti™ (trastuzumab-anns)
Amgen
June 2019
Zirabev™ (bevacizumab-bvzr)
Pfizer
June 2019
Hadlima™ (adalimumab-bwwd)
Samsung Bioepis/ Merck
July 2019
Ruxience™ (rituximab-pvvr)
Pfizer
July 2019
Abrilada™ (adalimumab-afzb)
Pfizer
November 2019
Ziextenzo® (pegfilgrastim-bmez)
Novartis/Sandoz
November 2019
®
15 | MAGELLANRX.COM
Commercially Available
-
-
-
-
-
-
-
Originator Product (Manufacturer) Neupogen® (Amgen) Remicade® (Janssen) Enbrel® (Amgen) Humira® (Abbvie) Remicade (Janssen) Humira (Abbvie) Avastin® (Genentech) Remicade (Janssen) Herceptin® (Genentech) Epogen® (Amgen) Procrit® (Janssen) Neulasta® (Amgen) Neupogen (Amgen) Humira (Abbvie) Neulasta (Amgen) Rituxan (Genentech) Herceptin (Genentech) Herceptin (Genentech) Herceptin (Genentech) Enbrel (Amgen) Herceptin (Genentech) Avastin (Genentech) Humira (Abbvie) Rituxan (Genentech) Humira (Abbvie) Neulasta (Amgen)
APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)
Manufacturer
Approval Date
Avsola™ (infliximab-axxq)
Amgen
December 2019
Nyvepria™ (pegfiltrastim-apgf)
Pfizer
June 2020
Hulio® (adalimumab-fkjp)
Mylan
July 2020
Riabni™ (rituximab-arrx)
Amgen
December 2020
Commercially Available
-
Originator Product (Manufacturer) Remicade (Janssen) Neulasta (Amgen) Humira (Abbvie) Rituxan (Genentech)
* Pfizer already has Inflectra on the market and has not announced plans to launch Ixifi.
Also available are Eli Lilly’s Basaglar® insulin glargine, a biosimilar agent to Sanofi’s Lantus®, and Sanofi’s Admelog® insulin lispro, approved as a biosimilar product to Eli Lilly’s Humalog®. In June 2020, the FDA approved insulin glargine (Semglee™) by Mylan/Biocon under an abbreviated 505(b)(2) New Drug Application (NDA) pathway; the reference product was Lantus. Semglee is considered a biologic under section 351(a) rather than a biosimilar. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play a role in market adoption of biosimilars. While < 2% of Americans use biologics, they account for almost 40% of all prescription drug spending. Moreover, they comprised 70% of growth in drug spending from 2010 to 2015. Not surprisingly, there is a growing body of evidence on predicted biologic spend and potential biosimilar savings. A 2020 report by IQVIA forecasts that biosimilars are on track to reduce overall US drug spend by $100 billion over the next 5 years. In fact, the next 5 years are expected to have an estimated 5-fold increase in savings relative to the past 5 years as more biosimilars launch and existing biosimilars see more utilization and reductions in price. Three biosimilar launches in 2019 saw substantial uptake within the first year of commercialization, these are: bevacizumab (42%), trastuzumab (38%), and rituximab (20%). In the US, it is estimated that biosimilars will cost 15% to 35% less than the originator product, although price dynamics vary. The potential cost savings, however, can vary based on the market segment where brand contracts can play a role. A 2017 report by the RAND Corporation estimates a $54 billion cost savings from biosimilars between 2017 and 2026. In July 2018, an FDA analysis reported that if Americans had access to FDA-approved biosimilars in 2017, it would have resulted in a $4.5 billion savings. A 2017 analysis by the Moran Company projects biosimilars could save the government an estimated $11.4 billion by 2027, but it would require the Centers for Medicare and Medicaid Services (CMS) to revise its reimbursement policy for biosimilars. Subsequently, in November 2017, the CMS revised its reimbursement policy. The CMS now issues a unique Healthcare Common Procedure Coding System (HCPCS) code to each individual biosimilar. Under this rule, Medicare Part B separately codes and pays for biosimilars and no longer groups them into a common payment code with originator agents. A June 2018 infliximab case study by the Pacific Research Institute forecasts annual savings of up to $465 million from increased use of biosimilars to replace a single biologic for commercial payers and Medicare. Biosimilars are paving the way for increased access to important biologic therapies that may increase survival and/ or QOL for many patients with difficult-to-treat diseases while also reducing costs.
16 | MAGELLANRX.COM
BIOSIMILAR OVERVIEW continued
IMMUNOLOGY
adalimumab SC AVT-02 and CHS-1420 are biosimilars to Abbvie’s Humira, a tumor necrosis factor alpha (TNFα) blocker indicated for the treatment of autoimmune disorders, including rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), ankylosing spondylitis (AS), plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD) in adults and children, ulcerative colitis (UC), hidradenitis suppurativa (HS), and non-infectious uveitis.
FDA APPROVAL TIMELINE Alvotech (AVT-02) September 2021
Coherus (CHS-1420) September 2021
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$16,872
$17,387
$12,802
$9,143
$7,085
The forecast is a projection of total US sales per year for the branded originator product.
ONCOLOGY
bevacizumab IV Aybintio, Bmab-100, BAT1706, and FKB238 are investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth factor (VEGF)-specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, non-squamous non-small cell lung cancer (nsNSCLC), glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.
FDA APPROVAL TIMELINE
Bio-Thera Solutions (BAT1706) September 2021 Centus/AstraZeneca (FKB238) Pending Merck/Samsung Bioepis (Aybintio) Pending Mylan/Biocon (Bmab-100) Pending
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$1,369
$1,079
$892
$787
$705
The forecast is a projection of total US sales per year for the branded originator product.
17 | MAGELLANRX.COM
BIOSIMILAR OVERVIEW continued
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/Amneal Pending
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$110
$95
$85
$78
$72
The forecast is a projection of total US sales per year for the branded originator product.
ENDOCRINE
insulin aspart SC Mylan/Biocon Mylan/Biocon is seeking biosimilar approval to Novo Nordisk’s Novolog®, a rapid-acting insulin to improve glycemic control in patients with T1DM or T2DM.
FDA APPROVAL TIMELINE April to June 2021
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$750
$640
$561
$517
$485
The forecast is a projection of total US sales per year for the branded originator product.
18 | MAGELLANRX.COM
BIOSIMILAR OVERVIEW continued
BLOOD MODIFIER
pegfilgrastim SC Lapelga, MSB-11455, and TPI-120 are investigational biosimilars to Amgen’s Neulasta, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs and in patients acutely exposed to myelosuppressive doses of radiation (HSARS).
FDA APPROVAL TIMELINE Apotex (Lapelga) Pending
Kashiv/Amneal (TPI-120) January 2022 Merck/Fresenius (MSB-11455) March 2021
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$1,609
$1,333
$1,132
$985
$876
The forecast is a projection of total US sales per year for the branded originator product.
OPHTHALMOLOGY
ranibizumab intravitreal Biogen/Samsung Bioepis Biogen/Samsung Bioepis are seeking biosimilar approval to Genentech’s Lucentis®, a vascular endothelial growth factor (VEGF) inhibitor indicated to treat wet age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy, and myopic choroidal neovascularization (mCNV).
FDA APPROVAL TIMELINE September to October 2021
FINANCIAL FORECAST (reported in millions) 2021
2022
2023
2024
2025
$1,727
$1,653
$1,488
$1,278
$1,104
The forecast is a projection of total US sales per year for the branded originator product.
19 | MAGELLANRX.COM
Keep on Your RADAR Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the MRx Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2025, are displayed. The financials are projected total annual US sales, reported in millions.
tirzepatide Diabetes
trilaciclib
abrocitinib
$593
$570
Oncology
Dermatology
adagrasib Oncology
$1,404
$1,035
somatrogon
aducanumab
$74
$2,501
Neurology
Endocrine
bardoxolone methyl
NVX-CoV2373 vaccine
Renal
COVID-19
$1,134
$1,637 mirikizumab
betibeglogene autotemcel (Zynteglo)
$324
$582
Hematology/Gene therapy
Immunology
mavacamten
deucravacitinib
$538
$1,305
Immunology
Cardiovascular
efgartigimod
lenadogene nolparvovec (GS010)
Immunology
$1,098
Ophthalmology/Gene therapy
$53
elivaldogene tavalentivec (Lenti-D)
ipatasertib
Neurology/Gene therapy
Oncology
$382
idecabtagene vicleucel
ďŹ rmacute eubacterial spores
$771
$598
Oncology
$65
Gastrointestinal
pecialty drug names appear in ď&#x201A;Ť S magenta throughout the publication.
Pipeline DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2022. It is not intended to be a comprehensive inventory of all drugs in the pipeline; emphasis is placed on drugs in high-impact categories. Investigational drugs with a Complete Response Letter (CRL) and those that have been withdrawn from development are also noted. APPLICATION SUBMITTED APPLICATION TO THE FDA SUBMITTED
IN PHASE 3 PHASE 3 TRIALS TRIALS
61%
63%
39%
37%
34%
35%
34%
Specialty
23%
14%
10%
9%
Traditional
Orphan Drug
Priority Review
Breakthrough Therapy
Biosimilar
pecialty drug names appear in ï&#x201A;« S magenta throughout the publication.
PIPELINE DRUG LIST Specialty drug names appear in magenta throughout the publication. NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
Submitted (New Drugs) lamotrigine
Eton
Partial seizures
Oral
Submitted – 505(b)(2) NDA
2021
ansofaxine
Luye
MDD
Oral
Submitted – NDA
January 2021
eflapegrastim
Spectrum
Neutropenia/leukopenia
SC
Submitted – BLA
Jan–Feb 2021
tepotinib
Merck
NSCLC (mesenchymalepithelial transition exon 14 [METex14] skipping)
Oral
Submitted – NDA; Breakthrough Therapy; Priority Review; RTOR
Jan–Mar 2021
inolimomab
Elsalys
GVHD (acute, steroidrefractory)
IM
Submitted – BLA; Orphan Drug; RTOR
Jan–Jul 2021
dostarlimab
GlaxoSmithKline
Endometrial cancer (dMMR/MSI-H, recurrent, 2nd-line)
IV
Submitted – BLA
01/14/2021
evinacumab
Regeneron
HoFH
IV
Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review
02/11/2021
trilaciclib
G1 Therapeutics/ Boehringer Ingelheim
SCLC
IV
Submitted – NDA; Breakthrough Therapy; Priority Review
02/15/2021
umbralisib
TG
Marginal zone lymphoma (≥ 1 prior anti-CD20 based regimen)
Oral
Submitted – NDA; seeking Accelerated Approval; Breakthrough Therapy; Orphan Drug; Priority Review
02/15/2021
casimersen
Sarepta
DMD (amenable to exon 45 skipping)
IV
Submitted – NDA; seeking Accelerated Approval; Orphan Drug; Priority Review
02/25/2021
melflufen
Oncopeptides
Multiple myeloma (triplerefractory)
IV
Submitted – NDA; Orphan Drug; Priority Review
02/26/2021
udenafil
Allergan
Single ventricle heart disease (ages ≥ 12 years)
Oral
Submitted – NDA; Orphan Drug
02/26/2021
paclitaxel/encequidar
Athenex
Breast cancer
Oral
Submitted – NDA; Priority Review
02/28/2021
taurolidine/heparin/citrate
Cormedix
Prevention of catheterrelated blood stream infections (hemodialysis patients)
IV
Submitted – NDA; Fast Track; QIDP; Priority Review
02/28/2021
pegfilgrastim (biosimilar to Amgen’s Neulasta)
Merck/Fresenius
Neutropenia/leukopenia
SC
Submitted – BLA
March 2021
ropeginterferon alfa-2b
Pharmaessentia
Polycythemia vera (in absence of symptomatic splenomegaly)
SC
Submitted – BLA; Orphan Drug
Mar–Apr 2021
budesonide oral suspension
Takeda
Eosinophilic esophagitis
Oral
Submitted – 505(b)(2) NDA; Breakthrough Therapy; Orphan Drug; Priority Review
Mar–Jun 2021
22 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
d-methylphenidate
Corium
ADHD
Oral
Submitted – 505(b)(2) NDA
aducanumab
Biogen/Eisai
Alzheimer’s disease
IV
Submitted – BLA; Fast 03/05/2021 Track; Priority Review
ponesimod
Janssen
MS (relapsing)
Oral
Submitted – NDA
03/18/2021
roxadustat
AstraZeneca
Anemia due to CKD (dialysis-dependent, dialysis-independent)
Oral
Submitted – NDA
03/20/2021
idecabtagene vicleucel
Bristol-Myers Squibb/ Bluebird Bio
Multiple myeloma (≥ 3 prior therapies)
IV
Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review
03/26/2021
dasiglucagon
Zealand
Hypoglycemia (diabetesrelated)
SC
Submitted – NDA; Orphan Drug
03/27/2021
tivozanib hydrochloride monohydrate
Aveo
RCC (relapsed/refractory)
Oral
Submitted – NDA
03/31/2021
eflornithine/sulindac
Mallinckrodt
Familial adenomatous polyposis
Oral
Submitted – 505(b)(2) NDA; seeking Accelerated Approval; Fast Track; Orphan Drug
Apr–Jun 2021
insulin aspart (biosimilar to Novo Nordisk’s Novolog)
Mylan/Biocon
T1DM; T2DM
SC
Submitted – BLA
Apr–Jun 2021
tanezumab
Pfizer
Osteoarthritis pain
IV
Submitted – BLA; Fast Apr–Jun 2021 Track
tralokinumab
AstraZeneca
Atopic dermatitis
SC
Submitted – BLA
Apr–Jun 2021
brincidofovir
Chimerix
Smallpox
Oral
Submitted – NDA; Fast Track; Orphan Drug; Priority Review
04/07/2021
fosdenopterin
Bridgebio
Molybdenum cofactor deficiency
IV
Submitted – NDA; 04/09/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease
estetrol/drospirenone
Mayne
Contraception
Oral
Submitted – NDA
04/16/2021
benzoyl peroxide
Sol-Gel
Rosacea
Transdermal
Submitted – 505(b)(2) NDA
04/26/2021
pegunigalsidase alfa
Chiesi
Fabry’s disease
IV
Submitted – BLA; seeking Accelerated Approval; Fast Track; Priority Review
04/27/2021
abrocitinib
Pfizer
Atopic dermatitis
Oral
Submitted – NDA; Breakthrough Therapy; Priority Review
04/30/2021
bupivacaine/meloxicam
Heron
Postsurgical pain
Instillation
Submitted – NDA; Breakthrough Therapy; Fast Track
05/13/2021
pegcetacoplan
Apellis
Paroxysmal nocturnal hemoglobinuria
SC
Submitted – NDA; Fast Track; Orphan Drug; Priority Review
05/14/2021
23 | MAGELLANRX.COM
03/02/2021
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
avalglucosidase alfa
Sanofi
Pompe disease
IV
Submitted – BLA; Breakthrough Therapy; Fast Track; Priority Review
05/18/2021
loncastuximab tesirine
ADC Therapeutics
DLBCL (relapsed/ refractory)
IV
Submitted – BLA; Orphan Drug; Priority Review
05/21/2021
dehydrated alcohol
Eton
Methanol poisoning
SC
Submitted – 505(b)(2) NDA; Orphan Drug
05/27/2021
leuprolide mesylate readyto-use, 6-month depot
Foresee
Prostate cancer
SC
Submitted – 505(b)(2) NDA
05/27/2021
zonisamide oral suspension
Eton
Partial seizures
Oral
Submitted – 505(b)(2) NDA
05/29/2021
belumosudil
Kadmon
GVHD (chronic)
Oral
Submitted – NDA; 05/30/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Project Orbis; RTOR
20-valent pneumococcal conjugate vaccine
Pfizer
Pneumococcal pneumonia prevention
IM
Submitted – BLA; Breakthrough Therapy; Fast Track; Priority Review
June 2021
cantharidin
Verrica
Molluscum contagiosum
Topical
Submitted – NDA
June 2021
infigratinib
Bridgebio
Biliary tract cancer
Oral
Submitted – NDA; June 2021 Fast Track; Orphan Drug; Priority Review; RTOR
relugolix/estradiol/ norethindrone
Myovant
Uterine fibroid-related Oral heavy menstrual bleeding
Submitted – NDA
06/01/2021
samidorphan/olanzapine
Alkermes
Bipolar disorder; Schizophrenia
Oral
Submitted – NDA
06/01/2021
plasminogen (human)
Liminal
Congenital plasminogen deficiency
IV
Submitted – BLA; Fast 06/05/2021 Track; Orphan Drug; Rare Pediatric Disease
ibrexafungerp
Scynexis
Vulvovaginal candidiasis
Oral
Submitted – NDA; Fast Track; Orphan Drug; QIDP; Priority Review
06/14/2021
umbralisib
TG
Follicular lymphoma (≥ 2 prior systemic therapies)
Oral
Submitted – NDA; seeking Accelerated Approval; Orphan Drug
06/15/2021
arimoclomol
Orphazyme
Niemann-Pick disease
Oral
Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review; Rare Pediatric Disease
06/17/2021
cyclosporine
Santen
Dry eye syndrome
Ophthalmic
Submitted – 505(b)(2) NDA; Orphan Drug
06/24/2021
lonapegsomatropin
Ascendis
Growth hormone deficiency (pediatrics)
SC
Submitted – BLA; Orphan Drug
06/25/2021
24 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
bimekizumab
UCB
PSO
SC
Submitted – BLA
anifrolumab
AstraZeneca
SLE
IV
Submitted – BLA; Fast Jul–Dec 2021 Track
sotorasib
Amgen
NSCLC (KRAS G12Cmutation, locally advanced/metastatic, ≥ 1 prior systemic therapy)
Oral
Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RTOR
Jul–Dec 2021
teplizumab
Provention
T1DM (delay/prevention)
IV
Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review
07/02/2021
avacopan
Chemocentryx
ANCA-associated vasculitis
Oral
Submitted – NDA; Orphan Drug
07/07/2021
finerenone
Bayer
Diabetic nephropathy
Oral
Submitted – NDA; Priority Review
07/09/2021
narsoplimab
Omeros
HSCT-associated thrombotic microangiopathy
IV, SC
Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review
07/16/2021
15-valent pneumococcal conjugate vaccine
Merck
Invasive pneumococcal disease prevention
IM
Submitted – BLA; Breakthrough Therapy; Priority Review
07/18/2021
retifanlimab
Incyte
Anal cancer (squamous cell, locally advanced/ metastatic, failed on/ intolerant to platinumbased chemotherapy)
IV
Submitted – BLA; Orphan Drug; Priority Review
07/25/2021
tretinoin/benzoyl peroxide
Sol-Gel
Acne vulgaris
Topical
Submitted – 505(b)(2) NDA
08/01/2021
topiramate oral solution
Eton
Partial seizures
Oral
Submitted – 505(b)(2) NDA
08/06/2021
vosoritide
Biomarin
Achondroplasia
SC
Submitted – NDA; Orphan Drug
08/20/2021
adalimumab (biosimilar to Abbvie’s Humira)
Alvotech
RA; AS; PSO; PsA; JIA; CD; UC
SC
Submitted – BLA
September 2021
adalimumab (biosimilar to Abbvie’s Humira)
Coherus
RA; AS; PSO; PsA; JIA; CD; UC
SC
Submitted – BLA
September 2021
bevacizumab (biosimilar to Genentech’s Avastin)
Bio-Thera Solutions
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Submitted – BLA
September 2021
ranibizumab (biosimilar to Genentech’s Lucentis)
Biogen/Samsung Bioepis
Wet AMD
Intravitreal
Submitted – BLA
Sep–Oct 2021
paliperidone palmitate 6-month injectable
Janssen
Schizophrenia
IM
Submitted – NDA
09/02/2021
dihydroergotamine mesylate
Impel Neuropharma
Migraine treatment
Intranasal
Submitted – 505(b)(2) NDA
09/06/2021
risperidone long-acting in situ microparticle
Rovi
Schizophrenia
IM
Submitted – 505(b)(2) NDA
09/24/2021
reltecimod
Atox
Necrotizing soft tissue infection (NSTI)-related organ dysfunction/failure (ages ≥ 12 years)
IV
Submitted – NDA; seeking Accelerated Approval; Fast Track; Orphan Drug
09/30/2021
25 | MAGELLANRX.COM
July 2021
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
cabotegravir long-acting
Viiv
HIV-1 infection preexposure prevention (PrEP)
IM
Submitted – NDA; Breakthrough Therapy
Late 2021– Early 2022
dexamethasone insert
Ocular Therapeutix
Allergic conjunctivitis
Intraocular
Submitted – NDA
October 2021
somatrogon
Pfizer/Opko
Growth hormone deficiency (pediatrics)
SC
Submitted – BLA; Orphan Drug
October 2021
amivantamab
Janssen
NSCLC (EGFR exon 20 insertion mutations, progressed on or after platinum-based chemotherapy)
IV
Submitted – BLA; Breakthrough Therapy
Oct–Dec 2021
efgartigimod
Argenx
Myasthenia gravis
IV
Submitted – BLA; Fast Oct–Dec 2021 Track; Orphan Drug
avapritinib
Blueprint Medicines
Mastocytosis (advanced, systemic)
Oral
Submitted – NDA; Breakthrough Therapy; Orphan Drug
10/15/2021
sodium oxybate (oncenightly)
Avadel
Narcolepsy-related excessive daytime sleepiness and cataplexy
Oral
Submitted – 505(b)(2) NDA; Orphan Drug
10/15/2021
varenicline
Oyster Point
Dry eye syndrome
Intranasal
Submitted – 505(b)(2) NDA
10/18/2021
phenylephrine 2.5%/ tropicamide 1%
Eyenovia
Pharmacologic mydriasis
Ophthalmic
Submitted – 505(b)(2) NDA
10/29/2021
sulopenem
Iterum
Uncomplicated UTI (quinolone-resistant)
IV, Oral
Submitted – NDA; Fast Track; QIDP
November 2021
difelikefalin
Cara
Pruritus (hemodialysisrelated)
IV
Submitted – NDA; Breakthrough Therapy
December 2021
odevixibat
Albireo
Progressive familial intrahepatic cholestasis
Oral
Submitted – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease
December 2021
oportuzumab monatox
Sesen
Bladder cancer (BCGunresponsive, nonmuscle invasive)
Intravesical
Submitted – BLA; Fast December Track 2021
trivalent hepatitis B vaccine
VBI Vaccines
Hepatitis B infection (prevention)
IM
Submitted – BLA
12/01/2021
pegfilgrastim (biosimilar to Amgen’s Neulasta)
Kashiv/Amneal
Neutropenia/leukopenia
SC
Submitted – BLA
January 2022
testosterone undecanoate
Marius
Hypogonadism
Oral
Submitted – 505(b)(2) NDA
January 2022
daridorexant
Idorsia
Insomnia
Oral
Submitted – NDA
01/08/2022
bevacizumab (biosimilar to Genentech’s Avastin)
Centus/AstraZeneca
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Submitted – BLA
Pending
bevacizumab (biosimilar to Genentech’s Avastin)
Merck/Samsung Bioepis
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Submitted – BLA
Pending
bevacizumab (biosimilar to Genentech’s Avastin)
Mylan/Biocon
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Submitted – BLA
Pending
26 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
filgrastim (biosimilar to Amgen’s Neupogen)
Apotex
Neutropenia/leukopenia
SC
Submitted – BLA
Pending
filgrastim (biosimilar to Amgen’s Neupogen)
Kashiv/Amneal
Neutropenia/leukopenia
IV, SC
Submitted – BLA
Pending
pegfilgrastim (biosimilar to Amgen’s Neulasta)
Apotex
Neutropenia/leukopenia
SC
Submitted – BLA
Pending
romiplostim (Nplate®)
Amgen
Acute radiation syndrome
SC
Submitted – sBLA; Orphan Drug; Priority Review
01/28/2021
Eli Lilly
Atopic dermatitis
Oral
Submitted – sNDA
Feb-Apr 2021
cabozantinib (Cabometyx )
Exelixis
RCC (advanced, in combination with nivolumab)
Oral
Submitted – sNDA; Priority Review
02/20/2021
nivolumab (Opdivo®)
Bristol-Myers Squibb
RCC (advanced, in combination with cabozantinib)
IV
Submitted – sBLA; Breakthrough Therapy; Fast Track; Priority Review
02/20/2021
cemiplimab-rwlc (Libtayo®)
Regeneron
NSCLC (1st-line, locally advanced or metastatic, ≥ 50% PD-L1 expression)
IV
Submitted – sBLA; Priority Review
02/26/2021
valsartan/sacubitril (Entresto®)
Novartis
Heart failure with preserved ejection fraction (HFpEF)
Oral
Submitted – sNDA; Fast Track
02/26/2021
cemiplimab-rwlc (Libtayo)
Regeneron
Basal cell carcinoma
IV
Submitted – sBLA; Priority Review
03/03/2021
axicabtagene ciloleucel (Yescarta)
Gilead
Follicular lymphoma, Marginal zone lymphoma (relapsed/refractory after ≥ 2 lines of therapy for both)
IV
Submitted – sBLA; Breakthrough Therapy; Orphan Drug; Priority Review
03/05/2021
rilonacept (Arcalyst®)
Regeneron
Recurrent pericarditis
SC
Submitted – sBLA; Breakthrough Therapy; Orphan Drug; Priority Review
03/19/2021
mirabegron (Myrbetriq®) tablet, oral suspension
Astellas
Neurogenic detrusor overactivity (ages ≥ 3 years)
Oral
Submitted – sNDA; Priority Review
03/28/2021
pembrolizumab (Keytruda®)
Merck
Breast cancer (highrisk early-stage TNBC, in combination with chemotherapy as neoadjuvant treatment); Breast cancer (adjuvant monotherapy after surgery)
IV
Submitted – sBLA; seeking Accelerated Approval; Breakthrough Therapy; Priority Review
03/29/2021
treprostinil (Tyvaso®) solution
United Therapeutics
Interstitial lung diseaseassociated pulmonary hypertension
Inhaled
Submitted – sNDA
April 2021
dapagliflozin (Farxiga®)
AstraZeneca
CKD (with or without T2DM)
Oral
Submitted – sNDA; Breakthrough Therapy; Fast Track; Priority Review
Apr-Jun 2021
tofacitinib (Xeljanz®/ Xeljanz XR®)
Pfizer
Axial spondyloarthritis
Oral
Submitted – sNDA
Apr-Jun 2021
Submitted (Supplementals)
baricitinib (Olumiant®) ®
27 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
upadacitinib (Rinvoq™)
Abbvie
PsA
Oral
Submitted – sNDA
04/01/2021
pimavanserin (Nuplazid®)
Acadua
Dementia-related hallucinations and delusions
Oral
Submitted – sNDA; Breakthrough Therapy
04/02/2021
pembrolizumab (Keytruda)
Merck
Esophageal cancer (locally advanced/ metastatic, 1stline, in combination with platinum- & fluoropyrimidine-based chemotherapy)
IV
Submitted – sBLA; Priority Review
04/13/2021
lorlatinib (Lorbrena®)
Pfizer
NSCLC (1st-line, ALK+)
Oral
Submitted – sNDA; Orphan Drug; Priority Review; RTOR
04/27/2021
tenapanor (Ibsrela®)
Ardelyx
Hyperphosphatemia (CKD dialysis-dependent patients)
Oral
Submitted – sNDA
04/29/2021
pirfenidone (Esbriet®)
Genentech
Interstitial lung disease (unclassified)
Oral
Submitted – sNDA; Breakthrough Therapy; Orphan Drug; Priority Review
May 2021
teriflunomide (Aubagio®)
Sanofi
MS (pediatric)
Oral
Submitted – sNDA
May 2021
nivolumab (Opdivo)
Bristol-Myers Squibb
Esophageal/ gastroesophageal junction cancer (resected, adjuvant setting, after neoadjuvant chemoradiation therapy)
IV
Submitted – sBLA; Orphan Drug; Priority Review
05/20/2021
nivolumab (Opdivo)
Bristol-Myers Squibb
Gastric/gastroesophageal junction cancer/ esophageal adenocarcinoma (advanced/metastatic, in combination with fluoropyrimidine- & platinum-based chemotherapy)
IV
Submitted – sBLA; Orphan Drug; Priority Review
05/25/2021
semaglutide (Ozempic®)
Novo Nordisk
Obesity/overweight (≥ 1 weight-related comorbidity)
SC
Submitted – sNDA; Priority Review
06/04/2021
upadacitinib (Rinvoq)
Abbvie
Axial spondyloarthritis
Oral
Submitted – sNDA
06/25/2021
secnidazole (Solosec )
Lupin
Trichomoniasis (adults and adolescents)
Oral
Submitted – sNDA
06/30/2021
mepolizumab (Nucala)
GlaxoSmithKline
Nasal polyposis
SC
Submitted – sNDA
Jul-Aug 2021
ruxolitinib (Jakafi) tablet
Incyte
GVHD (chronic)
Oral
Submitted – sNDA; Orphan Drug
Jul-Dec 2021
upadacitinib (Rinvoq)
Abbvie
Atopic dermatitis (adults & adolescents)
Oral
Submitted – sNDA; Breakthrough Therapy
08/19/2021
empagliflozin (Jardiance®)
Boehringer Ingelheim
Chronic heart failure (reduced ejection fraction)
Oral
Submitted – sNDA; Fast Track
September 2021
®
28 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
daratumumab/ hyaluronidase-fihj (Darzalex Faspro™)
Janssen
Multiple myeloma (relapsed/refractory, in combination with pomalidomide and dexamethason)
SC
Submitted – sBLA
09/21/2021
sacituzumab govitecanhziy (Trodelvy®)
Immunomedics
Breast cancer (metastatic, TNBC, ≥ 2 prior therapies)
IV
Submitted – sBLA; Breakthrough Therapy; Fast Track
10/08/2021
Phase 3 (New Drugs) abaloparatide-TD
Radius Health
Osteoporosis/osteopenia
Transdermal
Phase 3 – NDA
TBD
abicipar pegol
Allergan
Wet AMD
Intravitreal
Phase 3 – BLA
TBD
acoramidis
Eidos
Transthyretin amyloid cardiomyopathy (ATTRCM)
Oral
Phase 3 – NDA
TBD
Ad26.COV2-S vaccine
Janssen
COVID-19
IM
Phase 3 – BLA
TBD
adagrasib
Mirati
NSCLC
Oral
Phase 3 – NDA
TBD
adalimumab (biosimilar to Abbvie’s Humira)
Coherus
RA; AS; PSO; PsA; JIA; CD; UC
SC
Phase 3 – BLA
TBD
adalimumab (biosimilar to Abbvie’s Humira)
Fresenius
RA; AS; PSO; PsA; JIA; CD; UC
SC
Phase 3 – BLA
TBD
adalimumab (biosimilar to Abbvie’s Humira)
Mylan/Biocon
Hidradenitis suppurativa; Uveitis
SC
Phase 3 – BLA
TBD
adrabetadex
Mallinckrodt
Niemann-Pick disease
Intrathecal
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug; Rare Pediatric Disease
TBD
aflibercept (biosimilar to Regeneron’s Eylea®)
Mylan/Momenta
Diabetic macular edema
Intravitreal
Phase 3 – BLA
TBD
aflibercept (biosimilar to Regeneron’s Eylea)
Samsung Bioepis/Biogen
Wet AMD
Intravitreal
Phase 3 – BLA
TBD
aflibercept (biosimilar to Regeneron’s Eylea)
Santo/Formycon
Wet AMD
Intravitreal
Phase 3 – BLA
TBD
AKCEA-TTR-LRx
Akcea
Transthyretin amyloid cardiomyopathy (ATTR-CM, wild-type or hereditary)
N/A
Phase 3 – NDA
TBD
alpha 1 proteinase inhibitor
Kamada
Alpha-1 antitrypsin deficiency-related lung disease
Inhaled
Phase 3 – BLA; Orphan Drug
TBD
antolimab
Allakos
Gastroenteritis (eosinophilic)
IV
Phase 3 – BLA; Orphan Drug
TBD
apolipoprotein A-I (human)
CSL
Atherosclerosis
IV
Phase 3 – BLA
TBD
apomorphine continuous infusion pump
Supernus
Parkinson’s disease
SC
Phase 3 – NDA
TBD
ARO-AAT
Arrowhead
Alpha-1 antitrypsin deficiency-related liver disease
SC
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
asciminib
Novartis
Chronic myelogenous leukemia
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
ataluren
PTC
DMD
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
atogepant
Allergan
Migraine prevention
Oral
Phase 3 – NDA
TBD
29 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
autologous genetically modified human dermal fibroblasts
Castle Creek
Epidermolysis bullosa
Intradermal
Phase 3 – BLA; Fast Track; Orphan Drug; RMAT
TBD
AZD1222 vaccine
AstraZeneca
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
bamlanivimab
Eli Lilly
COVID-19
IV
Phase 3 – BLA
TBD
bardoxolone methyl
Reata
Alport syndrome; Polycystic kidney disease
Oral
Phase 3 – NDA; Orphan Drug
TBD
beremagene geperpavec
Krystal
Epidermolysis bullosa
Topical
Phase 3 – BLA; Fast Track; Orphan Drug; RMAT
TBD
betibeglogene autotemcel (Zynteglo)
Bluebird Bio
Sickle cell disease; Thalassemia
IV
Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug; RMAT
TBD
bevacizumab-vikg
Outlook
Wet AMD
Intravitreal
Phase 3 – BLA
TBD
bexagliflozin
Theracos
T2DM
Oral
Phase 3 – NDA
TBD
bimekizumab
UCB
Axial spondyloarthritis; Hidradenitis suppurativa; PsA
SC
Phase 3 – BLA
TBD
bintrafusp alfa
Merck
Biliary tract cancer
IV
Phase 3 – BLA; Orphan Drug
TBD
BIVV 001
Sanofi
Hemophilia A
IV
Phase 3 – BLA; Orphan Drug
TBD
brensocatib
Insmed
Bronchiectasis
Oral
Phase 3 – NDA; Breakthrough Therapy
TBD
budesonide
Calliditas
Immunoglobulin A (IgA) nephropathy (Berger’s disease)
Oral
Phase 3 – 505(b)(2) NDA; Orphan Drug
TBD
cannabidiol gel
Zynerba
Fragile X syndrome
Topical
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
capivasertib
AstraZeneca
Breast cancer
Oral
Phase 3 – NDA
TBD
capsaicin
Centrexion
Osteoarthritis
Intraarticular
Phase 3 – 505(b)(2) NDA; Fast Track
TBD
carglumic acid
Recordati
Hyperammonaemia (autosomal disorder related)
Oral
Phase 3 – NDA; Orphan Drug
TBD
CD24Fc
OncoImmune
COVID-19
IV
Phase 3 – BLA
TBD
ceftobiprole medocaril
Basilea
ABSSSI; Bacteremia
IV
Phase 3 – NDA; Fast Track; QIDP
TBD
ceftriaxone wearable micropump
scPharmaceuticals
Gram+/Gram- infection
SC
Phase 3 – NDA
TBD
cenicriviroc mesylate
Allergan
NASH
Oral
Phase 3 – NDA; Fast Track
TBD
ciltacabtagene autoleucel
Janssen
Multiple myeloma (relapsed/refractory)
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
30 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
cipaglucosidase alfa
Amicus
Pompe disease (in combination with miglustat)
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
clindamycin phosphate gel
Daré
Bacterial vaginosis
Intravaginal
Phase 3 – NDA; Fast Track; QIDP
TBD
CM-AT (pancreatic enzyme)
Curemark
Autism spectrum disorder
Oral
Phase 3 – BLA; Fast Track
TBD
crinecerfont
Neurocrine Biosciences
Congenital adrenal hyperplasia
Oral
Phase 3 – NDA
TBD
crisantaspase
Jazz
ALL; Lymphoblastic lymphoma
IM, IV
Phase 3 – BLA; Fast Track
TBD
crovalimab
Genentech
Paroxysmal nocturnal hemoglobinuria
IV, SC
Phase 3 – BLA; Orphan Drug
TBD
dactolisib
Restorbio
COVID-19
Oral
Phase 3 – NDA
TBD
dalcetrapib
Dalcor
Acute coronary syndrome (ADCY9 AA genotype)
Oral
Phase 3 – NDA
TBD
daprodustat
GlaxoSmithKline
Anemia due to CKD (dialysis-dependent, dialysis-independent)
Oral
Phase 3 – NDA
TBD
dehydrated human amnion chorion membrane
Mimedx
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
dersimelagon
Mitsubishi Tanabe
Porphyria
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
deucravacitinib
Bristol-Myers Squibb
PSO
Oral
Phase 3 – NDA
TBD
dexamethasone SR
Otonomy
Meniere’s disease
Intratympanic
Phase 3 – 505(b)(2) NDA; Fast Track
TBD
dexmedetomidine
Bioxcel
Schizophrenia-related acute aggitation
SL
Phase 3 – 505(b)(2) NDA; Fast Track
TBD
dextromethorphan/ bupropion
Axsome
MDD
Oral
Phase 3 – 505(b)(2) NDA; Breakthrough Therapy; Fast Track
TBD
dociparstat
Chimerix
COVID-19
IV
Phase 3 – NDA
TBD
donaperminogene seltoplasmid
Helixmith
Diabetic foot ulcers
IM
Phase 3 – BLA
TBD
doravirine/islatravir
Merck
HIV-1 infection
Oral
Phase 3 – NDA
TBD
dovitinib lactate
Allarity
RCC
Oral
Phase 3 – NDA
TBD
dust mite immunotherapy
Stallergenes Greer
Allergic rhinitis
SL
Phase 3 – BLA
TBD
EB-101 (gene therapy)
Abeona
Epidermolysis bullosa
Surgical application
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug; RMAT
TBD
eculizumab (biosimilar to Alexion’s Soliris)
Amgen
Paroxysmal nocturnal hemoglobinuria
IV
Phase 3 – BLA
TBD
efgartigimod
Argenx
Immune thrombocytopenic purpura
IV, SC
Phase 3 – BLA; Orphan Drug
TBD
31 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
elivaldogene autotemcel (Lenti-D)
Bluebird Bio
Adrenoleukodystrophy
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
enmetazobactam
Allecra
UTI (complicated)
IV
Phase 3 – NDA; Fast Track; QIDP
TBD
ensifentrine
Verona
COPD
Inhaled
Phase 3 – NDA
TBD
EP-2101 therapeutic vaccine
OSE Immunotherapeutics
NSCLC
SC
Phase 3 – NDA; Orphan Drug
TBD
episalvan
Amryt
Epidermolysis bullosa
Topical
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
eprenetapopt
Aprea
Myelodysplastic syndrome
IV
Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
etanercept (biosimilar to Amgen’s Enbrel)
Coherus
RA; Polyarticular 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
SC
Phase 3 – BLA
TBD
exebacase
Contrafect
Staphylococcus aureus bacteremia
IV
Phase 3 – BLA; Breakthrough Therapy; Fast Track
TBD
faricimab
Genentech
Diabetic macular edema; Wet AMD
Intravitreal
Phase 3 – BLA
TBD
fasinumab
Regeneron
Osteoarthritis
SC
Phase 3 – BLA
TBD
favipiravir
Dr. Reddy’s
COVID-19; Influenza
Oral
Phase 3 – NDA
TBD
fezagepras
Liminal
Alström syndrome
Oral
Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease
TBD
fezolinetant
Astellas
Menopause vasomotor symptoms
Oral
Phase 3 – NDA
TBD
fidanacogene elaparvovec
Pfizer
Hemophilia B
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
filgotinib
Gilead
RA; CD; UC
Oral
Phase 3 – NDA
TBD
firmacute eubacterial spores
Seres
C. difficile infection (recurrent)
Oral
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
fitusiran
Sanofi
Hemophilia A and B (with and without inhibitors)
SC
Phase 3 – NDA; Orphan Drug
TBD
follitropin alfa (biosimilar to EMD Serono’s Gonal-F®)
Allergan
Female reproductive disorder
SC
Phase 3 – BLA
TBD
follitropin alfa (biosimilar to EMD Serono’s Gonal-F)
Finox
Female reproductive disorder
SC
Phase 3 – BLA
TBD
follitropin delta
Ferring
Female reproductive disorder
IV
Phase 3 – BLA
TBD
32 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
ganaxolone
Marinus
Status epilepticus; CDKL5 deficiency disorderrelated seizures
IV, Oral
Phase 3 – NDA; Orphan Drug
TBD
gantenerumab
Genentech
Alzheimer’s disease
SC
Phase 3 – BLA
TBD
gepotidacin
GlaxoSmithKline
UTI (uncomplicated)
Oral
Phase 3 – NDA; QIDP
TBD
giroctocogene fitelparvovec
Pfizer
Hemophilia A
IV
Phase 3 – BLA; Fast Track; Orphan Drug; RMAT
TBD
givinostat
Italfarmaco
DMD
Oral
Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease
TBD
glatiramer acetate depot
Mylan
MS
IM
Phase 3 – NDA
TBD
glepaglutide
Zealand
Short bowel syndrome
SC
Phase 3 – NDA; Orphan Drug
TBD
glofitamab
Genentech
DLBCL
IV
Phase 3 – BLA
TBD
hydrocortisone granules
Diurnal
Congenital adrenal hyperplasia
Oral
Phase 3 – 505(b)(2) NDA; Orphan Drug
TBD
hypericin
Soligenix
Cutaneous T cell lymphoma
Topical
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
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
insulin icodec (onceweekly)
Novo Nordisk
T2DM
SC
Phase 3 – NDA
TBD
insulin tregopil
Biocon
T2DM
Oral
Phase 3 – NDA
TBD
ipatasertib
Genentech
Breast cancer (TNBC/HR+, 1st-line, in combination with chemotherapy); Prostate cancer
Oral
Phase 3 – NDA
TBD
KSI-301
Kodiak
Diabetic macular edema; Macualar edema related to retinal vein occlusion
Intravitreal
Phase 3 – BLA
TBD
L-citrulline
Asklepion
Acute lung injury
IV
Phase 3 – NDA; Orphan Drug
TBD
Lactobacillus reuteri
Infant Bacterial
Necrotizing enterocol
Oral
Phase 3 – BLA; Orphan Drug
TBD
lebrikizumab
Dermira
Atopic dermatitis
SC
Phase 3 – BLA; Fast Track
TBD
lecanemab
Eisai
Alzheimer’s disease
IV
Phase 3 – BLA
TBD
lenacapavir
Viiv
HIV-1 infection (heavily treatment-experienced)
Oral (lead-in), SC
Phase 3 – NDA; Breakthrough Therapy
TBD
lenadogene nolparvovec (GS010)
Gensight
Leber’s hereditary optic neuropathy
Intravitreal
Phase 3 – BLA; Orphan Drug
TBD
lenzilumab
Humanigen
COVID-19
IV
Phase 3 – BLA
TBD
33 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
leriglitazone
Minoryx
Adrenoleukodystrophy
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
leronlimab
Cytodyn
HIV-1 infection treatment (in combination therapy with HAART, highly treatment-experienced); COVID-19
SC
Phase 3 – BLA; Fast Track
TBD
levodopa/carbidopa patch pump
Mitsubishi Tanabe
Parkinson’s disease
SC
Phase 3 – 505(b)(2) NDA
TBD
levoketoconazole
Strongbridge
Cushing’s syndrome
Oral
Phase 3 – 505(b)(2) NDA; Orphan Drug
TBD
ligelizumab
Novartis
Urticaria
SC
Phase 3 – BLA; Breakthrough Therapy
TBD
linrodostat
Bristol-Myers Squibb
Bladder cancer
Oral
Phase 3 – NDA
TBD
linzagolix
Obseva
Endometriosis; Uterine fibroids
Oral
Phase 3 – NDA
TBD
lorecivivint
Samumed
Osteoarthritis (knee)
Intraarticular
Phase 3 – NDA
TBD
lucerastat
Idorsia
Fabry’s disease
Oral
Phase 3 – NDA; Orphan Drug
TBD
lutetium 177Lu-PSMA-617
Novartis
Prostate cancer
IV
Phase 3 – NDA
TBD
LYS-SAF302
Sarepta
Mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo A syndrome)
Intracerebral
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
magrolimab
Forty Seven
Myelodysplastic syndrome
IV
Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
maribavir
Takeda
Cytomegalovirus infection treatment
Oral
Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
masitinib
AB Science
Asthma; MS
Oral
Phase 3 – NDA
TBD
mavacamten
Myokardia
Obstructive hypertrophic cardiomyopathy
Oral
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
MBG453
Novartis
Myelodysplastic syndrome
IV
Phase 3 – BLA
TBD
metachromatic leukodystrophy gene therapy
Orchard
Metachromatic leukodystrophy
IV
Phase 3 – BLA; Orphan Drug; Rare Pediatric Disease; RMAT
TBD
microbiota suspension
Ferring
C. difficile infection (recurrent)
Rectal
Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
minocycline/edetate/ethyl alcohol
Citius
Catheter-related bloodstream infection (CRBSI)
IV
Phase 3 – 505(b)(2) NDA; Fast Track; QIDP
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
34 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
mitapivat
Agios
Pyruvate kinase deficiency
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
mobocertinib
Takeda
NSCLC
Oral
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
Moderna COVID-19 vaccine (mRNA-1273)
Moderna/NIAID
COVID-19
IM
Phase 3 – BLA; Fast Track
TBD
nabiximols
GW
MS-related spasticity
Oral submucosal
Phase 3 – NDA
TBD
nalbuphine ER
Trevi
Pruritus
Oral
Phase 3 – NDA
TBD
napabucasin
Sumitomo Dainippon
CRC
Oral
Phase 3 – NDA
TBD
natalizumab (biosimilar to Biogen’s Tysabri®)
Novartis
MS
IV
Phase 3 – BLA
TBD
nedosiran
Dicerna
Hyperoxaluria
SC
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug; Rare Pediatric Disease
TBD
nemolizumab
Galderma
Atopic dermatitis
SC
Phase 3 – BLA
TBD
nirsevimab
AstraZeneca
RSV infection prevention
IM
Phase 3 – BLA; Breakthrough Therapy; Fast Track
TBD
nomacopan
Akari
Paroxysmal nocturnal hemoglobinuria
SC
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
NVX-CoV2373 vaccine
Novavax
COVID-19
IM
Phase 3 – BLA
TBD
odevixibat
Albireo
Alagille syndrome; Oral Biliary Atresia (post Kasai hepatoportoenterostomy)
Phase 3 – NDA; Orphan Drug
TBD
olipudase alfa
Sanofi
Niemann-Pick disease
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
ondansetron ER once-daily
RedHill
Gastroenteritis
Oral
Phase 3 – 505(b)(2) NDA
TBD
oteseconazole
Mycovia
Vulvovaginal candidiasis
Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
OTL-103
Orchard
Wiskott-Aldrich syndrome IV
Phase 3 – BLA; Orphan Drug; RMAT
TBD
oxalobacter formigenes
Oxthera
Hyperoxaluria
Oral
Phase 3 – BLA; Orphan Drug
TBD
pacritinib
CTI
COVID-19; Myelofibrosis
Oral
Phase 3 – NDA
TBD
palovarotene
Ipsen
Fibrodysplasia ossificans progressiva
Oral
Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
pamrevlumab
Fibrogen
DMD; Idiopathic pulmonary fibrosis
IV
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
pegzilarginase
Aeglea
Arginase 1 deficiency
IV
Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
35 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
pevonedistat
Takeda
Myelodysplastic syndrome
IV
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
PF-06939926
Pfizer
DMD
IV
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
Pfizer-Biontech COVID-19 vaccine (BNT162b2)
Pfizer/Biontech
COVID-19
IM
Phase 3 – BLA; Emergency Use Authorization
TBD
plinabulin
Beyondspring
NSCLC; Neutropenia/ leukopenia
IV
Phase 3 – NDA; Breakthrough Therapy
TBD
pollinex quattro grass
Allergy
Allergic rhinitis
SC
Phase 3 – BLA
TBD
potassium citrate/ potassium bicarbonate
Advicenne
Renal tubular acidosis
Oral
Phase 3 – NDA
TBD
PTI-NC-733
Proteostasis
CF
Oral
Phase 3 – NDA; Fast Track
TBD
ranibizumab (biosimilar to Genentech’s Lucentis)
Coherus
Wet AMD
Intravitreal
Phase 3 – BLA
TBD
ranibizumab (biosimilar to Genentech’s Lucentis)
STADA Arzneimittel/ Bausch
Wet AMD
Intravitreal
Phase 3 – BLA
TBD
ranibizumab intravitreal implant
Genentech
Wet AMD
Intravitreal
Phase 3 – BLA
TBD
REGN-COV2
Regeneron
COVID-19
SC
Phase 3 – BLA
TBD
relacorilant
Corcept
Cushing’s syndrome
Oral
Phase 3 – NDA; Orphan Drug
TBD
relugolix/estradiol/ norethindrone
Myovant
Endometriosis
Oral
Phase 3 – NDA
TBD
reproxalap
Aldeyra
Dry eye syndrome
Ophthalmic
Phase 3 – NDA
TBD
resmetirom
Madrigal
NASH
Oral
Phase 3 – NDA; Fast Track
TBD
ridinilazole
Summit
C. difficile-associated diarrhea
Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
rigosertib
Onconova
Myelodysplastic syndrome
IV
Phase 3 – NDA; Orphan Drug
TBD
rilzabrutinib
Principia
Pemphigus vulgaris
Oral
Phase 3 – NDA; Orphan Drug
TBD
ritlecitinib
Pfizer
Alopecia areata
Oral
Phase 3 – NDA; Breakthrough Therapy
TBD
rituximab (biosimilar to Genentech’s Rituxan)
Archigen
RA; CLL/SLL; NHL (indolent); ANCAassociated vasculitis
IV
Phase 3 – BLA
TBD
roflumilast cream
Arcutis
PSO
Topical
Phase 3 – NDA
TBD
ruxolitinib cream
Incyte
Atopic dermatitis; Vitiligo
Topical
Phase 3 – NDA
TBD
seladelpar
Cymabay
Primary biliary cholangitis Oral
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
sepofarsen
Proqr
Leber’s congenital amaurosis
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
36 | MAGELLANRX.COM
Intravitreal
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
sodium hyaluronate/ triamcinolone hexacetonide
Anika
Osteoarthritis (knee)
Intraarticular
Phase 3 – NDA
TBD
sodium thiosulfate
Fennec
Chemotherapy-induced ototoxicity prevention
IV
Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
sofpironium
Brickell
Axillary hyperhidrosis
Topical
Phase 3 – NDA
TBD
sotagliflozin
Lexicon
Heart failure in patients with T2DM
Oral
Phase 3 – NDA; Orphan Drug
TBD
sparsentan
Retrophin
Focal segmental glomerulosclerosis; Immunoglobulin A (IgA) nephropathy (Berger’s disease)
Oral
Phase 3 – NDA; Orphan Drug
TBD
tapinarof
Roivant
PSO
Topical
Phase 3 – NDA
TBD
tebipenem pivoxil
Spero
UTI (complicated); Acute pyelonephritis
Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
tecarfarin
Espero
Anticoagulation
Oral
Phase 3 – NDA
TBD
teprasiran
Quark
Kidney injury prevention following cardiac surgery
IV
Phase 3 – NDA
TBD
tesetaxel
Odonate
Breast cancer
Oral
Phase 3 – NDA
TBD
tetrathiomolybdate
Alexion
Wilson’s disease
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
tezepelumab
Amgen
Asthma (severe, uncontrolled)
SC
Phase 3 – BLA; Breakthrough Therapy
TBD
timbetasin
Regenerx
Dry eye syndrome
Topical
Phase 3 – BLA
TBD
tiragolumab
Genentech
SCLC
IV
Phase 3 – BLA
TBD
tirzepatide
Eli Lilly
T2DM; Obesity
SC
Phase 3 – NDA
TBD
tisagenlecleucel-t (Kymriah)
Novartis
DLBCL (1st relapse)
IV
Phase 3 – BLA; Orphan Drug
TBD
tofersen
Biogen
Amyotrophic lateral sclerosis
Intrathecal
Phase 3 – NDA; Orphan Drug
TBD
tominersen
Genentech
Huntington’s disease
Intrathecal
Phase 3 – NDA; Orphan Drug
TBD
tonogenchoncel-L
Kolon Tissuegene
Osteoarthritis
Intraarticular
Phase 3 – BLA
TBD
tradipitant
Vanda
Atopic dermatitis; COVID-19; Emesis; Gastroparesis
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
trehalose
Seelos
Oculopharyngeal muscular dystrophy
IV
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
triamcinolone acetonide
Bausch Health
Uveitis
Intravitreal
Phase 3 – 505(b)(2) NDA
TBD
trientine tetrahydrochloride
GMP-Orphan
Wilson’s disease
Oral
Phase 3 – NDA
TBD
37 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
trofinetide
Acadia
Rett syndrome
Oral
Phase 3 – NDA; Fast Track; Orphan Drug; Rare Pediatric Disease
TBD
ublituximab
TG
MS
IV
Phase 3 – BLA
TBD
ublituximab + umbralisib
TG
CLL
IV + Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
ustekinumab (biosimilar to Janssen’s Stelara)
Amgen
PSO
IV, SC
Phase 3 – BLA
TBD
ustekinumab (biosimilar to Janssen’s Stelara)
Formycon
PSO
IV, SC
Phase 3 – BLA
TBD
valoctocogene roxaparvovec
Biomarin
Hemophilia A
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
vazegepant
Biohaven
Migraine treatment; COVID-19
Intranasal
Phase 3 – NDA
TBD
veliparib
Abbvie
Breast cancer; Ovarian cancer
Oral
Phase 3 – NDA; Orphan Drug
TBD
venglustat
Sanofi
GM2 gangliosidoses (TaySachs disease, Sandhoff disease, AB Variant); Polycystic kidney disease
Oral
Phase 3 – NDA; Orphan Drug
TBD
verbrinacogene setparvovec
Freeline
Hemophilia B
IV
Phase 3 – BLA
TBD
veverimer
Tricida
CKD-related metabolic acidosis
Oral
Phase 3 – NDA
TBD
VGX-3100 therapeutic vaccine
Inovio
Cervical dysplasia
IM
Phase 3 – BLA
TBD
(vic-)trastuzumab duocarmazine
Byondis
Breast cancer (HER2+)
IV
Phase 3 – BLA; Fast Track
TBD
volanesorsen
Akcea
Familial chylomicronemia syndrome
SC
Phase 3 – NDA; Orphan Drug
TBD
vonoprazan
Phathom
Esophagitis; H. pylori infection
Oral
Phase 3 – NDA; QIDP
TBD
vutrisiran
Alnylam
Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary); Transthyretin amyloid polyneuropathy
SC
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
zilucoplan
Ra
Myasthenia gravis
SC
Phase 3 – NDA; Orphan Drug
TBD
ziritaxestat
Galapagos
Idiopathic pulmonary fibrosis
Oral
Phase 3 – NDA; Orphan Drug
TBD
zoliflodacin
Entasis
Gonorrhea
Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
Phase 3 (Supplementals) anakinra (Kineret )
Swedish Orphan Biovitrum
COVID-19
SC
Phase 3 – sBLA
TBD
baricitinib (Olumiant)
Eli Lilly
Alopecia areata; COVID-19; SLE
Oral
Phase 3 – sNDA; Breakthrough Therapy
TBD
benralizumab (Fasenra®)
AstraZeneca
Nasal polyposis
SC
Phase 3 – sBLA
TBD
®
38 | MAGELLANRX.COM
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
brexpiprazole (Rexulti®)
Otsuka
Alzheimer’s diseaserelated agitation; PTSD
Oral
Phase 3 – sNDA; Fast Track
TBD
canakinumab (Ilaris®)
Novartis
COVID-19
SC
Phase 3 – sBLA
TBD
dapagliflozin (Farxiga)
AstraZeneca
Diabetic nephropathy; COVID-19
Oral
Phase 3 – sNDA; Breakthrough Therapy; Fast Track
TBD
dehydroepiandrosterone (Intrarosa®)
Millicent
Female sexual arousal disorder
Intravaginal
Phase 3 – sNDA
TBD
dupilumab (Dupixent®)
Sanofi
COPD; Eosinophilic esophagitis; Pruritus, Urticaris
SC
Phase 3 – sBLA
TBD
empagliflozin (Jardiance)
Boehringer Ingelheim
Chronic heart failure; CKD; Diabetic nephropathy
Oral
Phase 3 – sNDA; Fast Track
TBD
hydrogen peroxide (Eskata®)
Aclaris
Warts
Topical
Phase 3 – sNDA
TBD
immune globulin intravenous (human) 10% (Octagam®)
Octapharma
COVID-19
IV
Phase 3 – sBLA
TBD
L-lactic acid/citric acid/ potassium bitartrate (Phexxi®)
Evofem
Chlamydia trachomatis infection; Neisseria gonorrhoeae infection
Intravaginal
Phase 3 – sNDA; Fast Track; QIDP
TBD
lonafarnib (Zokinvy®)
Eiger
Hepatitis D infection
Oral
Phase 3 – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
mepolizumab (Nucala)
GlaxoSmithKline
COPD; Nasal polyposis
SC
Phase 3 – sBLA
TBD
meropenem/vaborbactam (Vabomere®)
Melinta
Bacteremia; HAP
IV
Phase 3 – sNDA; QIDP TBD
nitazoxanide (Alinia®)
Lupin
COVID-19; Influenza
Oral
Phase 3 – sNDA
TBD
obeticholic acid (Ocaliva )
Intercept
NASH
Oral
Phase 3 – sNDA; Breakthrough Therapy
TBD
omalizumab (Xolair®)
Genentech
Food allergies
SC
Phase 3 – sBLA; Breakthrough Therapy
TBD
ozanimod (Zeposia®)
Bristol-Myers Squibb
UC
Oral
Phase 3 – sNDA
TBD
patisiran (Onpattro )
Alnylam
Transthyretin amyloid cardiomyopathy (ATTR-CM, wild-type or hereditary)
IV
Phase 3 – sNDA
TBD
risankizumab-rzaa (Skyrizi®)
Abbvie
PsA; CD; UC
SC
Phase 3 – sBLA; Orphan Drug
TBD
rivaroxaban (Xarelto®)
Janssen
COVID-19
Oral
Phase 3 – sNDA
TBD
sacituzumab govitecanhziy (Trodelvy)
Immunomedics
Bladder cancer (3rd-line)
IV
Phase 3 – sBLA; Fast Track
TBD
selinexor (Xpovio®)
Karyopharm
Lipoarcoma
Oral
Phase 3 – sNDA; Orphan Drug
TBD
ticagrelor (Brilinta®)
AstraZeneca
Sickle cell disease
Oral
Phase 3 – sNDA
TBD
upadacitinib (Rinvoq)
Abbvie
CD; UC
Oral
Phase 3 – sNDA; Orphan Drug
TBD
valsartan/sacubitril (Entresto)
Novartis
Post-acute myocardial infarction
Oral
Phase 3 – sNDA
TBD
®
®
39 | MAGELLANRX.COM
PIPELINE DRUG LIST continued
Complete Response Letter (CRL)/Withdrawn Drugs NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
arbaclofen ER
Osmotica
MS-related spasticity
Oral
CRL
TBD
baloxavir marboxil (Xofluza®) oral granules, tablets
Genentech
Influenza (treatment, ages Oral 1-12 years; postexposure prophylaxis, ages 1 year to adults)
CRL
TBD
buprenorphine ER
Braeburn
Substance use disorder
SC
CRL
TBD
furosemide wearable pump Scpharmaceuticals
Congestive heart failure
SC
CRL
TBD
inclisiran
The Medicines Company
Dyslipidemia (for secondary prevention patients with ASCVD and familial hypercholesterolemia)
SC
CRL
TBD
naloxone (high dose)
US WorldMeds
Opioid overdoise
IM
CRL
TBD
sutimlimab
Sanofi
Cold agglutnin disease
IV
CRL
TBD
treprostinil dry powder
Liquidia
PAH
Inhaled
CRL
TBD
viloxazine
Supernus
ADHD (ages 6 to 17 years)
Oral
CRL
TBD
40 | MAGELLANRX.COM
GLOSSARY 6MWT 6 Minute Walking Test
CAP Community Acquired Pneumonia
ABSSSI Acute Bacterial Skin and Skin Structure Infection
CAR T Chimeric Antigen Receptor T-Cell
ACEI Angiotensin-Converting Enzyme Inhibitor ACR20 American College of Rheumatology 20% Improvement ACR50 American College of Rheumatology 50% Improvement ACR70 American College of Rheumatology 70% Improvement ADHD Attention Deficit Hyperactivity Disorder ADL Activities of Daily Living AED Anti-Epileptic Drug ALK Anaplastic Lymphoma Kinase ALL Acute Lymphoblastic Leukemia ALT Alanine Transaminase AMD Age-Related Macular Degeneration AML Acute Myeloid Leukemia ANCA Antineutrophil Cytoplasmic Antibodies ANDA Abbreviated New Drug Application ARB Angiotensin II Receptor Blocker ARNI Angiotensin Receptor II Blocker â&#x20AC;&#x201C; Neprilysin Inhibitor ART Antiretroviral Therapy ARV Antiretroviral AS Ankylosing Spondylitis ASCVD Atherosclerotic Cardiovascular Disease AST Aspartate Aminotransferase BCVA Best Corrected Visual Acuity BLA Biologics License Application BMI Body Mass Index BsUFA Biosimilar User Fee Act CABP Community Acquired Bacterial Pneumonia 41 | MAGELLANRX.COM
CD Crohn's Disease CDC Centers for Disease Control and Prevention CF Cystic Fibrosis CHF Congestive Heart Failure CI Confidence Interval CKD Chronic Kidney Disease CLL Chronic Lymphocytic Leukemia CNS Central Nervous System COPD Chronic Obstructive Pulmonary Disease COVID-19 Coronavirus Disease 2019 CRC Colorectal Cancer CRL Complete Response Letter CSF Colony Stimulating Factor CV Cardiovascular CVD Cardiovascular Disease DAS28-CRP Disease Activity Score-28 with C Reactive Protein DEA Drug Enforcement Administration DLBCL Diffuse Large B Cell Lymphoma DMARD Disease Modifying Antirheumatic Drug DMD Duchenne Muscular Dystrophy 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 estimated Glomerular Filtration Rate ER Extended-Release
GLOSSARY continued ESRD End-Stage Renal Disease
IGA Investigator's Global Assessment
FDA Food and Drug Administration
IM Intramuscular
FH Familial Hypercholesterolemia
ITP Immune Thrombocytopenic Purpura
FLT3 FMS-Like Tyrosine Kinase-3
ITT Intent-To-Treat
G-CSF Granulocyte Colony Stimulating Factor
IV Intravenous
GI Gastrointestinal
JIA Juvenile Idiopathic Arthritis
GIST Gastrointestinal Stromal Tumor
LDL Low-Density Lipoprotein
GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist
LDL-C Low-Density Lipoprotein Cholesterol
GM-CSF Granulocyte-Macrophage Colony Stimulating Factor
mAb Monoclonal Antibody
GVHD Graft Versus Host Disease H Half HAART Highly Active Antiretroviral Therapy HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HCC Hepatocellular Carcinoma HCP Healthcare Professional HCV Hepatitis C Virus HER Human Epidermal Growth Factor Receptor HER2 Human Epidermal Growth Factor Receptor 2
MACE Major Adverse Cardiovascular Events MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler MRI Magnetic Resonance Imaging MRSA Methicillin-Resistant Staphylococcus Aureus MS Multiple Sclerosis N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NDA New Drug Application NHL Non-Hodgkin Lymphoma
HFA Hydrofluoroalkane
NIAID National Institute of Allergy and Infectious Diseases
HIT Heparin Induced Thrombocytopenia
NSAID Non-Steroidal Anti-Inflammatory Drug
HIV Human Immunodeficiency Virus
NSCLC Non-Small Cell Lung Cancer
HIV-1 Human Immunodeficiency Virus-1
ODT Orally Disintegrating Tablet
HR Hazard Ratio
OR Odds Ratio
HSCT Hematopoietic Stem Cell Transplant
ORR Overall/Objective Response Rate
HTN Hypertension
OS Overall Survival
IBS Irritable Bowel Syndrome
PAH Pulmonary Arterial Hypertension
IBS-C Irritable Bowel Syndrome, Constipation Predominant
PARP Poly(ADP-ribose) polymerase
42 | MAGELLANRX.COM
PASI Psoriasis Area and Severity Index
GLOSSARY continued PASI 50 Psoriasis Area and Severity Index 50%
SCCHN Squamous Cell Cancer of the Head and Neck
PASI 70 Psoriasis Area and Severity Index 70%
SCLC Small Cell Lung Cancer
PASI 90 Psoriasis Area and Severity Index 90%
SCT Stem Cell Transplant
PASI 100 Psoriasis Area and Severity Index 100%
SGLT2 Sodium-Glucose Co-Transporter 2
PCI Percutaneous Coronary Intervention
SL Sublingual
PCSK9 Proprotein Convertase Subtilisin Kexin 9
SLE Systemic Lupus Erythematosus
PD-1 Programmed Death Protein 1
SLL Small Lymphocytic Lymphoma
PD-L1 Programmed Death-Ligand 1
sNDA supplemental New Drug Application
PDUFA Prescription Drug User Fee Application
SNRI Serotonin and Norepinephrine Reuptake Inhibitor
PFS Progression-Free Survival PGA Physician Global Assessment PsA Psoriatic Arthritis PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty
SOC Standard of Care sPGA static Physician Global Assessment SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection
PTSD Post-Traumatic Stress Disorder
T1DM Type 1 Diabetes Mellitus
Q Quarter
T2DM Type 2 Diabetes Mellitus
QIDP Qualified Infectious Diseases Product
TBD To Be Determined
QOL Quality of Life
TEAE Treatment-Emergent Adverse Events
R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone
TNBC Triple Negative Breast Cancer
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
43 | MAGELLANRX.COM
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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