October 2020
MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS
TABLE OF CONTENTS Introduction Pipeline Deep Dive
EDITORIAL STAFF Maryam Tabatabai, PharmD Editor in Chief Senior Director, Drug Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist
Keep on Your Radar
Consultant Panel
Pipeline Drug List
Michelle Booth, PharmD Director, Medical Pharmacy Strategy
Daphne Atria, PharmD, BCPS, CPE Strategic Clinical Pharmacist Consultant
Becky Borgert, PharmD, BCOP Director, Clinical Oncology Product Development
Glossary
Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist Robert Greer, RPh, BCOP Senior Director, Clinical Strategy and Programs Brian MacDonald, PharmD Senior Manager, Specialty Clinical Programs Troy Phelps Senior Director, COAR - Analytics
Nothing herein is or shall be construed as a promise or representation regarding past or future events and Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Magellan Rx Management.
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INTRODUCTION Welcome to the MRx Pipeline. This quarterly publication offers clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline. MRx Pipeline, our universal forecast, addresses trends applicable across market segments. Traditional and specialty drugs, agents under the pharmacy and medical benefits, new molecular entities, pertinent new and expanded indications for existing medications, and biosimilars are profiled in the report. Clinical analyses, financial outlook, and pre-regulatory status are considered as part of the evaluation process. The products housed in the MRx Pipeline have been researched in detail and developed in collaboration and in consultation with our internal team of clinical and analytics experts. Emerging therapeutics continue to grow and influence the clinical and financial landscape. Therefore, Magellan Rx Management has developed a systematic approach to determine the products with significant clinical impact. For the in-depth clinical evaluations, the products’ potential to meet an underserved need in the market by becoming the new standard of care, and the ability to replace existing therapies were investigated. The extent to which the pipeline drugs could shift market share on a formulary and their impact on disease prevalence were also important considerations. In order to assist payers with assessing the potential impact of these pipeline drugs, where available, a financial forecast has been included for select products. Primarily complemented by data from EvaluateTM, this pipeline report looks ahead at the 5-year projected annual US sales through the year 2024. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, such as the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business. So far in 2020, a total of 42 novel drugs have received FDA approval, including the first treatment for COVID-19. Last year at approximately the same timepoint, only 33 novel drugs had been approved, already representing a 21% increase in approvals in 2020. For the remainder of this year, 26 notable drugs filed with the agency are profiled, each of which has an anticipated FDA decision in 2020. The progress of these agents is being actively monitored through MRx Pipeline. In the past few years, game changers, such as chimeric antigen receptor (CAR) T therapies, have transformed the pipeline by delivery of drug therapy directly to the site of action in the body. As we look ahead, a continued trend toward the approval of specialty medications, drugs for rare and ultra rare diseases, growth of biosimilars, new treatment modalities using gene therapy, and additional CAR-T therapies are expected. Noteworthy pipeline trends to watch in 2020 include much-needed therapies and vaccines for COVID-19. There are also a number of first-time approvals anticipated in 2020 – the first-in-class oral agent for anemia in chronic kidney disease, first treatment for genetic deficiency obesity, first oral agent for hereditary angioedema, and the first injectable for dyslipidemia administered every 6 months. In the upcoming quarters, development of complex therapies, therapeutic options for rare hereditary diseases, oncology, immunology, neurology, and investigational agents will be on the MRx radar. Moreover, sprouting products for cardiology, ophthalmology, hematology, and women’s health await on the horizon. The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Novel agents that apply innovation to show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm.
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PIPELINE DEEP DIVE Objective evidence-based methodology was used to identify the Deep Dive drugs in the upcoming quarters. This section features a clinical overview and explores the potential place in therapy for these agents. Moreover, it addresses their FDA approval timeline and 5-year financial forecast.
SPECIALTY
PRIORITY REVIEW
88%
47% BIOSIMILAR
47%
BREAKTHROUGH THERAPY
12% ORPHAN DRUG
18%
pecialty drug names appear in ï‚« S magenta throughout the publication.
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NEUROLOGY
aducanumab IV Biogen/Eisai PROPOSED INDICATION Alzheimer’s disease (AD)
CLINICAL OVERVIEW
Aducanumab is a human monoclonal antibody that selectively binds to amyloid beta fibrils and soluble oligomers. Two identical 18-month, double-blind, phase 3 trials, EMERGE and ENGAGE, evaluated the efficacy and safety of aducanumab in patients 50 to 85 years of age with early AD (defined as mild cognitive impairment [MCI] due to AD and mild AD dementia). A total of 3,285 patients were randomized 1:1:1 to one of two dosing regimens of aducanumab (low-dose group, 3 mg/kg in patients who carry apolipoprotein E-e4 [APO-e4] gene variant or 6 mg/kg in patients who do not carry APO-e4; high-dose group, 6 mg/kg in patients who carry APOE-e4 or 10 mg/kg in patients who do not) or to placebo; at 18-months, the dose was increased to 10 mg/kg in all APO-e4 carriers. In March 2019, the initial interim analysis based on data from 1,748 patients who completed 18-months of therapy predicted that both trials would not meet their primary endpoint of Clinical Dementia Rating–Sum of Boxes (CDR-SB) score; this led to termination of both trials. However, further analyses revealed EMERGE demonstrated a significant reduction in CDR-SB with the highest dose of aducanumab 10 mg/kg compared to placebo, which was in part due to study protocol amendments that allowed more patients to receive the 10 mg/kg dose. Consequently, the trials were resumed. With 3 additional months of study data in the EMERGE trial, the high-dose aducanumab regimen demonstrated a significant 22% reduction in clinical decline (based on CDR-SB) compared to placebo (p=0.01). In addition, significant differences favoring the high-dose aducanumab versus placebo were seen with the following secondary measures: Alzheimer’s Disease Assessment Scale–Cognitive Subscale (difference, 27%; p=0.01), Alzheimer’s Disease Cooperative Study–Activities of Daily Living Inventory–Mild Cognitive Impairment (difference, 40%; p=0.001), and Mini Mental State Exam (difference, 18%; p=0.05). In ENGAGE, while trends in slowing decline of AD were seen with some secondary study endpoints, the aducanumab high-dose regimen failed to demonstrate a significant benefit in the primary endpoint of CDR-SB. In EMERGE and ENGAGE (reported as incidence in each trial, respectively), the most common TEAE with the aducanumab 10 mg/kg dose was transient amyloid-related imaging abnormalities-edema/effusion (ARIA-E; 34% and 35.5%), headache (19.4% and 20.4%), and amyloid-related imaging abnormalitiesmicrohemorrhage (ARIA-H; 18.6% and 17.6%). In the trials, 8.8% and 11.5% of patients, respectively, permanently discontinued treatment due to TEAEs, of which 6.6% and 7.3%, respectively, were due to ARIA. In each study, the incidence of all-cause death was low among patients treated with high-dose aducanumab (1.1% and 0.4%, respectively). Aducanumab was infused IV once monthly.
PLACE IN THERAPY
Alzheimer’s disease is a neurodegenerative disorder characterized by cognitive and memory decline, impairment of ADLs, and behavioral disturbances. It is the most common form of dementia. It is estimated to affect 5 million elderly people in the US, and the incidence is expected to nearly triple by 2050. Moreover, the presence of the APOE-e4 gene variant is considered a risk factor for developing AD. The gene variant is found in approximately 28% of the general public, and it is estimated to exist in 40% to 65% of patients diagnosed with AD. Presence of amyloid plaque as well as abnormalities of cholinergic, glutamate, and serotonin systems in the brain are thought to contribute to the development of AD. Current pharmacotherapy for AD includes oral acetylcholinesterase inhibitors (donepezil, galantamine, rivastigmine [also transdermal]), oral N-methylD-aspartate (NMDA) receptor antagonists (memantine), and a fixed-dose combination of the two classes (donepezil/memantine); all single-agent products are available as generics. Although troubled by mixed clinical results, if approved, aducanumab will be the first agent to treat early AD that reduces amyloid plaque. However, clearance of plaque is associated with vasogenic brain edema that limits the tolerable dose and 4 | magellanrx.com
aducanumab cont. PLACE IN THERAPY cont.
can lead to treatment discontinuation, particularly in patients with the APOE-e4 gene variant. As part of the FDA approval process, on November 6, 2020, the Peripheral and Central Nervous System Drugs Advisory Committee will review data for aducanumab as it relates to its clinical benefits and risks. Other agents that target amyloid beta protein in late-stage development for AD include gantenerumab, solanezumab, and BAN2401.
FDA APPROVAL TIMELINE March 7, 2021 ďƒź Fast Track
ďƒź Priority Review
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$0
$253
$595
$1,051
$1,651
The forecast is a projection of total US sales per year.
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MUSCULOSKELETAL
casimersen IV Sarepta PROPOSED INDICATION
Duchenne muscular dystrophy (DMD) with mutations amenable to exon 45 skipping
CLINICAL OVERVIEW
Casimersen is a phosphorodiamidate morpholino oligomer (PMO) that binds to exon 45 of dystrophin premRNA. The ongoing, double-blind, placebo-controlled, phase 3 ESSENCE trial is evaluating the safety and efficacy of casimersen in male patients with DMD. The baseline mean dystrophin protein level was 0.925% of normal. Based on interim data, casimersen therapy has resulted in a significant increase in mean dystrophin protein level to 1.736% of normal (p<0.001 compared to baseline); statistically significant differences from placebo have been observed at 48 weeks (p=0.009). Changes in functional abilities (e.g., 6MWT) and safety data were reported with this interim analysis. After 96 weeks of therapy, patients in the study are allowed to enter a 48-week, open-label extension phase during which all patients will receive active treatment. In the clinical trial, patients received an IV infusion of casimersen 30 mg/kg once per week for up to 96 weeks.
PLACE IN THERAPY
DMD is a rare, X-linked neuromuscular disorder characterized by progressive muscle degeneration and weakness. An estimated 400 to 600 boys are born with DMD each year in the US. In DMD, gene mutations lead to a lack of functional dystrophin protein involved in maintaining muscle fiber integrity. Onset of DMD occurs between 3 to 5 years of age. Most boys affected lose the ability to walk by age 12 years. Moreover, death due to respiratory or cardiac failure typically occurs before age 30. The standard of care for DMD includes systemic corticosteroids (prednisone, deflazacort [Emflaza®; the only FDA-approved steroid for DMD]) to delay progression of muscle weakness and improve respiratory function; however, side effects such as weight gain, bone fractures, and cataracts are associated with corticosteroid therapy. Approximately 8% of individuals diagnosed with DMD carry the exon 45 mutation. Casimersen allows sections of defective genetic code with the exon 45 mutation to be bypassed during the dystrophin manufacturing process, creating a partially functional dystrophin protein. Sarepta brought the first antisense oligonucleotide treatments for DMD to the US market, including eteplirsen (Exondys 51®) and golodirsen (Vyondys 53™), which target exons 51 and 53, respectively. Viltolarsen (Viltepso™) by Nippon Shinyaku is also approved and targets exon 53. Interim data reveals that casimersen increases the surrogate marker of mean dystrophin protein by 0.811 percentage points; this increase is in line with the the change in dystrophin protein that supported golodirsen’s FDA approval. Sarepta is seeking Accelerated Approval for casimersen. If casimersen is approved, it will be the first antisense oligonucleotide available in the US to treat DMD with mutations amenable to exon 45 skipping.
FDA APPROVAL TIMELINE February 25, 2021 Orphan Drug
Priority Review
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$0
$94
$136
$157
$187
The forecast is a projection of total US sales per year.
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CARDIOLOGY
inclisiran SC Novartis/The Medicines Company PROPOSED INDICATION
Treatment of dyslipidemia for secondary prevention of patients with atherosclerotic cardiovascular disease (ASCVD) and familial hypercholesterolemia (FH)
CLINICAL OVERVIEW
Inclisiran is a small interfering RNA (siRNA) therapeutic agent that inhibits hepatic production of PCSK9. The randomized, double-blind, placebo-controlled, phase 3 ORION trial program demonstrated LDL-C lowering with inclisiran in addition to background antilipid therapy in patients with ASCVD or FH and elevated LDL-C despite maximally tolerated statin therapy. In the ORION-10 (n=1,561) and ORION-11 (n=1,617) trials, inclisiran reduced LDL-C (primary endpoint) by 51.3% and 49.9%, respectively, compared to placebo at day 510 (p<0.001 for both). Decreased total cholesterol, non-HDL-C, apolipoprotein B, and triglycerides, as well as increased HDL-C, were reported with inclisiran compared to placebo. In patients with heterozygous FH (HeFH), the ORION-9 trial (n=482) reported a 47.9% reduction in LDL-C with inclisiran compared to placebo (p<0.001). The most common adverse effect reported with inclisiran was transient mild to moderate injection site reactions. In ORION-10 and ORION-11, overall deaths occurred at similar rates with inclisiran and placebo, and a prespecified analysis reported a lower rate of a CV composite endpoint with inclisiran compared to placebo (inclisiran, 7.4% and 7.8%, respectively; placebo, 10.2% and 10.3%, respectively); however, data were too limited to establish meaningful CV outcome conclusions. Inclisiran was HCP-administered as SC doses of 284 mg on days 1 and 90, and every 6 months thereafter.
PLACE IN THERAPY
CVD is the leading cause of death in the US. Individuals with established ASCVD are at high risk for a CV event. Also, FH occurs in approximately 830,000 Americans and is characterized by an elevated LDL-C and premature ASCVD. LDL-C lowering is a main objective of secondary prevention. Statins are the drugs of choice to reduce LDL-C levels, but their use is limited by resistance and intolerance to therapy. When target LDL-C is not achieved despite optimized oral statin therapy, oral ezetimibe or bempedoic acid (Nexletol®) or an injectable PCSK9-inhibiting mAb (alirocumab [Praluent®], evolocumab [Repatha®]) may be added. The phase 3 ORION program demonstrated robust LDL-C lowering with inclisiran as adjunct to background antilipid therapy. If approved, inclisiran will be the first-in-class siRNA-targeting PCSK9 agent and an alternative to PCSK9-inhibiting mAbs in patients who are not at goal with lifestyle modification and maximally tolerated statin therapy. Inclisiran offers every 6-month HCP-administered SC maintenance dosing, while PCSK9-inhibiting mAbs allow for once-monthly SC self-administration. Although there are no head-to-head phase 3 trials between the 2 antilipid classes, noncomparative study data report slightly less LDL-C lowering with inclisiran (range, 48% to 53%) versus PCSK9-inhibitor mAbs (range, 55% to 59%). Unlike statins, no liver, muscle, or kidney toxicity or need for dose adjustments in patients with renal impairments have been reported with inclisiran. While the PCSK9-inhibiting mAbs demonstrate risk reduction of CV events, CV outcomes trials with inclisiran are ongoing. In the United Kingdom (UK), Novartis is collaborating with the UK National Health Service on CV outcomes, and inclisiran will be provided to UK patients for secondary prevention through a population-level agreement pending UK regulatory approval and National Institute for Health and Care Excellence (NICE) assessment.
FDA APPROVAL TIMELINE December 2020 Orphan Drug
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$0
$136
$248
$376
$575
The forecast is a projection of total US sales per year.
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ONCOLOGY
naxitamab IV Y-mAbs Therapeutics PROPOSED INDICATION
Relapsed/refractory, high-risk neuroblastoma
CLINICAL OVERVIEW
Naxitamab is a humanized 3F8 monoclonal antibody (mAb) that targets ganglioside GD2, a cell surface antigen found on neuroblastoma tumors. Cytokines, such as GM-CSF, enhance the antibody-dependent cellular cytotoxicity (ADCC). Submission to the FDA for naxitamab was supported by a phase 2 clinical study (12-230) in patients with high-risk neuroblastoma. Patients were stratified into 3 groups. Among patients with primary refractory disease (n=28), naxitamab therapy resulted in an ORR of 78% and a 2-year PFS rate of 50%. In patients who relapsed and were resistant to salvage therapy (n=30), naxitamab led to an ORR of 37% and PFS of 36%. Lastly, in patients in second or later complete remission (n=44), naxitamab demonstrated a 52% PFS rate; however, this group was not evaluable for ORR. All patients received naxitamab 3 mg/kg administered IV over 30 minutes on days 1, 3, and 5 of each cycle. GM-CSF was also administered per the study protocol. Treatment was continued for 5 monthly cycles after complete response or very good partial response was achieved. Treatment was administered in an outpatient setting.
PLACE IN THERAPY
Neuroblastoma is a solid cancer that originates in immature neurons (neuroblasts) of the sympathetic nervous system. The rare disease accounts for approximately 6% of all childhood cancers and is the most common cancer in infants. The average age at diagnosis is 1 to 2 years, with the majority (90%) of cases identified by ≤ 5 years of age. Metastatic disease is often detected at diagnosis. Aggressive treatment is used for high-risk neuroblastoma. Induction and consolidation therapies include surgery, high-dose chemotherapy, radiation, and autologous stem cell transplant, while maintenance therapy includes isotretinoin, immune-activating cytokines (GM-CSF, interleukin-2), and GD2-targeting mAbs. If approved, naxitamab will be the second GD2-targeting mAb available to treat high-risk neuroblastoma, following dinutuximab (Unituxin®), a chimeric anti-GD2 mAb. Dinutuximab, given in combination with GMCSF, intereukin-2, and isotretinoin, is associated with serious infusion reactions, neurotoxicity, and antidrug antibodies (ADA; 20%). Naxitamab has high affinity for GD2 and was designed to reduce ADAs and boost ADCC while retaining complement mediated cytotoxicity (CMC). Low-immunogenicity with the product has been confirmed, and it appears to have an improved safety profile. Additionally, naxitamab may offer a more convenient administration regimen compared to dinutuximab; naxitamab is infused IV over 30 minutes as 3 doses/cycle, compared to 4 doses/cycle administered IV over 10 to 20 hours for dinutuximab. Notably, naxitamab has the potential to be administered on an outpatient basis, while dinutuximab must be given in a hospital setting.
FDA APPROVAL TIMELINE November 30, 2020
Breakthrough Therapy
Orphan Drug
Priority Review
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$7
$27
$63
$111
$165
The forecast is a projection of total US sales per year.
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Rare Pediatric Disease
ONCOLOGY
paclitaxel/encequidar oral Athenex PROPOSED INDICATION Breast cancer
CLINICAL OVERVIEW
Athenex's oral formulation of paclitaxel, a microtubule inhibitor, is co-administered with encequidar, a GI tract specific P-glycoprotein (P-gp) pump inhibitor that is designed to allow for oral absorption of paclitaxel. In a pivotal phase 3 trial, oral paclitaxel/encequidar (OPE) was compared to IV paclitaxel in patients with metastatic breast cancer, including patients with HR+/HER2-, HR+/HER2+, TNBC, and unknown HR/HER2 receptor status. Patients were randomized 2:1 to monotherapy with oral and IV formulations, respectively. In the intent-to-treat population (n=360), the primary endpoint of overall tumor response rate (ORR) was significantly higher with OPE compared to the IV paclitaxel (40.4% versus 25.6%; p=0.01). While analyses are ongoing, to date, the reported median duration of confirmed response is 39 weeks with OPE and 30.1 weeks with IV paclitaxel, and overall survival and PFS are favoring the oral over the IV formulation (p=0.035 and p=0.77, respectively). OPE was associated with less neuropathy; however, higher rates of hematologic adverse effects, including grade 3 neutropenia (14.8% versus 6.9% with IV paclitaxel) and one incidence of grade 5 anemia were reported with OPE. GI adverse events were more prevalent with the oral formulation compared to IV paclitaxel, including grade 3 diarrhea (4.9% versus 1.5%), nausea (3.8% versus 0.2%), and vomiting (4.9% versus 0.7%); the study protocol was amended to allow for use of medications to mitigate GI toxicities. OPE was administered 3 consecutive days per week as a 30 mg capsule of solubilized paclitaxel plus a 15 mg tablet of encequidar. The IV formulation was given every 3 weeks.
PLACE IN THERAPY
It is estimated that 279,100 people in the US will be diagnosed with breast cancer and 42,690 will die from the disease in 2020. It is the most common malignancy and second leading cause of death in American women. Taxanes are the cornerstone of breast cancer treatment. Paclitaxel is currently approved as an IV formulation for the treatment of metastatic breast cancer after failure of anthracycline-containing chemotherapy as well as first-line treatment for advanced or metastatic NSCLC and metastatic pancreatic cancers. In the pivotal trial, OPE demonstrated significant improvement in ORR compared to IV paclitaxel in patients with metastatic breast cancer. OPE leads to significantly less peripheral neuropathy, a long-term and potentially debilitating effect of IV paclitaxel. OPE is, however, associated with short-term hematologic and GI adverse effects. Intravenous paclitaxel is administered in combination with chemotherapy and/or immunomodulator therapy as every 1-week (fewer side effects) or 3-week regimens. OPE will replace IV paclitaxel within the chemotherapy regimen. It is also anticipated to reduce the need for adjunctive pretreatment with corticosteroids, which are typically given to mitigate the hypersensitivity reactions of IV paclitaxel solubilized with cremophor. OPE does have a complex oral administration regimen that requires fasting for 9 hours per day, 3 consecutive days per week. Oral paclitaxel is in phase 2 trials for cutaneous angiosarcoma and advanced solid tumors. The oral formulation has not been compared in clinical trials to the solvent-free IV nanoparticle albumin-bound (nab)-paclitaxel (AbraxaneÂŽ) formulation designed to reduce solvent-related side effects and improve tumor uptake.
FDA APPROVAL TIMELINE February 28, 2021 ď&#x192;ź Priority Review
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$0
$33
$116
$218
$322
The forecast is a projection of total US sales per year.
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ONCOLOGY/WOMEN’S HEALTH
relugolix oral Myovant
PROPOSED INDICATIONS
• Advanced prostate cancer (PC) • Uterine fibroid (UF)-related heavy menstrual bleeding
CLINICAL OVERVIEW
Relugolix is a gonadotropin-releasing hormone (GnRH) receptor antagonist. It reduces the production of testosterone and estradiol, hormones that stimulate growth of prostate cancer and uterine fibroids, respectively. Prostate cancer The 48-week, open-label, phase 3 HERO trial compared the safety and efficacy of relugolix with leuprolide in 930 men with androgen-sensitive advanced PC. Patients were randomized 2:1 to oral relugolix 360 mg on day 1, followed by 120 mg once daily, or SC leuprolide depot 22.5 mg every 3 months. After 48 weeks of therapy, relugolix was superior to leuprolide in maintaining testosterone suppression to castration levels (< 50 ng/dL) (96.7% versus 88.8%, respectively; p<0.001 for superiority). The study also reported similar rates of castration resistance-free survival between the groups (74% versus 75%, respectively). Greater testosterone recovery was seen with relugolix compared to leuprolide (testosterone level, 270.76 ng/dL versus 12.26 ng/dL, respectively) 90 days after therapy discontinuation. In addition, the risk of MACE was lower with relugolix versus leuprolide (2.9% versus 6.2%, respectively), including in men with a history of MACE (3.6% versus 17.8%, respectively). The overall safety profile was similar between the groups. In a phase 2 trial, the rate of sustained castration (testosterone < 50 ng/dL) was 93% with relugolix and 85% with the GnRH receptor antagonist degarelix with 24 weeks of therapy. Testosterone recovery was also more rapid with relugolix compared to degarelix (257 ng/dL versus 30 ng/dL, respectively) 12 weeks after therapy discontinuation. Uterine fibroids The 24-week, double-blind, phase 3 LIBERTY-1 and LIBERTY-2 trials assessed the safety and efficacy of a once-daily oral combination of relugolix 40 mg/estradiol 1 mg/norethindrone 0.5 mg for use in 770 premenopausal women with heavy menstrual bleeding related to UF. Response to therapy was defined as menstrual blood loss (MBL) < 80 mL and a ≥ 50% MBL reduction from baseline (primary endpoint). Both trials demonstrated that relugolix combination therapy led to a significantly greater response rate compared to placebo (LIBERTY-1, 73.4% versus 18.9%, respectively; LIBERTY-2, 71.2% versus 14.7%, respectively; p<0.0001 for both). In both trials, combination therapy resulted in a 84.3% mean reduction in MBL from baseline (p<0.0001). Secondary endpoints significantly favored relugolix combination therapy, including reduction in UF pain, improved anemia, change in uterine volume, and improved QOL; however, changes in UF volume and bone density did not differ significantly between the groups. Relugolix combination therapy was generally well tolerated.
PLACE IN THERAPY Prostate cancer
Prostate cancer is the second most common cancer in men in the US, with an anticipated 191,930 new cases and 33,330 deaths attributed to the disease in 2020. Treatment decisions should be individualized, based on disease stage/grade, and health, life expectancy, and patient preference. The majority of prostate cancers are hormonally dependent. Due to the hormone responsiveness of the tumor, androgen deprivation therapy (ADT) is a cornerstone of PC treatment, particularly for advanced or metastatic disease. ADT suppresses serum testosterone concentrations to castration levels (< 50 ng/dL) and can be accomplished with either bilateral orchiectomy or with the administration of a GnRH agonist (e.g., leuprolide [Lupron®, Eligard®], goserelin [Zoladex®], histrelin [Vantas®], triptorelin [Trelstar®]) or a GnRH antagonist (e.g., degarelix [Firmagon®]). ADT may be combined with radiation therapy for locally advanced PC.
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relugolix cont. PLACE IN THERAPY cont.
Relugolix is a GnRH antagonist, and unlike GnRH agonists, is not associated with testosterone flare due to the rise and fall of testosterone levels in patients with PC. Notably, relugolix's once-daily oral formulation may provide treatment flexibility and may be preferred over the injectable (IM, SC) formulations for the GnRH agonists administered every 1, 3, 4, or 6 months (depending on formulation). Goserelin is available as a 12-month SC implant. The GnRH antagonist degarelix is HCP-administered SC once monthly. Uterine fibroids Uterine fibroids (UFs) are the most common benign gynecologic tumors. They affect approximately 50% to 60% of premenopausal women and are more common among Black or African American women (80%). UFs are caused by overgrowth of connective tissue and smooth muscle in the uterus. While most cases are asymptomatic, UFs can lead to heavy menstrual bleeding and pain and can cause infertility. Treatment approaches include surgery (myomectomy, hysterectomy, uterine artery embolization) and medical therapies, which include oral contraceptives and progestin-releasing intrauterine devices (IUDs) to reduce heavy menstrual bleeding, NSAIDs to reduce pain, and mifepristone and GnRH agonists to shrink the fibroids. GnRH agonists are associated with bone loss and vasomotor symptoms that may be mitigated with low-dose “add-back” therapy using a combination of estrogen and progestin. In addition, tranexamic acid (Lysteda®) is approved for the treatment of cyclic heavy menstrual bleeding; it is dosed 3 times daily, and given for a maximum of 5 days during monthly menstruation. If approved for UF, relugolix will be the second oral GnRH antagonist combined with estradiol and norethindrone, following elagolix/estradiol/norethindrone (Oriahnn™), for the management of heavy menstrual bleeding associated with UFs in premenopausal women.
FDA APPROVAL TIMELINE
Prostate cancer – December 20, 2020 Priority Review Uterine fibroids – June 1, 2021
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$0
$17
$91
$181
$273
The forecast is a projection of total US sales per year.
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ONCOLOGY
trilaciclib IV G1 Therapeutics/Boehringer Ingelheim PROPOSED INDICATION
Myelopreservation in small cell lung cancer (SCLC) patients being treated with chemotherapy
CLINICAL OVERVIEW
Trilaciclib is a cyclin-dependent kinase 4 and 6 (CDK4/6) inhibitor that protects hematopoietic stem and progenitor cells by preventing proliferation during chemotherapy administration. Three double-blind, phase 2 trials evaluated the safety and efficacy of trilaciclib in patients with extensive stage SCLC. Patients were randomized to trilaciclib (n=123) or placebo (n=119) as add-on to standard chemotherapy that comprised etoposide/carboplatin ± atezolizumab, or topotecan. Pooled data from the 3 trials revealed trilaciclib resulted in a significant (p<0.05) decrease in myelosuppression as evident by incidence of the following (versus placebo, respectively): mean duration of severe neutropenia in cycle 1 (0 versus 4 days), severe neutropenia (11.4% versus 52.9%), G-CSF administration (28.5% versus 56.3%), and grade 3/4 thrombocytopenia (19.5% versus 36.1%). The overall survival and PFS were similar between the groups. Trilaciclib was administered IV prior to chemotherapy.
PLACE IN THERAPY
It is estimated that 228,820 new cases of lung cancer will be diagnosed in the US in 2020, of which approximately 13% will be SCLC. Nearly all cases of SCLC are due to cigarette smoking. SCLC is characterized by rapid growth and widespread metastases. Limited-stage SCLC indicates that only 1 side of the chest is affected and can likely be cured with chemotherapy and radiation therapy. SCLC is considered extensivestage when it has spread throughout the lung, to both sides of the chest, or to other parts of the body, and chemotherapy is used to control rather than cure the disease. First-line treatment for extensive-stage SCLC is chemotherapy (etoposide plus a platinum agent) with or without PD-L1 immunotherapy (atezolizumab [Tecentriq®] or durvalumab [Infinzi®]). While SCLC typically responds to initial therapy, long-term survival is rare. Moreover, the standard chemotherapy regimen is associated with myelosuppression, particularly in elderly patients. Neutropenia can be managed using colony stimulating growth factors (e.g., GM-CSF, G-CSF), but cumulative thrombocytopenia is a dose-limiting factor; in addition, routine use of growth factors is not recommended during initial therapy. If approved, trilaciclib will be the first CDK4/6 inhibitor to mitigate chemotherapy-induced myelosuppression. It has the potential to improve QOL and optimize the standard chemotherapy regimen as well as reduce the need for rescue interventions (growth factors, blood or platelet transfusions). While safety data for trilaciclib have not been announced, the CDK4/6 inhibitors as a class are generally well tolerated. Other agents in this class include abemaciclib (Verzenio®), palbociclib (Ibrance®), and ribociclib (Kisqali®). None of the 3 agents are approved for myelopreservation; rather all are indicated for the treatment of select patients with advanced/metastatic breast cancer. Trilaciclib is in phase 2 trials for the treatment of metastatic triple-negative breast cancer; final overall survival data are expected in Q4 2020.
FDA APPROVAL TIMELINE February 15, 2021
Breakthrough Therapy
Priority Review
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$0
$16
$62
$146
$312
The forecast is a projection of total US sales per year.
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CARDIOLOGY
vericiguat oral Merck/Bayer PROPOSED INDICATION Chronic heart failure (HF)
CLINICAL OVERVIEW
Vericiguat is a soluble guanylate cyclase (sGC) stimulator that increases the level of cyclic guanosine monophosphate (cGMP), an important messenger in several signaling pathways in the CV system. The randomized, double-blind, placebo-controlled, phase 3 VICTORIA trial evaluated the efficacy of vericiguat administered in combination with available HF therapies in 5,050 patients with worsening chronic HF (New York Heart Association [NYHA] II-IV) with reduced left ventricular ejection fraction (LVEF < 45%) and elevated natriuretic peptide levels (within the previous 30 days). Patients were at high risk of hospitalization and CV death following a recent HF decompensation. During a median of 10.8 months, vericiguat reduced the risk for HF hospitalization or CV death (composite primary endpoint) compared to placebo (35.5% versus 38.5%, respectively; HR, 0.9; 95% CI, 0.82 to 0.98; p=0.019). This difference was driven by a decrease in hospitalization for HF (HR, 0.9; 95% CI, 0.81 to 1; p=0.048); there was no significant difference in CV death between the groups (HR, 0.93; 95% CI, 0.81 to 1.06; p=0.269). Overall, the safety profile for vericiguat was similar to placebo. Vericiguat was studied at a dosage of up to 10 mg orally once daily.
PLACE IN THERAPY
An estimated 6.2 million adults in the US have HF. It is a chronic, progressive condition in which the heart does not pump enough blood to meet the body's demand and leads to a high degree of morbidity and mortality. Approximately 66% of HF cases have reduced LVEF (HFrEF). Risk factors for HF include coronary artery disease, diabetes, hypertension, obesity, valvular heart disease, smoking, and sedentary lifestyle. While there is no cure for HF, treatment options include lifestyle changes and medications, as well as implantable devices and surgery in select cases. Improved cardiac function and life expectancy has been demonstrated with the following pharmacotherapies: ACEIs, ARBs, ARNIs (sacubitril/valsartan [Entresto®]), beta blockers, hydralazine/isosorbide dinitrate, the SGLT2 inhibitor dapagliflozin (Farxiga®), and mineralocorticoid receptor agonists. Ivabradine (Corlanor®), a hyperpolarization-activated cyclic nucleotide-gated channel blocker, is also indicated in select patients with HFrEF. If approved, vericiguat will provide another therapeutic option for patients with HFrEF. Vericiguat may compete with oral sacubitril/valsartan (Entresto) and the oral antidiabetic agent dapagliflozin (Farxiga) in the HFrEF space. Both agents have demonstrated significant reductions in both CV death and HF hospitalization and at higher rates than reported for vericiguat (vericiguat, HR=0.9; Entresto, HR=0.8; Farxiga, HR=0.74). Notably, in the clinical trial, prespecified events with vericiguat were accumulated earlier than anticipated (10.8 months, versus 24 and 27 months in Farxiga and Entresto trials, respectively); this reduced exposure time may have contributed to its lower response rate compared to Farxiga and Entresto. All 3 agents are administered orally; vericiguat and Farxiga are taken once daily, while Entresto is dosed twice a day. The SLGT2 inhibitors canagliflozin (Invokana®) and empagliflozin (Jardiance®) are also in phase 3 trials for HF. Other drugs in phase 3 trials for HF target p38 mitogen-activated protein kinase (ARRY-797), aldose reductase (AT-001), mineralocorticoid receptor (finerenone), and myosin (mavacamten, omecamtive mercarbil).
FDA APPROVAL TIMELINE January 20, 2021 Priority Review
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$0
$29
$67
$121
$169
The forecast is a projection of total US sales per year.
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IMMUNOLOGY
voclosporin oral Aurinia PROPOSED INDICATION Lupus nephritis (LN)
CLINICAL OVERVIEW
Voclosporin is a next-generation calcineurin inhibitor (CNI) that blocks interleukin-2 (IL-2) expression and T-cell mediated immune responses and stabilizes podocytes in the kidneys. Safety and efficacy of voclosporin as add-on to background therapy of mycophenolate mofetil (MMF) plus taper of low-dose corticosteroids were demonstrated in the double-blind, phase 3 AURORA and phase 2b AURA-LV studies. In AURORA, 357 adults with LN were randomized to voclosporin or placebo. The primary endpoint was renal response, defined as urine protein/creatinine ratio (UPCR) ≤ 0.5 mg/mg, eGFR ≥ 60 mL/min or no confirmed decrease from baseline in eGFR of > 20%, sustained low-dose steroid use, and no use of rescue medication. The renal response rate at week 52 with voclosporin was superior compared to placebo (40.8% versus 22.5%, respectively; p<0.001); statistically significant differences between the groups were seen as early as 24 weeks. Approximately 10% of patients in each group achieved a decrease in eGFR by > 30%. Response with voclosporin was demonstrated across racial/ethnic groups (e.g., Black, Hispanic/Latino, Asian). In the AURA-LV trial, a higher rate of complete renal remission was seen with voclosporin compared to placebo at week 24 (32.6% versus 19.3%, respectively; p=0.046) and week 48 (49.4% versus 23.9%, respectively; p<0.001). Voclosporin was well tolerated. In both trials, infection was the most common adverse event reported. In AURORA, 1 death occurred in the voclosporin group and 5 in the placebo group. However, in the earlier AURA-LV study, the number of deaths was higher with voclosporin compared to placebo (10 [11.2%] versus 1 [1.1%], respectively); notably, the majority of deaths were reported at 2 study sites outside the US with compromised access to care and were not attributed to the study drug. For both clinical trials above, the data reflect oral doses of voclosporin of 23.7 mg twice daily. AURA-LV also included a dose of 39.5 mg twice daily; superiority to placebo was seen with both doses.
PLACE IN THERAPY
An estimated 1.5 million Americans have some form of lupus, with 90% being women. Moreover, it is 2 to 3 times more likely to affect women of color (e.g., Black, Hispanic/Latino, Asian, Native American) compared to Caucasians. Systemic lupus erythematosus (SLE) accounts for approximately 70% of all cases. Lupus nephritis occurs in up to 60% of patients with SLE, typically evident within 5 years of SLE diagnosis. Notably, LN progresses to ESRD in 5% to 20% of patients. Initial treatment of LN includes corticosteroids plus MMF or cyclophosphamide; corticosteroids plus azathioprine appears to be less beneficial. Complete renal response rates are 10% to 40% at 12 months with SOC therapy. Limited data suggest improved response when tacrolimus, a CNI, is added to an MMF plus glucocorticosteroid regimen. Once complete or partial response is achieved, MMF and azathioprine are the most common agents used for maintenance treatment. Voclosporin, a next-generation CNI, is an analog of cyclosporine. It may have a more predictable pharmacokinetic profile compared to legacy CNIs and may not need therapeutic monitoring. Significant improvement in renal response was observed when voclosporin was added to the SOC (MMF), regardless of race or ethnicity. If approved, voclosporin will be the first medication FDA-approved specifically to treat LN.
FDA APPROVAL TIMELINE January 22, 2021 Fast Track
Priority Review
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$0
$90
$273
$429
$654
The forecast is a projection of total US sales per year.
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Biosimilar Overview CLINICAL OVERVIEW
Biosimilars are very different from generic drugs in that they are not exact duplicates of their reference biologic product. The FDA approval process for biosimilars is designed to ensure that the biosimilar product is highly similar to the reference product without having any meaningful clinical differences. Many controversies surround biosimilars, and regulatory and litigation hurdles remain. The FDA has issued final and draft guidances. Select FDA biosimilar guidances are noted here. In January 2017, the agency issued final guidance on the nonproprietary naming of biologic products, which also applies to biosimilars. The biological products must bear a core name followed by a distinguishing 4-letter, lowercase, hyphenated suffix that is devoid of meaning. The FDA is still considering how to implement the nomenclature for previously approved biosimilar products. The international nonproprietary name (INN) impacts interchangeability as it affects pharmacists’ ability to substitute an interchangeable biosimilar for the reference product. The FDA withdrew the September 2017 draft industry guidance on determining similarity of a proposed biosimilar product to its reference product to allow for further consideration of the most current and relevant scientific methods in evaluating analytical data. The agency will focus on providing flexibility for efficient development of biosimilars while maintaining high scientific standards. In July 2018, the FDA finalized its guidance on labeling biosimilars. The guidance pertains to prescribing information (PI) but does not contain specific recommendations on interchangeability in the labeling. The labeling guidance provides recommendations on how to include, identify, and differentiate the biosimilar and the reference product in various sections of the PI. The basic premise remains that the originator product’s safety and effectiveness can be relied upon for HCPs to make prescribing decisions; therefore, a biosimilar should include relevant data from the originator in its PI. In May 2019, the agency released its final guidance on interchangeability. Several states had already enacted biosimilar substitution legislation. Biosimilars are expected to receive full extrapolation for the eligible indications of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will likely need to be considered individually. Insulins were historically regulated by the FDA as small molecules. However, effective March 23, 2020, drugs such as insulin and growth hormone were deemed biologics and transitioned from the drug pathway to the biologics pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.
PLACE IN THERAPY
The patents of several biologic drugs are set to expire in the next few years, opening the US market for biosimilar entry; however, patent litigation has resulted in significant launch delays of FDA-approved biosimilars. In June 2017, the US Supreme Court issued 2 landmark rulings: (1) allowing a biosimilar manufacturer to provide launch notice of commercial marketing to the originator manufacturer before or after FDA approval of the biosimilar product and (2) eliminating any federal requirement for disclosure, also known as the “patent dance.” Some states, however, mandate disclosure. These decisions may bring biosimilars to the market sooner and potentially create price competition in the marketplace. In July 2018, the FDA unveiled its Biosimilar Action Plan (BAP), a series of 11 steps to encourage biosimilar market competition, some of which were previously announced or underway. The BAP contains 4 key strategies: (1) improving the biosimilar development and approval process; (2) maximizing scientific and regulatory clarity for sponsors; (3) providing effective communications for patients, clinicians, and payers; and (4) reducing unfair tactics that may delay market approval and entry. The BAP strives to promote access to biosimilar products and reduce healthcare costs.
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To date, a total of 28 biosimilars have received FDA approval. Of these, only 18 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)
Manufacturer
Approval Date
Zarxio® (filgrastim-sndz)
Sandoz
March 2015
Inflectra® (infliximab-dyyb)
Pfizer/Celltrion
April 2016
Erelzi™ (etanercept-szzs)
Sandoz
August 2016
Amjevita™ (adalimumab-atto)
Amgen
September 2016
Renflexis® (infliximab-abda)
Samsung Bioepis/ Merck
May 2017
Cyltezo® (adalimumab-adbm)
Boehringer Ingelheim
August 2017
Mvasi™ (bevacizumab-awwb)
Amgen
September 2017
Ixifi™ (infliximab-qbtx)*
Pfizer
December 2017
Ogivri™ (trastuzumab-dkst)
Mylan
December 2017
Retacrit® (epoetin alfa-epbx)
Pfizer/Hospira
May 2018
Fulphila® (pegfilgrastim-jmdb)
Mylan
June 2018
Nivestym® (filgrastim-aafi)
Pfizer
July 2018
Hyrimoz™ (adalimumab-adaz)
Sandoz
October 2018
Udenyca® (pegfilgrastim-cbqv)
Coherus
November 2018
Truxima® (rituximab-abbs)
Celltrion/Teva
November 2018
Herzuma® (trastuzumab-pkrb)
Celltrion/Teva
December 2018
Ontruzant® (trastuzumab-dttb)
Samsung Bioepis/ Merck
January 2019
Trazimera™ (trastuzumab-qyyp)
Pfizer
March 2019
Eticovo™ (etanercept-ykro)
Samsung Bioepis/ Merck
April 2019
Kanjinti™ (trastuzumab-anns)
Amgen
June 2019
Zirabev™ (bevacizumab-bvzr)
Pfizer
June 2019
Hadlima™ (adalimumab-bwwd)
Samsung Bioepis/ Merck
July 2019
Ruxience™ (rituximab-pvvr)
Pfizer
July 2019
Abrilada™ (adalimumab-afzb)
Pfizer
November 2019
Ziextenzo® (pegfilgrastim-bmez)
Novartis/Sandoz
November 2019
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Commercially Available
-
-
-
-
-
-
-
Originator Product (Manufacturer) Neupogen® (Amgen) Remicade® (Janssen) Enbrel® (Amgen) Humira® (Abbvie) Remicade (Janssen) Humira (Abbvie) Avastin® (Genentech) Remicade (Janssen) Herceptin (Genentech) Epogen (Amgen) Procrit (Janssen) Neulasta® (Amgen) Neupogen (Amgen) Humira (Abbvie) Neulasta (Amgen) Rituxan (Genentech) Herceptin (Genentech) Herceptin (Genentech) Herceptin (Genentech) Enbrel (Amgen) Herceptin (Genentech) Avastin (Genentech) Humira (Abbvie) Rituxan (Genentech) Humira (Abbvie) Neulasta (Amgen)
APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)
Manufacturer
Approval Date
Avsola™ (infliximab-axxq)
Amgen
December 2019
Nyvepria™ (pegfiltrastim-apgf)
Pfizer/Hospira
June 2020
Hulio® (adalimumab-fkjp)
Mylan
July 2020
Commercially Available
-
Originator Product (Manufacturer) Remicade (Janssen) Neulasta (Amgen) Neulasta (Amgen)
* Pfizer already has Inflectra on the market and has not announced plans to launch Ixifi.
Also available are Eli Lilly’s Basaglar® insulin glargine, a biosimilar agent to Sanofi’s Lantus®, and Sanofi’s Admelog® insulin lispro, approved as a biosimilar product to Eli Lilly’s Humalog®. In June 2020, the FDA approved insulin glargine (Semglee™) by Mylan/Biocon under an abbreviated 505(b)(2) New Drug Application (NDA) pathway; the reference product was Lantus. Semglee is considered a biologic under section 351(a) rather than a biosimilar. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play a role in market adoption of biosimilars. While < 2% of Americans use biologics, they account for almost 40% of all prescription drug spending. Moreover, they comprised 70% of growth in drug spending from 2010 to 2015. Not surprisingly, there is a growing body of evidence on predicted biologic spend and potential biosimilar savings. A 2020 report by IQVIA forecasts that biosimilars are on track to reduce overall US drug spend by $100 billion over the next 5 years. In fact, the next 5 years are expected to have an estimated 5-fold increase in savings relative to the past 5 years as more biosimilars launch and existing biosimilars see more utilization and reductions in price. Three recent biosimilar launches in 2019 saw substantial uptake within the first year of commercialization, these are: bevacizumab (42%), trastuzumab (38%), and rituximab (20%). In the US, it is estimated that biosimilars will cost 15% to 35% less than the originator product, although price dynamics vary. The potential cost savings, however, can vary based on the market segment where brand contracts can play a role. A 2017 report by the RAND Corporation estimates a $54 billion cost savings from biosimilars between 2017 and 2026. In July 2018, an FDA analysis reported that if Americans had access to FDA-approved biosimilars in 2017, it would have resulted in a $4.5 billion savings. A 2017 analysis by the Moran Company projects biosimilars could save the government an estimated $11.4 billion by 2027, but it would require the Centers for Medicare and Medicaid Services (CMS) to revise its reimbursement policy for biosimilars. Subsequently, in November 2017, the CMS revised its reimbursement policy. The CMS now issues a unique Healthcare Common Procedure Coding System (HCPCS) code to each individual biosimilar. Under this rule, Medicare Part B separately codes and pays for biosimilars and no longer groups them into a common payment code with originator agents. A June 2018 infliximab case study by the Pacific Research Institute forecasts annual savings of up to $465 million from increased use of biosimilars to replace a single biologic for commercial payers and Medicare. Biosimilars are paving the way for increased access to important biologic therapies that may increase survival and/ or QOL for many patients with difficult-to-treat diseases while also reducing costs.
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BIOSIMILAR OVERVIEW continued
ONCOLOGY
bevacizumab IV Aybintio and Bmab-100 are investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth (VEGF)specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, non-squamous non-small cell lung cancer (nsNSCLC), glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.
FDA APPROVAL TIMELINE
Merck/Samsung Bioepis (Aybintio) Pending Mylan/Biocon (Bmab-100) December 25, 2020
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$2,076
$1,534
$1,278
$1,098
$901
The forecast is a projection of total US sales per year for the branded originator product.
BLOOD MODIFIER
filgrastim IV, SC Apotex and Kashiv are seeking biosimilars to Amgen’s Neupogen, a leukocyte growth factor indicated for use in patients: with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs; following induction or consolidation chemotherapy for acute myeloid leukemia (AML); with nonmyeloid malignancies who are undergoing myeloablative chemotherapy followed by bone marrow transplantation; to mobilize autologous hematopoietic progenitor cells for collection by leukapheresis; with symptomatic congenital neutropenia‚ cyclic neutropenia‚ or idiopathic neutropenia; and who are acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).
FDA APPROVAL TIMELINE Apotex (Grastofil) Pending Kashiv Pending
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$127
$104
$89
$80
$73
The forecast is a projection of total US sales per year for the branded originator product.
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BIOSIMILAR OVERVIEW continued
ENDOCRINE
insulin aspart SC Mylan/Biocon Mylan/Biocon is seeking biosimilar approval to Novo Nordisk’s Novolog®, a rapid-acting insulin to improve glycemic control in patients with T1DM or T2DM.
FDA APPROVAL TIMELINE April to June 2021
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$948
$740
$643
$578
$533
The forecast is a projection of total US sales per year for the branded originator product.
BLOOD MODIFIER
pegfilgrastim SC Lapelga and MSB-11455 are investigational biosimilars to Amgen’s Neulasta, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs and in patients acutely exposed to myelosuppressive doses of radiation (HSARS).
FDA APPROVAL TIMELINE Apotex (Lapelga) Pending
Merck/Fresenius (MSB-11455) January to March 2021
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$1,980
$1,576
$1,295
$1,093
$948
The forecast is a projection of total US sales per year for the branded originator product.
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BIOSIMILAR OVERVIEW continued
ONCOLOGY
rituximab (ABP-798) IV Amgen/Allergan ABP-798 is an investigational biosimilar to Genentech’s Rituxan, a CD20-directed cytolytic antibody indicated for the treatment of non-Hodgkin’s lymphoma (NHL), chronic lymphocytic leukemia (CLL), rheumatoid arthritis (RA), and antineutrophil cytoplasmic antibody-associated vasculitis.
FDA APPROVAL TIMELINE December 18, 2020
FINANCIAL FORECAST (reported in millions) 2020
2021
2022
2023
2024
$3,514
$2,480
$1,819
$1,444
$1,127
The forecast is a projection of total US sales per year for the branded originator product.
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KEEP ON YOUR RADAR Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the MRx Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2024, are displayed. The financials are projected total annual US sales, reported in millions. tirzepatide
bardoxolone methyl
$652
$507
Diabetes
Renal
NVX-CoV2373 vaccine
belumosudil
$1,615
$610
Immunology
COVID-19
betibeglogene autotemcel (Zynteglo)
mRNA-1273 vaccine COVID-19
Hematology/Gene therapy
$2,349
$366
mirikizumab
bimekizumab
$552
$465
Immunology
Immunology
mavacamten
deucravacitinib
$886
$835
Cardiovascular
Immunology
lonapegsomatropin
efgartigimod
$623
$754
Endocrine
Immunology
lenadogene nolparvovec (GS010) Ophthalmology/ Gene therapy
$41
elivaldogene tavalentivec (Lenti-D) ipatasertib Oncology
Neurology/Gene therapy
$49
$530 pecialty drug names appear in ď&#x201A;Ť S magenta throughout the publication.
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PIPELINE DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2021. It is not intended to be a comprehensive inventory of all drugs in the pipeline; emphasis is placed on drugs in high-impact categories. Investigational drugs with a Complete Response Letter (CRL) and those that have been withdrawn from development are also noted. APPLICATION APPLICATION SUBMITTED SUBMITTED TO THE FDA
IN PHASE PHASE 33 TRIALS TRIALS
59% 41% 34% 26% 25% 7%
Specialty
65% 35% 32% 13% 8%
Traditional
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Orphan Drug
Priority Review
Breakthrough Therapy
Biosimilar
PIPELINE DRUG LIST Specialty drug names appear in magenta throughout the publication. NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
Submitted (New Drugs) loteprednol etabonate 0.25%
Kala
Dry eye disease
Ophthalmic
Submitted – 505(b)(2) NDA
10/30/2020
mannitol (dry powder)
Cheisi
CF (adults)
Inhaled
Submitted – NDA; Fast Track; Orphan Drug
10/30/2020
viloxazine
Supernus
ADHD
Oral
Submitted – NDA
11/06/2020
amphetamine sulfate IR (tamper-resistant)
Arbor
ADHD (adults, pediatrics ages ≥ 3 years)
Oral
Submitted – NDA
11/13/2020
samidorphan/olanzapine
Alkermes
Bipolar disorder; Schizophrenia
Oral
Submitted – NDA
11/13/2020
sutimlimab
Sanofi
Cold agglutnin disease
IV
Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review
11/13/2020
naloxone single-dose prefilled syringe
US Worldmeds
Opioid overdose
IM
Submitted – 505(b)(2) NDA
11/15/2020
lisocabtagene maraleucel
Bristol-Myers Squibb
DLBCL (relapsed/ refractory)
IV
Submitted – BLA; 11/16/2020 Breakthrough Therapy; Orphan Drug; Priority Review; RMAT
lonafarnib
Eiger
Hutchinson–Gilford Progeria syndrome
Oral
Submitted – NDA; 11/20/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease
treprostinil dry powder
Liquidia
PAH
Inhaled
Submitted – 505(b)(2) NDA
setmelanotide
Rhythm
Obesity (pro-opiomelanocortin and leptin receptor deficiency)
SC
Submitted – NDA; 11/27/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease
naxitamab
Y-mAbs Therapeutics
Neuroblastoma (relapsed/ IV refractory, high-risk)
Submitted – BLA; 11/30/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease
inclisiran
Novartis/The Medicines Company
Dyslipidemia (for secondary prevention patients with ASCVD and familial hypercholesterolemia)
SC
Submitted – NDA; Orphan Drug
tanezumab
Pfizer
Osteoarthritis pain
IV
Submitted – BLA; Fast December Track 2020
berotralstat
Biocryst
Hereditary angioedema (attack prevention)
Oral
Submitted – NDA; Fast Track; Orphan Drug
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11/24/2020
December 2020
12/03/2020
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
lumasiran
Alnylam
Hyperoxaluria
SC
Submitted – NDA; 12/07/2020 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease
margetuximab
Macrogenics
Breast cancer (HER2+, in combination with chemotherapy)
IV
Submitted – BLA; Fast 12/18/2020 Track
rituximab (biosimilar to Genentech’s Rituxan)
Amgen/Allergan
RA; CLL/SLL; NHL (indolent); ANCAassociated vasculitis
IV
Submitted – BLA
12/18/2020
relugolix
Myovant
Prostate cancer (advanced)
Oral
Submitted – NDA; Priority Review
12/20/2020
roxadustat
AstraZeneca
Anemia due to CKD (dialysis-independent; dialysis-dependent)
Oral
Submitted – NDA
12/20/2020
ansofaxine
Luye
MDD
Oral
Submitted – NDA
12/25/2020
bevacizumab (biosimilar to Genentech’s Avastin)
Mylan/Biocon
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Submitted – BLA
12/25/2020
vibegron
Urovant
Overactive bladder
Oral
Submitted – NDA
12/25/2020
arbaclofen ER
Osmotica
MS-associated spasticity
Oral
Submitted – 505(b)(2) NDA
12/29/2020
furosemide wearable pump Scpharmaceuticals
Congestive heart failure
SC
Submitted – 505(b)(2) NDA
12/30/2020
tirbanibulin
Almirall
Actinic keratoses
Topical
Submitted – NDA
12/30/2020
belumosudil
Kadmon
Chronic GVHD
Oral
Submitted – NDA; Breakthrough Therapy; Orphan Drug; RTOR
Jan–Mar 2021
pegfilgrastim (biosimilar to Amgen’s Neulasta)
Merck/Fresenius
Neutropenia/leukopenia
SC
Submitted – BLA
Jan–Mar 2021
vericiguat
Merck/Bayer
Chronic heart failure
Oral
Submitted – NDA; Priority Review
01/20/2021
voclosporin
Aurinia
Lupus nephritis
Oral
Submitted – NDA; Fast Track; Priority Review
01/22/2021
pegunigalsidase alfa
Chiesi
Fabry’s disease
IV
Submitted – BLA; seeking Accelerated Approval; Fast Track; Priority Review
01/27/2021
tepotinib
Merck
NSCLC (mesenchymalepithelial transition exon 14 [METex14] skipping)
Oral
Submitted – NDA; Breakthrough Therapy; Priority Review; RTOR
February 2021
evinacumab
Regeneron
Homozygous familial hypercholesterolemia
IV
Submitted – BLA; Breakthrough Therapy; Priority Review
02/11/2021
24 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
umbralisib
TG
Marginal zone lymphoma (≥ 1 prior anti-CD20 based regimen)
Oral
Submitted – NDA; seeking Accelerated Approval; Breakthrough Therapy; Orphan Drug; Priority Review
02/15/2021
trilaciclib
G1 Therapeutics/ Boehringer Ingelheim
SCLC
IV
Submitted – NDA; Breakthrough Therapy; Priority Review
02/15/2021
casimersen
Sarepta
DMD (amenable to exon 45 skipping)
IV
Submitted – NDA; seeking Accelerated Approval; Orphan Drug; Priority Review
02/25/2021
melflufen
Oncopeptides
Multiple myeloma (triple refractory)
IV
Submitted – NDA; Orphan Drug; Priority Review
02/26/2021
paclitaxel/encequidar
Athenex
Breast cancer
Oral
Submitted – NDA; Priority Review
02/28/2021
taurolidine/heparin/citrate
Cormedix
Prevention of catheterrelated sepsis (hemodialysis patients)
IV
Submitted – NDA; Fast Track; Priority Review; QIDP
02/28/2021
ropeginterferon alfa-2b
Pharmaessentia
Polycythemia vera (in absence of symptomatic splenomegaly)
SC
Submitted – BLA; Orphan Drug
Mar–Apr 2021
serdexmethylphenidate
Kempharm
ADHD
Oral
Submitted – 505(b)(2) NDA
03/02/2021
plasminogen (human)
Liminal
Congenital plasminogen deficiency
IV
Submitted – BLA; Fast Track; Orphan Drug; Rare Pediatric Disease
03/05/2021
aducanumab
Biogen/Eisai
Alzheimer’s disease
IV
Submitted – BLA; Fast 03/07/2021 Track; Priority Review
arimoclomol
Orphazyme
Niemann-Pick disease
Oral
Submitted – NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review; Rare Pediatric Disease
03/17/2021
ponesimod
Janssen
MS (relapsing)
Oral
Submitted – NDA
03/18/2021
dasiglucagon
Zealand
Hypoglycemia (diabetesrelated)
SC
Submitted – NDA; Orphan Drug
03/27/2021
idecabtagene vicleucel
Bristol-Myers Squibb/ Bluebird Bio
Multiple myeloma (≥ 3 prior therapies)
IV
Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review
03/27/2021
tivozanib
AVEO
RCC (relapsed/refractory)
Oral
Submitted – NDA
03/31/2021
inolimomab
Elsalys
GVHD (acute, steroidrefractory)
IM
Submitted – BLA; Orphan Drug; RTOR
April 2021
eflornithine/sulindac
Mallinckrodt
Familial adenomatous polyposis
Oral
Submitted – 505(b)(2) NDA; seeking Accelerated Approval; Fast Track; Orphan Drug
Apr–Jun 2021
25 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
insulin aspart (biosimilar to Novo Nordisk’s Novolog)
Mylan/Biocon
T1DM; T2DM
SC
Submitted – BLA
Apr–Jun 2021
tralokinumab
AstraZeneca
Atopic dermatitis
SC
Submitted – BLA
Apr–Jun 2021
fosdenopterin
Bridgebio
Molybdenum cofactor deficiency (MoCD)
IV
Submitted – NDA; 04/09/2021 Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatric Disease
estetrol/drospirenone
Mayne
Contraception
Oral
Submitted – NDA
04/16/2021
treprostinil dry powder
United Therapeutics
PAH
Inhaled
Submitted – 505(b)(2) NDA
04/17/2021
benzoyl peroxide
Sol-gel
Rosacea
Transdermal
Submitted – 505(b)(2) NDA
04/26/2021
abrocitinib
Pfizer
Atopic dermatitis
Oral
Submitted – NDA; Breakthrough Therapy
04/30/2021
dehydrated alcohol
Eton
Methanol poisoning
SC
Submitted – NDA; Orphan Drug
05/27/2021
leuprolide mesylate readyto-use, 6-month depot
Foresee
Prostate cancer
SC
Submitted – 505(b)(2) NDA
05/27/2021
zonisamide oral suspension
Eton
Partial seizures
Oral
Submitted – 505(b)(2) NDA
05/28/2021
udenafil
Allergan
Single ventricle heart disease (ages ≥ 12 years)
Oral
Submitted – NDA; Orphan Drug
June 2020
relugolix/estradiol/ norethindrone
Myovant
Uterine fibroid-related Oral heavy menstrual bleeding
Submitted – NDA
06/01/2021
umbralisib
TG
Follicular lymphoma (≥ 2 prior systemic therapies)
Oral
Submitted – NDA; seeking Accelerated Approval; Orphan Drug
06/15/2021
lonapegsomatropin
Ascendis
Growth hormone deficiency (pediatrics)
SC
Submitted – BLA; Orphan Drug
06/25/2021
proteolytic enzymes
Mediwound
Burn injury debridement
Topical
Submitted – BLA; Orphan Drug
06/29/2021
bimekizumab
UCB
PSO
SC
Submitted – BLA
July 2021
avacopan
Chemocentryx
ANCA-associated vasculitis
Oral
Submitted – NDA; Orphan Drug
07/07/2021
apomorphine infusion pump
Supernus
Parkinson’s disease (offepisodes)
SC
Submitted – NDA
07/14/2021
topiramate oral solution
Eton
Partial seizures
Oral
Submitted – 505(b)(2) NDA
08/06/2021
vosoritide
Biomarin
Achondroplasia
SC
Submitted – NDA; Orphan Drug
08/20/2021
pegcetacoplan
Apellis
Paroxysmal nocturnal hemoglobinuria
SC
Submitted – NDA; Fast Track; Orphan Drug
09/15/2021
loncastuximab tesirine
ADC
DLBCL (relapsed/ refractory)
IV
Submitted – BLA; Orphan Drug
09/21/2021
pneumococcal multivalent vaccine
Pfizer
Invasive pneumococcal disease prevention
IM
Submitted – BLA; Breakthrough Therapy; Fast Track
October 2021
26 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
bevacizumab (biosimilar to Genentech’s Avastin)
Merck/Samsung Bioepis
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Submitted – BLA
Pending
filgrastim (biosimilar to Amgen’s Neupogen)
Apotex
Neutropenia/leukopenia
IV, SC
Submitted – BLA
Pending
filgrastim (biosimilar to Amgen’s Neupogen)
Kashiv
Neutropenia/leukopenia
IV, SC
Submitted – BLA
Pending
pegfilgrastim (biosimilar to Amgen’s Neulasta)
Apotex
Neutropenia/leukopenia
SC
Submitted – BLA
Pending
linaclotide acetate (Linzess®)
Ironwood
IBS (treatment of abdominal symptoms)
Oral
Submitted – sNDA
October 2020
ticagrelor (Brilinta®)
AstraZeneca
Ischemic stroke
Oral
Submitted – sNDA; Priority Review
Oct–Nov 2020
durvalumab (Imfinzi®) 4-week fixed dose regimen
AstraZeneca
Bladder cancer; NSCLC
IV
Submitted – sBLA
Oct–Dec 2020
alectinib (Alecensa®)
Genentech
NSCLC (1st-line, ALK+)
Oral
Submitted – sNDA
November 2020
estradiol/progesterone (Bijuva®) 0.5 mg/100 mg
TherapeuticsMD
Menopause (including hormone replacement therapy [HRT])
Oral
Submitted – sNDA
11/16/2020
baloxavir marboxil (Xofluza®) oral granules, tablets
Genentech
Influenza (treatment, ages 1-12 years; postexposure prophylaxis, ages 1 year to adults)
Oral
Submitted – sNDA
11/23/2020
pembrolizumab (Keytruda®)
Merck
Breast cancer (locally recurrent unresectable or metastatic, TNBC, PD-L1positive; in combination with chemotherapy)
IV
Submitted – sBLA; seeking Accelerated Approval; Breakthrough Therapy; Priority Review
11/27/2020
tasimelteon (Hetlioz®) capsule and oral liquid
Vanda
Smith-Magenis syndrome (adults and children)
Oral
Submitted – sNDA; Orphan Drug; Priority Review
12/01/2020
ocrelizumab (Ocrevus®) 2-hour twice-yearly infusion
Genentech
MS (relapsing and primary IV progressive)
Submitted – sBLA
12/14/2020
elexacaftor/tezacaftor/ ivacaftor and ivacaftor (Trikafta®)
Vertex
CF (with additional rare CFTR mutations)
Oral
Submitted – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug
12/30/2020
ivacaftor (Kalydeco®)
Vertex
CF (with additional rare CFTR mutations)
Oral
Submitted – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug
12/30/2020
tezacaftor/ivacaftor and ivacaftor (Symdeko®)
Vertex
CF (with additional rare CFTR mutations)
Oral
Submitted – sNDA; Breakthrough Therapy; Fast Track; Orphan Drug
12/30/2020
crizotinib (Xalkori®)
Pfizer
Anaplastic large cell lymphoma (relapsed/ refractory, pediatric)
Oral
Submitted – sNDA; Breakthrough Therapy
January 2021
Submitted (Supplementals)
27 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
lacosamide (Vimpat®)
UCB
Primary generalized tonic-clonic seizures (adjunctive treatment, ages ≥ 4 years)
IV, Oral
Submitted – sNDA
January 2021
omalizumab (Xolair®) self-administered
Genentech
Asthma; Urticaria
SC
Submitted – sBLA
Jan–Mar 2021
peginterferon beta-1a (Plegridy®)
Biogen
MS (relasping/remitting, active secondary progressive)
IM
Submitted – sBLA; Fast Track
February 2021
baricitinib (Olumiant®)
Eli Lilly
Atopic dermatitis
Oral
Submitted – sNDA
Feb–Apr 2021
cabozantinib (Cabometyx®)
Exelixis
RCC (advanced, 1st-line, in combination with nivoluab)
Oral
Submitted – sNDA; Priority Review
02/20/2021
nivolumab (Opdivo®)
Bristol-Myers Squibb
RCC (advanced, in combination with cabozantinib)
IV
Submitted – sBLA; Breakthrough Therapy; Fast Track; Priority Review
02/20/2021
sacubitril/valsartan (Entresto)
Novartis
Heart failure with preserved ejection fraction (HFpEF)
Oral
Submitted – sNDA
02/26/2021
pralsetinib (Gavreto™)
Blueprint Medicines
Thyroid cancer (RETfusion+)
Oral
Submitted – sNDA; Breakthrough Therapy; Priority Review; RTOR
02/28/2021
belimumab (Benlysta®)
GlaxoSmithKline
Lupus nephritis
IV
Submitted – sBLA; Breakthrough Therapy
Mar–May 2021
rimegepant (Nurtec™ ODT)
Biohaven
Migraine prevention
Oral
Submitted – sNDA
May–Jun 2021
selinexor (Xpovio )
Karyopharm
Multiple myeloma (after ≥ Oral 1 prior line of therapy)
Submitted – sNDA; Orphan Drug
03/19/2021
bupivacaine liposome suspension (Exparel®)
Pacira
Postsurgical pain (ages ≥ 6 years)
IM
Submitted – sNDA
03/22/2021
pembrolizumab (Keytruda)
Merck
Breast cancer (highrisk, early-stage, TNBC, neoadjuvant, in combination with chemotherapy); Breast cancer (high-risk, earlystage, TNBC, adjuvant, monotherapy)
IV
Submitted – sBLA; seeking Accelerated Approval; Breakthrough Therapy; Priority Review
03/29/2021
upadacitinib (Rinvoq™)
Abbvie
PsA
Oral
Submitted – sNDA
04/01/2021
pimavanserin (Nuplazid )
Acadia
Dementia-related hallucinations and delusions
Oral
Submitted – sNDA; Breakthrough Therapy
04/03/2021
alirocumab (Praluent®)
Regeneron
Homozygous familial hypercholesterolemia
SC
Submitted – sBLA; Orphan Drug
04/04/2021
treprostinil (Tyvaso®)
United Therapeutics
Interstitial lung diseaseassociated pulmonary hypertension
Inhaled
Submitted – sNDA; Priority Review
04/14/2021
ibrutinib (Imbruvica®)
Abbvie
Waldenstrom macroglobulinemia (in combination with rituximab)
Oral
Submitted – sNDA; Breakthrough Therapy; Orphan Drug
04/23/2021
®
®
28 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
tenapanor (Ibsrela®)
Ardelyx
Hyperphosphatemia (in CKD dialysis-dependent patients)
Oral
Submitted – sNDA
04/29/2021
omadacycline (Nuzyra®)
Paratek
CABP (oral-only dosing)
IV, Oral
Submitted – sNDA; Fast Track; QIDP
05/31/2021
upadacitinib (Rinvoq)
Abbvie
Axial spondyloarthritis
Oral
Submitted – sNDA
06/25/2021
axicabtagene ciloleucel (Yescarta®)
Gilead
Follicular lymphoma, Marginal zone lymphoma (relapsed/refractory, after ≥ 2 lines of therapy for both)
IV
Submitted – sBLA; Breakthrough Therapy; Orphan Drug
July 2021
daratumumab/ hyaluronidase-fihj (Darzalex Faspro™)
Janssen
Amyloidosis (light-chain)
SC
Submitted – sBLA; RTOR
July 2021
levonorgestrel (Mirena®)
Bayer
Contraception (up to 7 years duration)
Intrauterine
Submitted – sNDA
July 2021
selexipag (Uptravi®)
Janssen
PAH (WHO FC II-III, temporarily unable to take oral formulation)
IV
Submitted – sNDA; Orphan Drug
07/30/2021
upadacitinib (Rinvoq)
Abbvie
Atopic dermatitis (adults, adolescents)
Oral
Submitted – sNDA; Breakthrough Therapy
08/21/2021
abaloparatide-TD
Radius Health
Osteoporosis/osteopenia
Transdermal
Phase 3 – NDA
TBD
Ad26COVS1 vaccine
Janssen
COVID-19
IM
Phase 3 – BLA
TBD
Ad5-nCoV vaccine
Cansino/Beijing Institute of Biotechnology
COVID-19
IM
Phase 3 – BLA
TBD
adalimumab (biosimilar to Abbvie’s Humira)
Coherus
RA; AS; PSO; PsA; JIA; CD; UC
SC
Phase 3 – BLA
TBD
adalimumab (biosimilar to Abbvie’s Humira)
Fresenius
RA; AS; PSO; PsA; JIA; CD; UC
SC
Phase 3 – BLA
TBD
adalimumab (biosimilar to Abbvie’s Humira)
Momenta
RA; AS; PSO; PsA; JIA; CD; UC
SC
Phase 3 – BLA
TBD
adalimumab (biosimilar to Abbvie’s Humira)
Mylan/Biocon
RA; AS; PSO; PsA; JIA; CD; UC
SC
Phase 3 – BLA
TBD
aflibercept (biosimilar to Regeneron’s Eylea®)
Mylan/Biocon
Diabetic macular edema
Intraocular
Phase 3 – BLA
TBD
AG10
Eidos
Transthyretin amyloid cardiomyopathy (ATTRCM)
Oral
Phase 3 – NDA
TBD
alicaforsen
Atlantic Healthcare
Pouchitis
Rectal
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
alpha 1 proteinase inhibitor
Kamada
Alpha-1 antitrypsin deficiency
Inhaled
Phase 3 – BLA; Orphan Drug
TBD
amivantamab
Janssen
NSCLC
IV
Phase 3 – BLA; Breakthrough Therapy
TBD
andolast
Mylan
Asthma
Inhaled
Phase 3 – NDA
TBD
anifrolumab
AstraZeneca
SLE
IV
Phase 3 – BLA; Fast Track
TBD
anthrax vaccine adsorbed
Emergent
Anthrax infection
IM
Phase 3 – BLA; Fast Track
TBD
Phase 3 (New Drugs)
29 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
antolimab
Allakos
Gastroenteritis (eosinophilic esophagitis)
IV
Phase 3 – BLA; Orphan Drug
TBD
ARO-AAT
Arrowhead
Liver disease (alpha-1 antitrypsin deficiency)
SC
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
asciminib
Novartis
Chronic myelogenous leukemia (CML)
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
ataluren
PTC
DMD
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
AT-GAA
Amicus
Pompe disease
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
atogepant
Allergan
Migraine prevention
Oral
Phase 3 – NDA
TBD
autologous genetically modified human dermal fibroblasts
Castle Creek
Epidermolysis bullosa
Intradermal
Phase 3 – BLA; Fast Track; Orphan Drug; RMAT
TBD
avalglucosidase alfa
Sanofi
Pompe disease
IV
Phase 3 – BLA; Breakthrough Therapy; Fast Track
TBD
AZD-1222 vaccine (formerly ChAdOx1-S)
AstraZeneca/University of Oxford
COVID-19
IM
Phase 3 – BLA
TBD
baclofen/naltrexone/ sorbitol
Pharnext
Charcot-Marie-Tooth disease
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
balixafortide
Polyphor
Breast cancer
IV
Phase 3 – NDA; Fast Track
TBD
balstilimab
Agenus
Cervical cancer
IV
Phase 3 – BLA; Fast Track
TBD
bamlanivimab
Eli Lilly
COVID-19
IV
Phase 3 – BLA
TBD
bardoxolone methyl
Reata
Alport syndrome
Oral
Phase 3 – NDA; Orphan Drug
TBD
beremagene geperpavec
Krystal
Epidermolysis bullosa
Topical
Phase 3 – BLA; Fast Track; Orphan Drug; RMAT
TBD
betibeglogene autotemcel (Zynteglo)
Bluebird Bio
Thalassemia; Sickle cell disease
IV
Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug. RMAT
TBD
bevacizumab
Outlook
Wet AMD
Intraocular
Phase 3 – BLA
TBD
bevacizumab (biosimilar to Genentech’s Avastin)
Bio-Thera Solutions
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Phase 3 – BLA
TBD
bevacizumab (biosimilar to Genentech’s Avastin)
Boehringer Ingelheim
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Phase 3 – BLA
TBD
bevacizumab (biosimilar to Genentech’s Avastin)
Kyowa Kirin/AstraZeneca
Brain cancer; Cervical cancer; CRC; NSCLC; Ovarian cancer; RCC
IV
Phase 3 – BLA
TBD
bexagliflozin
Theracos
T2DM
Oral
Phase 3 – NDA
TBD
bimekizumab
UCB
Axial spondyloarthritis; PsA
SC
Phase 3 – BLA
TBD
bintrafusp alfa
Merck
Biliary tract cancer
IV
Phase 3 – BLA; Orphan Drug
TBD
30 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
BIVV 001
Sanofi
Hemophilia A
IV
Phase 3 – BLA; Orphan Drug
TBD
BNT162 vaccine (multiple variants)
Biontech/Pfizer
COVID-19
IM
Phase 3 – BLA; Fast Track
TBD
budenoside
Calliditas
Immunoglobulin A (IgA) nephropathy (Berger’s disease)
Oral
Phase 3 – 505(b)(2) NDA; Orphan Drug
TBD
cabotegravir long-acting
Viiv
HIV-1 infection preexposure prevention (PrEP)
IM
Phase 3 – NDA
TBD
calmangafodipir
Pledpharma
Chemotherapy-induced peripheral neuropathy
IV
Phase 3 – NDA
TBD
cannabidiol gel
Zynerba
Fragile X syndrome
Transdermal
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
capivasertib
AstraZeneca
Breast cancer
Oral
Phase 3 – NDA
TBD
capsaicin
Centrexion
Osteoarthritis
Intraarticular
Phase 3 – NDA; Fast Track
TBD
carglumic acid
Recordati
Hyperammonaemia (autosomal disorderrelated)
Oral
Phase 3 – NDA; Orphan Drug
TBD
CD24Fc
OncoImmune
COVID-19
IV
Phase 3 – BLA
TBD
ceftobiprole medocaril
Basilea
ABSSSI
IV
Phase 3 – NDA; Fast Track; QIDP
TBD
cenicriviroc mesylate
Allergan
NASH
Oral
Phase 3 – NDA; Fast Track
TBD
ciltacabtagene autoleucel
Janssen
Multiple myeloma
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
clindamycin phosphate gel
Daré
Bacterial vaginosis
Intravaginal
Phase 3 – NDA; QIDP
TBD
CM-AT (pancreatic enzyme)
Curemark
Autism spectrum disorders
Oral
Phase 3 – BLA; Fast Track
TBD
crisantaspase
Jazz
ALL
IM, IV
Phase 3 – BLA; Fast Track
TBD
CSL112
CSL
Atherosclerosis
IV
Phase 3 – BLA
TBD
dactolisib
Restorbio
COVID-19
Oral
Phase 3 – NDA
TBD
dalcetrapib
Dalcor
Dyslipidemia/ hypercholesterolemia
Oral
Phase 3 – NDA
TBD
daprodustat
GlaxoSmithKline
Anemia due to CKD (dialysis-dependent, dialysis-independent)
Oral
Phase 3 – NDA
TBD
daridorexant
Idorsia
Insomnia
Oral
Phase 3 – NDA
TBD
decuprate
Alexion
Wilson’s disease
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
dehydrated human Mimedx amnion-chorion membrane
Achilles tendonitis; Plantar fasciitis
IV
Phase 3 – BLA
TBD
denosumab (biosimilar to Amgen’s Prolia®)
Novartis
Osteoporosis/osteopenia
SC
Phase 3 – BLA
TBD
deramanido
Otsuka
Tuberculosis
Oral
Phase 3 – NDA
TBD
deucravacitinib
Bristol-Myers Squibb
PSO
Oral
Phase 3 – NDA
TBD
dexamethasone SR
Otonomy
Meniere’s disease
Intratympanic
Phase 3 – 505(b)(2) NDA; Fast Track
TBD
31 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
dexmedetomidine
Bioxcel
Schizophrenia
SL
Phase 3 – NDA; Fast Track
TBD
dextromethorphan/ bupropion
Axsome
MDD
Oral
Phase 3 – 505(b)(2) NDA; Breakthrough Therapy; Fast Track
TBD
difelikefalin
Enteris
Pruritus (hemodialysisrelated)
IV
Phase 3 – NDA; Breakthrough Therapy
TBD
dociparstat
Chimerix
COVID-19
IV
Phase 3 – NDA
TBD
docosahexaenoic acid
Micelle
Sickle cell disease
Oral
Phase 3 – NDA; Orphan Drug
TBD
dovitinib
Oncology Venture
RCC
Oral
Phase 3 – NDA
TBD
dusquetide
Soligenix
Mucositis
IV
Phase 3 – NDA; Fast Track
TBD
dust mite immunotherapy
Stallergenes
Allergic rhinitis
SL
Phase 3 – BLA
TBD
EB-101 (gene therapy)
Abeona
Epidermolysis bullosa
Surgical application
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug; RMAT
TBD
edasalonexent
Catabasis
DMD
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
efgartigimod
Argenx
Myasthenia gravis
IV
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
elamipretide
Stealth Bio
Barth syndrome
SC
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
elivaldogene tavalentivec (Lenti-D)
Bluebird Bio
Adrenomyeloneuropathy (adrenoleukodystrophy)
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
enmetazobactam
Allecra
UTI (complicated)
IV
Phase 3 – NDA; Fast Track; QIDP
TBD
EP-2101 therapeutic vaccine
OSE Immunotherapeutics
NSCLC
SC
Phase 3 – NDA; Orphan Drug
TBD
eprenetapopt
Aprea
Myelodysplastic syndrome (tumor protein p53 mutation)
IV
Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
erdosteine
Alitair
COPD
Oral
Phase 3 – NDA
TBD
estetrol
Mithra
Menopause vasomotor symptoms
Oral
Phase 3 – NDA
TBD
etanercept (biosimilar to Amgen’s Enbrel)
Coherus
RA; PSO
SC
Phase 3 – BLA
TBD
etranacogene dezaparvovec
Uniqure
Hemophilia B
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
etrasimod
Arena
UC
Oral
Phase 3 – NDA
TBD
etrolizumab
Genentech
CD
SC
Phase 3 – BLA
TBD
faricimab
Genentech
Diabetic macular edema; Wet AMD
Intraocular
Phase 3 – BLA
TBD
fasinumab
Regeneron
Osteoarthritis
SC
Phase 3 – BLA
TBD
32 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
fexapotide triflutate
Nymox
Benign prostatic hyperplasia
Intratumoral
Phase 3 – NDA
TBD
fezolinetant
Astellas
Menopause vasomotor symptoms
Oral
Phase 3 – NDA
TBD
fidanacogene elaparvovec
Pfizer
Hemophilia B
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
filgotinib
Gilead
PsA; UC
Oral
Phase 3 – NDA
TBD
filsuvez
Amryt
Epidermolysis bullosa
Topical
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
finerenone
Bayer
Diabetic nephropathy
Oral
Phase 3 – NDA
TBD
fitusiran
Sanofi
Hemophilia A and B (with and without inhibitors)
SC
Phase 3 – NDA; Orphan Drug
TBD
follitropin alfa (biosimilar to EMD Serono’s Gonal-F®)
Allergan
Female reproductive disorder
SC
Phase 3 – BLA
TBD
follitropin alfa (biosimilar to EMD Serono’s Gonal-F)
Finox
Female reproductive disorder
SC
Phase 3 – BLA
TBD
follitropin delta
Ferring
Female reproductive disorder
IV
Phase 3 – BLA
TBD
ganaxolone
Marinus
Seizure disorders
IV, Oral
Phase 3 – NDA; Orphan Drug
TBD
gefapixant
Merck
Chronic cough
Oral
Phase 3 – NDA
TBD
gepotidacin
GlaxoSmithKline
UTI (uncomplicated)
Oral
Phase 3 – NDA; QIDP
TBD
givinostat
Italfarmaco
DMD
Oral
Phase 3 – NDA
TBD
glatiramer acetate depot
Mylan
MS
IM
Phase 3 – 505(b)(2) NDA
TBD
glepaglutide
Zealand
Short bowel syndrome
SC
Phase 3 – NDA; Orphan Drug
TBD
glycopyrrolate MDI
AstraZeneca
COPD
Inhaled
Phase 3 – NDA
TBD
hydrocortisone granules
Diurnal
Congenital adrenal hyperplasia
Oral
Phase 3 – 505(b)(2) NDA; Orphan Drug
TBD
hypericin
Soligenix
Cutaneous T-cell lymphoma (CTCL)
Topical
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
ibrexafungerp
Scynexis
Vulvovaginal candidiasis
Oral
Phase 3 – NDA; Fast Track; Orphan Drug; QIDP
TBD
idursulfase
Takeda
Mucopolysaccharidosis II Intrathecal (MPS II; Hunter syndrome)
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
infigratinib
Bridgebio
Biliary tract cancer
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
infliximab (biosimilar to Janssen’s Remicade)
Nichi-Iko
RA; AS; PSO; PsA; CD; UC
IV
Phase 3 – BLA
TBD
insulin aspart (biosimilar to Novo Nordisk’s Novolog)
Sanofi
T1DM; T2DM
SC
Phase 3 – BLA
TBD
insulin glargine (biosimilar to Sanofi’s Lantus)
Gan & Lee
T1DM; T2DM
SC
Phase 3 – BLA
TBD
iodine I-131 monoclonal antibody
Actinium
Myeloablation prior to allogeneic HSCT to treat AML
IV
Phase 3 – BLA; Orphan Drug
TBD
ipatasertib
Genentech
Prostate cancer
Oral
Phase 3 – NDA
TBD
ipatasertib
Genentech
Breast cancer
Oral
Phase 3 – NDA
TBD
33 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
JNJ-78436735
Janssen
COVID-19
N/A
Phase 3 – BLA
TBD
KSI-301
Kodiak
Diabetic macular edema; Macualar edema related to retinal vein occlusion
Intraocular
Phase 3 – BLA
TBD
Lactobacillus reuteri
Infant Bacterial Therapeutics
Necrotizing enterocolitis
Oral
Phase 3 – BLA; Orphan Drug
TBD
L-citrulline
Asklepion
Acute lung injury
IV
Phase 3 – NDA; Orphan Drug
TBD
lebrikizumab
Dermira
Atopic dermatitis
SC
Phase 3 – BLA; Fast Track
TBD
lenacapavir
Gilead
HIV-1 infection (heavily treatment-experienced)
Oral (lead-in dose), SC
Phase 3 – NDA; Breakthrough Therapy
TBD
lenadogene nolparvovec (GS010)
Gensight
Leber’s hereditary optic neuropathy
Intraocular
Phase 3 – BLA; Orphan Drug
TBD
lenzilumab
Humanigen
COVID-19
IV
Phase 3 – BLA
TBD
leriglitazone
Minoryx
Adrenomyeloneuropathy (adrenoleukodystrophy)
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
leronlimab
Cytodyn
COVID-19
SC
Phase 3 – BLA
TBD
levodopa/carbidopa patch pump
Mitsubishi Tanabe
Parkinson’s disease
SC
Phase 3 – 505(b)(2) NDA
TBD
levoketoconazole
Strongbridge
Cushing’s syndrome
Oral
Phase 3 – 505(b)(2) NDA; Orphan Drug
TBD
ligelizumab
Novartis
Urticaria
SC
Phase 3 – BLA
TBD
linzagolix
ObsEva
Endometriosis; Uterine fibroids
Oral
Phase 3 – NDA
TBD
L-lactic acid/citric acid/ potassium bitartrate
Evofem
Urinary tract and reproductive tract infections (antibacterial)
Intravaginal
Phase 3 – NDA; Fast Track; QIDP
TBD
lonafarnib
Eiger
Hepatitis D infection
Oral
Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
lorecivivint
Samumed
Osteoarthritis (knee)
Intraarticular
Phase 3 – NDA
TBD
lumateperone
Intra-Cellular Therapies
Bipolar disorder
Oral
Phase 3 – NDA
TBD
lutetium 177Lu-PSMA-617
Novartis
Prostate cancer
IV
Phase 3 – NDA
TBD
LYS-SAF302
Sarepta
Mucopolysaccharidosis IIIA (MPS IIIA; Sanfilippo A syndrome)
Intracerebral
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
maribavir
Takeda
Cytomegalovirus infection treatment
Oral
Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
masitinib
AB Science
Asthma
Oral
Phase 3 – NDA
TBD
mavacamten
Myokardia
Obstructive hypertrophic cardiomyopathy
Oral
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
MBG453
Novartis
Myelodysplastic syndrome
IV
Phase 3 – NDA
TBD
34 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
metachromatic leukodystrophy gene therapy
Orchard
Metachromatic leukodystrophy
IV
Phase 3 – BLA; Orphan Drug
TBD
microbiota suspension
Ferring
Clostridium difficile infection (recurrent)
Rectal
Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
micropine
Eyenovia
Pharmacologic mydriasis
Topical
Phase 3 – NDA
TBD
minocycline
Citius
Catheter-related bacteremia
IV
Phase 3 – 505(b)(2) NDA; Fast Track; QIDP
TBD
mirikizumab
Eli Lilly
PSO; UC
IV, SC
Phase 3 – BLA
TBD
mirvetuximab soravtansine
Immunogen
Ovarian cancer
IV
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
mitapivat
Agios
Pyruvate kinase deficiency
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
mobocertinib
Takeda
NSCLC
Oral
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
mRNA-1273 vaccine
Moderna/NIAID
COVID-19
IM
Phase 3 – BLA; Fast Track
TBD
MT-7117
Mitsubishi Tanabe
Porphyria
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
nabiximols
GW
MS-related spasticity
Oral transmucosal
Phase 3 – NDA
TBD
nadofaragene firadenovec
Trizell
Mesothelioma
Percutaneous injection
Phase 3 – BLA
TBD
nalbuphine ER
Trevi
Pruritus
Oral
Phase 3 – NDA
TBD
napabucasin
Sumitomo Dainippon
CRC
Oral
Phase 3 – NDA
TBD
narsoplimab
Omeros
HSCT-Associated thrombotic microangiopathy
IV, SC
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
natalizumab (biosimilar to Biogen’s Tysabri®)
Novartis
MS
IV
Phase 3 – BLA
TBD
NBI-74788
Neurocrine Biosciences
Congenital adrenal hyperplasia
Oral
Phase 3 – NDA
TBD
nedosiran
Dicerna
Hyperoxaluria
SC
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
nemolizumab
Galderma
Atopic dermatitis
SC
Phase 3 – BLA
TBD
nemorexant
Idorsia
Insomnia
Oral
Phase 3 – NDA
TBD
nirsevimab
AstraZeneca
RSV infection prevention
IM
Phase 3 – BLA; Breakthrough Therapy; Fast Track
TBD
NVX-CoV2373 vaccine
Novavax
COVID-19
IM
Phase 3 – BLA
TBD
odevixibat
Albireo
Progressive familial intrahepatic cholestasis
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
35 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
olipudase alfa
Sanofi
Niemann-Pick disease
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
omidubicel
Gamida Cell
Bone marrow transplant and stem cell transplant
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
ondansetron ER once-daily
Redhill
Gastroenteritis
Oral
Phase 3 – 505(b)(2) NDA
TBD
oportuzumab monatox
Sesen Bio
Bladder cancer (BCGunresponsive, nonmuscle invasive)
Intravesical
Phase 3 – BLA; Fast Track
TBD
OTL-103
Orchard
Wiskott-Aldrich syndrome IV
Phase 3 – BLA; Orphan Drug; RMAT
TBD
oxalobacter formigenes
Oxthera
Hyperoxaluria
Oral
Phase 3 – BLA; Orphan Drug
TBD
pacritinib
CTI Bio
Myelofibrosis
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
paliperidone palmitate 6-month injectable
Janssen
Schizophrenia
IM
Phase 3 – NDA
TBD
palovarotene
Ipsen
Fibrodysplasia ossificans progressiva
Oral
Phase 3 – NDA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
pamrevlumab
Fibrogen
Idiopathic pulmonary fibrosis
IV
Phase 3 – BLA; Fast Track; Orphan Drug
TBD
pegzilarginase
Aeglea
Arginase 1 deficiency
IV
Phase 3 – BLA; Breakthrough Therapy; Fast Track; Orphan Drug
TBD
pevonedistat
Takeda
Myelodysplastic syndrome
IV
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
plinabulin
Beyondspring
NSCLC
IV
Phase 3 – NDA
TBD
plinabulin
Beyondspring
Neutropenia/leukopenia
IV
Phase 3 – NDA; Breakthrough Therapy
TBD
pollinex quattro grass
Allergy Therapeutics
Allergic rhinitis
SC
Phase 3 – BLA
TBD
PTI-NC-733
Proteostasis Therapeutics CF
Oral
Phase 3 – NDA; Fast Track
TBD
ranibizumab (biosimilar to Genentech’s Lucentis®)
Biogen/Samsung Bioepis
Wet AMD
Intraocular
Phase 3 – BLA
TBD
ranibizumab (biosimilar to Genentech’s Lucentis)
Coherus/Santo
Wet AMD
Intraocular
Phase 3 – BLA
TBD
ranibizumab (biosimilar to Genentech’s Lucentis)
STADA Arzneimittel
Wet AMD
Intraocular
Phase 3 – BLA
TBD
REGN-COV2
Regeneron
COVID-19
SC
Phase 3 – BLA
TBD
relacorilant
Corcept
Cushing’s syndrome
Oral
Phase 3 – NDA; Orphan Drug
TBD
reltecimod
Atox
Necrotizing soft tissue infection
IV
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
36 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
relugolix/estradiol/ norethindrone
Myovant
Endometriosis
Oral
Phase 3 – NDA
TBD
reproxalap
Aldeyra
Dry eye syndrome
Ophthalmic
Phase 3 – NDA
TBD
resmetirom
Madrigal
NASH
Oral
Phase 3 – NDA; Fast Track
TBD
ridinilazole
Summit
C. difficile-associated diarrhea
Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
rilzabrutinib
Principia Bio
Pemphigus vulgaris
Oral
Phase 3 – NDA; Orphan Drug
TBD
risperidone long-acting in situ microparticle
Laboratorios Farmacéuticos
Schizophrenia
IM
Phase 3 – 505(b)(2) NDA
TBD
ritlecitinib
Pfizer
Alopecia areata
Oral
Phase 3 – NDA; Breakthrough Therapy
TBD
rituximab (biosimilar to Genentech’s Rituxan)
Archigen
RA; CLL/SLL; NHL (indolent); ANCAassociated vasculitis
IV
Phase 3 – BLA
TBD
rivoceranib
LSK Biopartners
Gastric cancer
Oral
Phase 3 – NDA; Orphan Drug
TBD
roflumilast cream
Arcutis
PSO
Topical
Phase 3 – NDA
TBD
RSV nanoparticle vaccine
Novavax
RSV prevention
IM
Phase 3 – BLA; Fast Track
TBD
SAR408701
Sanofi
NSCLC
IV
Phase 3 – BLA
TBD
SARS-CoV-2 inactivated vaccine
Sinovac
COVID-19
IM
Phase 3 – BLA
TBD
SARS-CoV-2 inactivated vaccine (Vero cell)
Sinopharm/Beijing
COVID-19
IM
Phase 3 – BLA
TBD
SARS-CoV-2 inactivated vaccine (Vero cell)
Sinopharm/Wuhan
COVID-19
IM
Phase 3 – BLA
TBD
SARS-CoV-2 vaccine (adeno-based)
Gamaleya Research Institute
COVID-19
IM
Phase 3 – BLA
TBD
scCeftriaxone
scPharmaceuticals
Antibacterial (general)
SC
Phase 3 – NDA
TBD
seladelpar
Cymabay
Primary biliary cholangitis/hepatic fibrosis
Oral
Phase 3 – NDA; Breakthrough Therapy; Orphan Drug
TBD
sepofarsen
Proqr
Leber’s congenital amaurosis
Intraocular
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
SER-109
Seres
C. difficile infection (recurrent)
Oral
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
sodium hyaluronate/ triamcinolone hexacetonide
Anika
Osteoarthritis (knee)
Intraarticular
Phase 3 – NDA
TBD
sodium oxybate oncenightly dosing
Avadel
Narcolepsy
Oral
Phase 3 – 505(b)(2) NDA; Orphan Drug
TBD
sofpironium
Brickell
Axillary hyperhidrosis
Topical
Phase 3 – NDA
TBD
somatrogon
Opko
Growth hormone deficiency (pediatric)
SC
Phase 3 – BLA; Orphan Drug
TBD
sparsentan
Retrophin
Focal segmental glomerulosclerosis
Oral
Phase 3 – NDA; Orphan Drug
TBD
37 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
spartalizumab
Novartis
Melanoma
IV
Phase 3 – BLA
TBD
sulopenem etzadroxil
Iterum
UTI (uncomplicated, quinolone-resistant)
IV, Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
tabelecleucel
Atara
Epstein-Barr virus-associated post-transplant lymphoproliferative disease
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
tapinarof
Roivant
PSO
Topical
Phase 3 – NDA
TBD
tebipenem pivoxil
Spero
Urinary tract and reproductive tract infections (antibacterial)
Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
tecarfarin
Espero
Anticoagulation
Oral
Phase 3 – NDA
TBD
teplizumab
Provention Bio
T1DM (prevention or delay)
IV
Phase 3 – BLA; Breakthrough Therapy; Orphan Drug
TBD
tesetaxel
Odonate
Breast cancer
Oral
Phase 3 – NDA
TBD
tezepelumab
AstraZeneca
Asthma (severe, uncontrolled)
SC
Phase 3 – BLA; Breakthrough Therapy
TBD
timbetasin
Regenerx
Dry eye syndrome
Ophthalmic
Phase 3 – BLA
TBD
tirzepatide
Eli Lilly
T1DM
SC
Phase 3 – NDA
TBD
tofersen
Biogen
Amyotrophic lateral sclerosis
Intrathecal
Phase 3 – NDA; Orphan Drug
TBD
tominersen
Genentech
Huntington’s disease
Intrathecal
Phase 3 – NDA; Orphan Drug
TBD
tonogenchoncel-L
Kolon Tissuegene
Osteoarthritis
Intraarticular
Phase 3 – BLA
TBD
tradipitant
Vanda
Gastroparesis; Emesis; COVID-19
Oral
Phase 3 – NDA
TBD
trastuzumab (biosimilar to Genentech’s Herceptin)
Novartis
Breast cancer; Gastric/ gastroesophageal cancer
IV
Phase 3 – BLA
TBD
trastuzumab (biosimilar to Genentech’s Herceptin)
Tanvex
Breast cancer; Gastric/ gastroesophageal cancer
IV
Phase 3 – BLA
TBD
trastuzumab duocarmazine
Synthon Bio
Breast cancer
IV
Phase 3 – BLA; Fast Track
TBD
triamcinolone acetonide
Clearside
Uveitis
Intraocular
Phase 3 – 505(b)(2) NDA
TBD
tripotassium citrate monohydrate/potassium hydrogen carbonate microtablet
Advicenne
Renal tubular acidosis
Oral
Phase 3 – NDA
TBD
trivalent hepatitis B vaccine
VBI Vaccines
Hepatitis B infection prevention
IM
Phase 3 – BLA
TBD
trofinetide
Acadia
Rett syndrome
Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
ublituximab
TG
MS
IV
Phase 3 – BLA
TBD
ublituximab + umbralisib
TG
CLL/SLL
IV + Oral
Phase 3 – NDA; Fast Track; Orphan Drug
TBD
umbralisib
TG
Mantle cell lymphoma
Oral
Phase 3 – NDA
TBD
vadadustat
Akebia
Anemia due to CKD (dialysis-dependent)
Oral
Phase 3 – NDA
TBD
38 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
vazegepant
Biohaven
Migraine treatment; COVID-19
Intranasal
Phase 3 – NDA
TBD
veliparib
Abbvie
Breast cancer; Ovarian cancer
Oral
Phase 3 – NDA
TBD
venglustat
Sanofi
Polycystic kidney disease
Oral
Phase 3 – NDA
TBD
VGX-3100 therapeutic vaccine
Inovio
Cervical dysplasia
IM
Phase 3 – BLA
TBD
vilanterol trifenatate
GlaxoSmithKline
Asthma; COPD
Inhaled
Phase 3 – NDA
TBD
visomitin
Mitotech
Dry eye syndrome
Ophthalmic
Phase 3 – NDA
TBD
vonoprazan fumarate
Phathom
Esophagitis; H. pylori infection
Oral
Phase 3 – NDA; QIDP
TBD
VT-1161
Mycovia
Vulvovaginal candidiasis
Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
vutrisiran
Alnylam
Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary)
SC
Phase 3 – NDA; Orphan Drug
TBD
ziritaxestat
Galapos
Idiopathic pulmonary fibrosis
Oral
Phase 3 – NDA; Orphan Drug
TBD
zoliflodacin
Entasis
Gonorrhea
Oral
Phase 3 – NDA; Fast Track; QIDP
TBD
albuterol (ProAir® RespiClick®)
Teva
COPD
Inhaled
Phase 3 – sNDA
TBD
anakinra (Kineret®)
Swedish Orphan Biovitrum
COVID-19
SC
Phase 3 – sBLA
TBD
bacille Calmette-Guérin (BCG) (TICE® strain) vaccine
Merck
COVID-19
Intradermal
Phase 3 ─ sBLA
TBD
baricitinib (Olumiant®)
Eli Lilly
COVID-19
Oral
Phase 3 – sNDA
TBD
benralizumab (Fasenra®)
AstraZeneca
Nasal polyposis
SC
Phase 3 – sBLA
TBD
brexpiprazole (Rexulti )
Otsuka
Alzheimer’s diseaserelated agitation; PTSD
Oral
Phase 3 – sNDA; Fast Track
TBD
canakinumab (Ilaris®)
Novartis
COVID-19
IV
Phase 3 – BLA
TBD
dapagliflozin (Farxiga)
AstraZeneca
CKD; Diabetic nephropathy; COVID-19
Oral
Phase 3 – sNDA; Breakthrough Therapy; Fast Track
TBD
dupilumab (Dupixent®)
Sanofi
Esophagitis; Pruritus; Urticaria
SC
Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug
TBD
empagliflozin (Jardiance)
Boehringer Ingelheim
Chronic heart failure; CKD; Diabetic nephropathy
Oral
Phase 3 – sNDA; Fast Track
TBD
fenfluramine (Fintepla®)
Zogenix
Lennox-Gastaut syndrome
Oral
Phase 3 – sNDA; Orphan Drug
TBD
hydrogen peroxide (Eskata®)
Aclaris
Warts
Topical
Phase 3 – sNDA
TBD
immunoglobulin 5% (Octagam®)
Octapharma
COVID-19
IV
Phase 3 – sBLA
TBD
mepolizumab (Nucala®)
GlaxoSmithKline
Nasal polyposis
SC
Phase 3 – sBLA
TBD
meropenem/vaborbactam (Vabomere®)
Melinta
Bacteremia; HAP
IV
Phase 3 – sNDA; QIDP TBD
Phase 3 (Supplementals)
®
39 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
nitazoxanide (Alinia®)
Lupin
Influenza treatment; COVID-19
Oral
Phase 3 – sNDA
TBD
omalizumab (Xolair®) autoinjector
Genentech
Food allergies
SC
Phase 3 – sBLA; Breakthrough Therapy
TBD
ozanimod (Zeposia®)
Celgene
UC
Oral
Phase 3 – sNDA
TBD
polatuzumab vedotin-piiq (Polivy®)
Genentech
DLBLC (1st-line)
IV
Phase 3 – sBLA; Orphan Drug
TBD
prasterone (Intrarosa®)
AM
Female sexual arousal disorder
Intravaginal
Phase 3 – sNDA
TBD
ravulizumab-cwvz (Ultomiris®)
Alexion
COVID-19
IV
Phase 3 – sBLA
TBD
rilonacept (Arcalyst®)
Regeneron
Recurrent pericarditis
SC
Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug
TBD
risankizumab-rzaa (Skyrizi®)
Abbvie
PsA; CD; UC
IV, SC
Phase 3 – sBLA; Orphan Drug
TBD
ruxolitinib (Jakafi®)
Incyte
COVID-19
Oral
Phase 3 – sNDA
TBD
sacituzumab govitecanhziy (Trodelvy™)
Immunomedic
Bladder cancer
IV
Phase 3 – sBLA; Fast Track
TBD
sacubitril/valsartan (Entresto)
Novartis
Post-acute myocardial infarction
Oral
Phase 3 – sNDA; Fast Track
TBD
secnidazole (Solosec®)
Lupin
Trichomoniasis
Oral
Phase 3 – sNDA
TBD
selinexor (Xpovio)
Karyopharm
Liposarcoma
Oral
Phase 3 – sNDA; Orphan Drug
TBD
semaglutide (Ozempic®)
Novo Nordisk
Obesity
SC
Phase 3 – sNDA
TBD
teriflunomide (Aubagio )
Sanofi
MS (pediatrics)
Oral
Phase 3 – sNDA
TBD
ticagrelor (Brilinta )
AstraZeneca
Sickle cell disease
Oral
Phase 3 – sNDA
TBD
tisagenlecleucel-t (Kymriah™)
Novartis
DLBCL (relapsed/ refractory in 1st relapse)
IV
Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug
TBD
tocilizumab (Actemra®)
Genentech
COVID-19
IV
Phase 3 – sBLA
TBD
tofacitinib (Xeljanz / Xeljanz XR®)
Pfizer
JIA
Oral
Phase 3 – sNDA
TBD
®
®
®
Complete Response Letter (CRL)/Withdrawn Drugs NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
dantrolene
Eagle
Heat stroke (exertional)
IV
CRL
TBD
diazepam film
Aquestive
Seizure clusters
Oral transmucosal
CRL
TBD
filgotinib (Jyseleca®)
Gilead
RA
Oral
CRL
TBD
sodium thiosulfate
Fennec
Cisplatin-induced ototoxicity prevention
IV
CRL
TBD
terlipressin
Mallinckrodt
Hepatorenal syndrome type 1
IV
CRL
TBD
tramadol
Fortress
Pain (moderate to severe, medically supervised setting)
IV
CRL
TBD
40 | magellanrx.com
PIPELINE DRUG LIST continued NAME
MANUFACTURER
DOSAGE FORM
CLINICAL USE
APPROVAL STATUS
FDA APPROVAL
valoctocogene roxaparvovec
Biomarin
Hemophilia A
IV
CRL
TBD
veverimer
Tricida
CKD-related metabolic acidosis
Oral
CRL
TBD
viaskin peanut immunotherapy
DBV
Peanut allergy (ages 4 to 11 years)
Transdermal
CRL
TBD
zolmitriptan micro-needle patch
Zosano
Migraine treatment
Transdermal
CRL
TBD
41 | magellanrx.com
GLOSSARY 6MWT 6 Minute Walking Test
CD Crohn's Disease
ABSSSI Acute Bacterial Skin and Skin Structure Infection
CDC Centers for Disease Control and Prevention
ACEI Angiotensin-Converting Enzyme Inhibitor ACR20 American College of Rheumatology 20% Improvement ACR50 American College of Rheumatology 50% Improvement ACR70 American College of Rheumatology 70% Improvement ADHD Attention Deficit Hyperactivity Disorder ADL Activities of Daily Living AED Anti-Epileptic Drug ALK Anaplastic Lymphoma Kinase ALL Acute Lymphoblastic Leukemia ALT Alanine Transaminase AMD Age-Related Macular Degeneration AML Acute Myeloid Leukemia ANCA Antineutrophil Cytoplasmic Antibody ANDA Abbreviated New Drug Application ARB Angiotensin II Receptor Blocker ARNI Angiotensin Receptor II Blocker 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 BsUFA Biosimilar User Fee Act BMI Body Mass Index CABP Community Acquired Bacterial Pneumonia CAP Community Acquired Pneumonia 42 | magellanrx.com
CF Cystic Fibrosis CHF Congestive Heart Failure CI Confidence Interval CKD Chronic Kidney Disease CLL Chronic Lymphocytic Leukemia CNS Central Nervous System COPD Chronic Obstructive Pulmonary Disease COVID-19 Coronavirus Disease 2019 CRC Colorectal Cancer CRL Complete Response Letter CSF Colony Stimulating Factor CV Cardiovascular CVD Cardiovascular Disease DAS28-CRP Disease Activity Score-28 with C Reactive Protein DEA Drug Enforcement Administration DLBCL Diffuse Large B Cell Lymphoma DMD Duchenne Muscular Dystrophy DMARD Disease Modifying Antirheumatic Drug DNA Deoxyribonucleic Acid DOR Duration of Response DPP-4 Dipeptidyl Peptidase 4 DR Delayed-Release ECOG Eastern Cooperative Oncology Group EDSS Expanded Disability Status Scale EGFR Epidermal Growth Factor Receptor ER Extended-Release FDA Food and Drug Administration FH Familial Hypercholesterolemia FLT3 FMS-Like Tyrosine Kinase-3
GLOSSARY continued G-CSF Granulocyte Colony Stimulating Factor
LDL-C Low-Density Lipoprotein Cholesterol
GM-CSF Granulocyte-Macrophage Colony Stimulating Factor
mAb Monoclonal Antibody
GI Gastrointestinal GIST Gastrointestinal Stromal Tumor GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist GVHD Graft Versus Host Disease H Half HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HCC Hepatocellular Carcinoma HCP Healthcare Professional HCV Hepatitis C Virus HER Human Epidermal Growth Factor Receptor HER2 Human Epidermal Growth Factor Receptor 2 HFA Hydrofluoroalkane HIT Heparin Induced Thrombocytopenia HIV Human Immunodeficiency Virus HIV-1 Human Immunodeficiency Virus-1 HR Hazard Ratio HSCT Hematopoietic Stem Cell Transplant HTN Hypertension IBS Irritable Bowel Syndrome IBS-C Irritable Bowel Syndrome, Constipation Predominant
MACE Major Adverse Cardiovascular Events MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler MRI Magnetic Resonance Imaging MRSA Methicillin-Resistant Staphylococcus Aureus MS Multiple Sclerosis N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NDA New Drug Application NHL Non-Hodgkin Lymphoma NIAID National Institute of Allergy and Infectious Diseases NSAID Non-Steroidal Anti-Inflammatory Drug NSCLC Non-Small Cell Lung Cancer ODT Orally Disintegrating Tablet OR Odds Ratio ORR Overall/Objective Response Rate OS Overall Survival PAH Pulmonary Arterial Hypertension PARP Poly (ADP-ribose) Polymerase PASI 50 Psoriasis Area and Severity Index ≥ 50% PASI 70 Psoriasis Area and Severity Index ≥ 70% PASI 90 Psoriasis Area and Severity Index ≥ 90%
IM Intramuscular
PCI Percutaneous Coronary Intervention
ITP Immune Thrombocytopenic Purpura
PD-1 Programmed Death Protein 1
ITT Intent-To-Treat
PD-L1 Programmed Death-Ligand 1
IV Intravenous
PDUFA Prescription Drug User Fee Application
JIA Juvenile Idiopathic Arthritis
PFS Progression-Free Survival
43 | magellanrx.com
GLOSSARY continued PGA Physician Global Assessment
sPGA Static Physician Global Assessment
PsA Psoriatic Arthritis
SNRI Serotonin and Norepinephrine Reuptake Inhibitor
PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty
SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor
PTSD Post-Traumatic Stress Disorder
SSSI Skin and Skin Structure Infection
Q Quarter
T1DM Type 1 Diabetes Mellitus
QIDP Qualified Infectious Diseases Product
T2DM Type 2 Diabetes Mellitus
QOL Quality of Life
TBD To Be Determined
R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone
TEAE Treatment-Emergent Adverse Events
RA Rheumatoid Arthritis RBC Red Blood Cell RCC Renal Cell Carcinoma REMS Risk Evaluation and Mitigation Strategy RMAT Regenerative Medicine Advanced Therapy RNA Ribonucleic Acid RRR Relative Risk Reduction RSV Respiratory Syncytial Virus RTOR Real-Time Oncology Review sBLA supplemental Biologics License Application SC Subcutaneous SCCHN Squamous Cell Cancer of the Head and Neck SCLC Small Cell Lung Cancer SCT Stem Cell Transplant SGLT2 Sodium-Glucose Co-Transporter-2 SL Sublingual SLE Systemic Lupus Erythematosus SLL Small Lymphocytic Lymphoma sNDA supplemental New Drug Application SOC Standard of Care
44 | magellanrx.com
TNBC Triple Negative Breast Cancer TNF Tumor Necrosis Factor TNFα Tumor Necrosis Factor-alpha UA Unstable Angina UC Ulcerative Colitis US United States UTI Urinary Tract Infection VAS Visual Analog Scale VEGF Vascular Endothelial Growth Factor VTE Venous Thromboembolism WBC White Blood Cell WHO World Health Organization XR Extended-Release
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