MRx Pipeline - July 2022 by Prime/Magellan Rx

MR x PIPELINE MRx AAVIEW & TRADITIONAL TRADITIONALDRUGS DRUGS VIEWINTO INTO UPCOMING UPCOMING SPECIALTY SPECIALTY &

JULY 2022 JANUARY 2022

Table of CONTENTS

EDITORIAL STAFF Maryam Tabatabai, PharmD Editor-in-Chief Vice President, Clinical Information Carole Kerzic, RPh Executive Editor Drug Information Pharmacist

EDITOR-IN-CHIEF'S MESSAGE

Consultant Panel

PIPELINE DEEP DIVE

Michelle Booth, PharmD Director, Specialty Clinical Solutions

KEEP ON YOUR RADAR

PIPELINE DRUG LIST

GLOSSARY

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

Robert Greer, RPh, BCOP Vice President, Clinical Strategy and Programs Katie Lockhart Manager, Forecasting and Pharmacoeconomics Brian MacDonald, PharmD Director, Specialty Clinical Strategy Simone Ndujiuba, PharmD, BCOP Director, Clinical Strategy and Innovation, Oncology Nothing herein is or shall be construed as a promise or representation regarding past or future events and Magellan Rx Management expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Magellan Rx Management.

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

METHODOLOGY

Emerging therapeutics continue to grow and influence the clinical and financial landscape. Therefore, Magellan Rx Management has developed a systematic approach to determine the products with significant clinical impact. For the in-depth clinical evaluations, the products’ potential to meet an underserved need in the market by becoming the new standard of care, and the ability to replace existing therapies were investigated. The extent to which the pipeline drugs could shift market share on a formulary and their impact on disease prevalence were also important considerations. In order to assist payers with assessing the potential impact of these pipeline drugs, where available, a financial forecast has been included for select products. Primarily complemented by data from EvaluateTM, this pipeline report looks ahead at the 5-year projected annual US sales through the year 2026. These figures are not specific to a particular commercial or government line of business; rather, they look at forecasted total US sales. Depending on a variety of factors, including the therapeutic categories, eventual FDA-approved indications, populations within the plan, and other indices, the financial impact could vary by different lines of business.

REFLECTION

Thus far in 2022, the FDA has approved 16 novel drugs, about half the number of approvals compared to the same time last year. There continues to be a trend for drugs approved with at least 1 of the FDA’s expedited approval methods and drugs designated as Orphan Drugs. This year has also brought the FDA approval and authorization of COVID-19 vaccines as well as their expanded use in younger ages. In 2022 oral treatment options for COVID-19 received EUAs and the FDA authorized state-licensed pharmacists to prescribe the oral antiviral PaxlovidTM to eligible patients. While numbers do not tell the entire story, they do represent significant innovation in patient care and advance public health for the American public.

ON THE HORIZON

As we look ahead, there is a continued trend towards the approval of specialty medications and drugs for rare conditions, with 68% and 38% of approvals expected, respectively, for agents with applications submitted to the FDA. There are 4 agents seeking FDA’s Accelerated Approval, which allows for earlier drug approval for serious conditions that fill an unlet need based on a surrogate endpoint reasonably likely to predict a clinical benefit. New treatment modalities using gene therapy including for hemophila and the growth of biosimilars including interchangeable biosimilars are expected. Further, the launch of Humira® biosimilars in 2023 are highly anticipated. Other noteworthy pipeline trends to watch include the development of complex therapies, oncology, immunology, immunotherapy, new monoclonal antibodies for Alzheimer’s disease, and therapeutic options for rare hereditary diseases. Moreover, sprouting products for RSV, behavioral health including women’s mental health, and therapeutic psychedelics await on the horizon. The drug pipeline ecosphere will continue to evolve as it faces challenges and successes. Innovative agents that show positive results without compromising patient safety and access offer true therapeutic advances and hold the promise to alter the treatment paradigm Maryam Tabatabai, PharmD Editor-in-Chief, MRx Pipeline

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

SPECIALTY

PRIORITY REVIEW

BREAKTHROUGH THERAPY

79%

21%

11%

BIOSIMILAR

ORPHAN DRUG

64%

18%

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

HEMATOLOGY

daprodustat oral GlaxoSmithKline PROPOSED INDICATIONS

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

CLINICAL OVERVIEW

Daprodustat is a hypoxia-inducible factor-prolyl hydroxylase inhibitor (HIF PHI). HIF PHIs stimulate erythropoietin production in the kidneys and liver, improve iron storage, and downregulate hepcidin – a key regulator of iron homeostasis. Two open-label, phase 3 trials evaluated daprodustat in patients with CKD-related anemia (Hb, 8 to 11.5 g/dL). In ASCEND-D (NCT02879305; n=2,964) patients on dialysis were randomized to oral daprodustat or an injectable ESA (epoetin alfa if on hemodialysis or darbepoetin alfa if on peritoneal dialysis). In ASCEND-ND (NCT03400033; n=3,872), patients not on dialysis were randomized to daprodustat or darbepoetin alfa. In both trials, daprodustat demonstrated non-inferiority to ESAs for the primary efficacy endpoint of the mean change in Hb from baseline to weeks 28 through 52 (ASCEND-D, 0.28 versus 0.1 g/dL, respectively [p<0.001]; ASCEND-ND, 0.74 versus 0.66 g/dL, respectively [p<0.001]). In both trials, daprodustat also demonstrated noninferiority to ESA therapy in the primary safety endpoint of rate of first occurrence of MACE (ASCEND-D, 25.2% versus 26.7%, respectively [p<0.001]; ASCEND-ND, 19.5% versus 19.2%, respectively [p=0.005]), during the 2.5 years of follow up in ASCEND-D and 1.9 years in ASCEND-ND. Non-inferiority of daprodustat relative to ESA therapy was also seen when MACE was expanded to include thrombotic events and hospitalization for HF. In both trials, ophthalmic complications, including proliferative retinopathy, occurred at similar rates between the treatment groups. In ASCEND-ND, daprodustat was associated with significantly higher rates of cancer-related death or tumor progression (3.7% versus 2.5%, respectively [p=0.04]) and esophageal/gastric erosions (3.6% versus 2.1%, respectively [p=0.005]) compared to ESAs; this was not seen in ASCEND-D.

PLACE IN THERAPY

An estimated 37 million (1 in 7) people in the US have CKD. The incidence varies according to population, including African Americans (16%), Hispanics (14%), Caucasians (13%), and Asians (13%). Anemia is prevalent in patients with CKD and worsens as CKD progresses. Anemia can lead to arrhythmia, cardiomegaly, and HF as well as fatigue and decreased QOL. Treatment with iron, vitamin B12 and folic acid supplementation, ESAs (epoetin alfa [Epoetin®, Procrit®, biosimilars], darbepoetin alfa [Aranesp®]), and RBC transfusions is used to manage anemia. However, transfusions can reduce the patient’s eligibility for kidney transplant and increase risk of infections, HF, allergic reactions, and iron overload. In addition, ESAs are associated with an increased risk of MACE and thromboembolism. If approved, daprodustat will be the first HIF PHI agent in the US for CKD-related anemia. Like ESAs, HIF PHIs are associated with increased CV and cancer risks. In clinical trials, daprodustat was non-inferior to ESAs in improving or maintaining Hb levels and showed no increase in CVD, including thrombosis, compared to ESA therapy. However, cancer-related death or tumor progression was more prevalent in non-dialysis patients treated with daprodustat than those given an ESA. Notably, as with other investigational HIF PHI agents, daprodustat did not slow the progression of CKD when compared to ESAs in clinical trials. Other HIF PHIs in phase 3 trials for CKD-related anemia include oral roxadustat and vadadustat. After previous submissions, both roxadustat and vadadustat received Complete Response Letters (CRLs) from the FDA requesting additional study data due to safety concerns (e.g., MACE, thromboembolic events, drug-induced liver injury).

FDA APPROVAL TIMELINE February 1, 2023

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$58

$195

$535

$808

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HEMATOLOGY

etranacogene dezaparvovec IV CSL Behring PROPOSED INDICATIONS Hemophilia B

CLINICAL OVERVIEW

Etranacogene dezaparvovec (etrana-dez) is a recombinant adeno-associated viral vector of serotype 5 (AAV5) gene therapy containing a codon-optimized Padua gene variant human factor IX (FIX) transgene. Etrana-dez was evaluated in the open-label, single-arm, phase 3 HOPE-B trial (NCT03569891) in 54 adult males with moderately severe or severe hemophilia B, defined as having ≤ 2% of normal FIX activity. Patients enrolled required prophylactic FIX replacement therapy. Thirty-one patients had pre-existing AAV5-neutralizing antibodies (NAbs), while 23 did not. Etrana-dez led to a mean FIX activity of 39 IU/dL at 6 months after the dose and 36.9 IU/dL at 18 months post dose. Steady-state FIX transgene expression likely occurred during 7 to 18 months post dose. During this time, the adjusted annualized bleeding rate (ABR) decreased by 64% (p=0.0002) and all FIX-treated bleeds declined by 77% (p<0.0001). Notably, 98% of patients who received a full dose of etrana-dez discontinued prophylactic FIX replacement therapy. Etrana-dez was generally well tolerated, with no treatment-related deaths reported. FIX inhibitors were not detected. In addition, in a phase 2b study (NCT03489291), sustained FIX activity was demonstrated at 2 years in 3 patients who received the full dose of etrana-dez. The mean FIX activity was 44.2% of normal at 2 years. Etrana-dez was administered as a one-time dose of 2 x 1013 genome copies per kilogram (gc/kg) via IV infusion.

PLACE IN THERAPY

Hemophilia B is a rare genetic bleeding disorder caused by FIX gene mutations. In a subset of patients, hemophilia B is acquired after the production of FIX-directed antibodies in the body. Hemophilia B accounts for 20% of all hemophilia cases, while hemophilia A accounts for 80%. In hemophilia B, insufficient levels of FIX, a blood clotting protein, may result in prolonged bleeding. In moderate cases, FIX level is 1% to 5% of normal, and in severe cases, it is < 1% of normal. Bleeding symptoms may arise spontaneously or after a minor injury and can occur in the muscles, joints, organs, and brain. Incidence of hemophilia B is approximately 1 in 25,000 male births. The preferred treatment for hemophilia B is recombinant FIX therapy (Alprolix®, Benefix®, Idelvion®, Ixinity®, Rebinyn®, and Rixubis®). Other options include plasma-derived FIX concentrates (Alphanine SD®, Mononine®, and Profilnine SD®), plasma-derived activated prothrombin complex (Feiba®; for select patients with FIX inhibitors), and fresh frozen plasma. If approved, etrana-dez will be the first gene replacement DMT for the treatment of hemophilia B. In clinical trials, it produced a durable improvement in FIX activity after a single dose. Most patients treated with etranadez discontinued prophylactic FIX replacement therapy. The viral gene therapy fidanacogene elaparvovec by Pfizer/Roche is in phase 3 trials for hemophilia B, with data expected in Q1 2023.

FDA APPROVAL TIMELINE November 2022

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$14

$91

$155

$236

$290

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ONCOLOGY

futibatinib oral Obseva PROPOSED INDICATIONS

Locally advanced or metastatic cholangiocarcinoma (CCA) harboring fibroblast growth factor receptor 2 (FGFR2) gene rearrangements, including gene fusions

CLINICAL OVERVIEW

Futibatinib is a selective and irreversible tyrosine kinase inhibitor of FGFR1, 2, 3, and 4. Futibatinib was evaluated in the single-arm, phase 2 FOENIX-CCA2 study (NCT02052778) in 103 patients with locally advanced or metastatic, unresectable, intrahepatic CCA with FGFR2 gene rearrangement, including fusions. Patients had received ≥ 1 prior systemic treatment (e.g., gemcitabine plus platinumbased chemotherapy). At a median follow-up of 25 months, the primary endpoint of ORR as assessed by independent central review was 41.7%. The study also reported a median PFS of 8.9 months, median OS of 20 months, median DOR of 9.5 months, and disease control rate of 82.5%. The most common TEAEs were of mild to moderate severity. These included alopecia, dry mouth, diarrhea, dry skin, and fatigue. No treatmentrelated deaths occurred. The phase 3, open-label FOENIX-CCA3 trial is underway comparing futibatinib with cisplatin plus gemcitabine chemotherapy as first-line treatment in patients with intrahepatic CCA. The futibatinib dosage was 20 mg orally once daily until disease progression or unacceptable toxicity.

PLACE IN THERAPY

CCAs originate in the bile duct epithelium. Approximately 8,000 cases are diagnosed each year in the US, with approximately 10% of those cases being intrahepatic CCA and 90% being extrahepatic CCA. The average age at diagnosis is 70 to 72 years. Five-year survival rates for localized, regional, and distant intrahepatic CCAs are 24%, 9%, and 2%, respectively, and for extrahepatic CCAs are 17%, 16%, and 2%, respectively. The only potential curative treatment for CCA is complete surgical resection. However, most patients are not surgical candidates since advanced disease is often found at initial diagnosis. According to the NCCN, primary treatment options for patients with unresectable or metastatic disease include: systemic chemotherapy (preferred gemcitabine plus cisplatin), enrollment in a clinical trial, or best supportive care. Fluoropyrimidine chemoradiation may also be used in patients with unresectable disease. If approved, futibatinib will be the third medication available to treat CCA that harbors FGFR2 gene rearrangements, following the oral kinase inhibitors infigratinib (Truseltiq®) and pemigatinib (Pemazyre®). Infigratinib and pemigatinib are indicated for unresectable intrahepatic and extrahepatic CCA. While the application to the FDA for approval of futibatinib for the treatment of CCA does not specify intrahepatic CCA only, the pivotal phase 2 trial only reports on its use in patients with unresectable intrahepatic CCA. Futibatinib is also in phase 2 trials for bladder cancer and solid tumors.

FDA APPROVAL TIMELINE September 30, 2022

 Breakthrough Therapy

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$16

$30

$48

$64

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NEUROLOGY

lecanemab IV Eisai/Biogen PROPOSED INDICATIONS

Early Alzheimer’s disease (AD) with confirmed presence of amyloid pathology in the brain

CLINICAL OVERVIEW

Lecanemab is an immunoglobulin G1 (lgG1) antibody that is designed to eliminate neurotoxic amyloid-beta (Aβ) protofibrils. Lecanemab was evaluated in the double-blind, placebo-controlled, phase 2, proof-of-concept Study 201 CORE trial (NCT01767311). It enrolled 856 individuals with early AD (mild cognitive impairment due to AD and mild AD dementia) with amyloid plaque detected by amyloid positron emission tomography (PET) or cerebral spinal fluid (CSF) Aβ1–42. At 12 months, based on a Bayesian adaptive analysis, the trial did not meet the primary endpoint of super-superiority to placebo as measured by the AD Composite Score (ADCOMS), which indicates a > 80% probability of > 25% slowing in cognitive decline. However, at 18 months, the study reported that lecanemab 10 mg/kg given IV every 2 weeks led to significant improvements in key secondary endpoints. These included a reduction of amyloid in the brain, based on an adjusted mean change from baseline by −0.3 in the PET standard update value ratio (SUVr) (mean baseline, 1.37). Slowing of cognitive decline by 30% based on ADCOMS, by 26% based on the Clinical Dementia Rating Scale-Sum of Boxes (CDR-SB), and by 47% based on the AD Assessment Scale–Cognitive Subscale (ADAS-cog) was also observed at 18 months. In addition, > 80% of individuals became amyloid negative (per visual read). The rate of amyloid-related imaging abnormalities-edema/effusion (ARIA-E) was 9.9% with lecanemab (18.8% were symptomatic) compared to 0.8% with placebo during the first year of treatment.

PLACE IN THERAPY

AD is a neurodegenerative disorder characterized by cognitive and memory decline that is associated with impairment of ADLs and behavioral disturbances. It is the most common form of dementia. AD is estimated to affect over 5 million elderly people in the US, and its incidence is expected to nearly triple by 2060. The presence in the brain of amyloid plaque, as well as abnormalities of cholinergic, glutamate, and serotonin systems, are thought to contribute to the development of AD. Current pharmacotherapy for mild to moderate AD includes transdermal and/or oral acetylcholinesterase inhibitors (donepezil, galantamine, rivastigmine) and oral N-methyl-D-asparate (NMDA) receptor antagonists (memantine). A fixed-dose combination of donepezil and memantine is available for moderate to severe disease. In addition, the once-monthly IV amyloid betadirected antibody aducanumab-avwa (Aduhelm®) was granted an Accelerated Approval for early AD in June 2021. Its FDA-approval was met with significant controversy due to uncertainty of benefit. Moreover, CMS limited coverage of aducanumab-avwa to patients enrolled in clinical trials, and coverage of future anti-amyloids would also be restricted unless full approval is granted on the basis of confirmatory trials. Hence, results of the phase 3 CLARITY AD confirmatory trial, expected in fall 2022, will be central to the potential future of lecanemab. If approved, lecanemab will be the second amyloid-targeting DMT for early AD. In non-comparative clinical trials, ARIA-E, which may lead to dose interruption or treatment discontinuation, was reported less often with lecanemab (9.9%) than with aducanumab-avwa (35%). Other injectable anti-amyloid antibodies in late-stage development for AD include donanemab, gantenerumab, and solanezumab.

FDA APPROVAL TIMELINE January 6, 2023

 Breakthrough Therapy

 Fast Track

 Priority Review

 seeking Accelerated Approval

Eisai plans to submit for full approval by March 31, 2023.

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$70

$241

$496

$762

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ONCOLOGY

mirvetuximab soravtansine IV Immunogen PROPOSED INDICATIONS

Ovarian cancer that is folate receptor alpha (FRα)-high platinum-resistant after 1 to 3 prior systemic treatments

CLINICAL OVERVIEW

Mirvetuximab soravtansine is an investigational ADC. It contains DM4, a tubulin-targeting cytotoxic maytansinoid, which is linked to a folate receptor alpha (FRα)-binding antibody. FORWARD-1 (NCT02631876) is an open-label, phase 3 trial in 366 women with platinum-resistant medium or high FRα-positive advanced epithelial ovarian cancer (EOC), primary peritoneal cancer, or fallopian tube cancer who had been treated with 1 to 3 prior systemic regimens. Women with ECOG status of 0 or 1 were randomized 2:1 to mirvetuximab soravtansine 6 mg/kg IV every 21 days or investigator’s choice chemotherapy (e.g., paclitaxel, pegylated liposomal doxorubicin, topotecan). After a median follow-up of 12. 5 months, mirvetuximab soravtansine did not lead to a significant difference over chemotherapy in the primary endpoint of PFS in either the ITT population (HR, 0.981; p=0.897) or the FRα high group (HR 0.693; p=0.049, not significant based on the Hochberg analysis protocol). For the FRα high population, nonsignificant improvements, as determined by the analysis protocol, were also reported in secondary endpoints, including OS (HR, 0.618) and ORR (22% versus 10% for mirvetuximab soravtansine versus chemotherapy, respectively). TEAEs included nausea, diarrhea, fatigue, blurred vision, keratopathy, and peripheral neuropathy. Fewer grade ≥ 3 TEAEs were reported with mirvetuximab than with chemotherapy (25.1% versus 44%, respectively). The phase 3, single-arm SORAYA trial evaluated mirvetuximab soravtansine in 106 patients with FRα high platinum-resistant EOC who had received 1 to 3 prior therapies, including bevacizumab. Per investigator assessment, at data cut-off, the primary endpoint of ORR was 32.4%, of which 5 cases were complete responses. The disease control rate was 51.4%, and tumor reduction was observed in 71.4% of patients. The median time to response was 1.5 months, median DOR was 6.9 months, median PFS was 4.3 months, and median OS was 13.8 months. The most common TEAEs were blurred vision, keratopathy, and nausea.

PLACE IN THERAPY

EOC accounts for approximately 90% of all malignant ovarian neoplasms. It is the major gynecologic cancer that leads to death, with a 5-year survival rate of 48%. Prognosis for many women, particularly those with advanced, recurrent, or resistant disease, remains poor. It is estimated that 19,880 new cases of EOC will be diagnosed and 12,810 related deaths will occur in the US in 2022. Treatment involves debulking surgery, followed by systemic chemotherapy (e.g., platinum- and taxanebased agents, ± bevacizumab [Avastin®, biosimilars]). An oral PARP inhibitor (e.g., niraparib [Zejula®], olaparib [Lynparza®], and rucaparib [Rubraca®]) may accompany maintenance chemotherapy. Notably, EOC tumors often become platinum-resistant, restricting further treatment options. FRα is found in over 80% of ovarian carcinomas and is associated with poorly-differentiated, aggressive tumors. Mirvetuximab soravtansine is a first-in-class FRα-targeting ADC. While it appears to be better tolerated than chemotherapy, mirvetuximab soravtansine demonstrated mixed results in treating platinumresistant EOC compared to standard chemotherapy. If approved, it will provide another treatment for women with limited options.

FDA APPROVAL TIMELINE November 28, 2022  Fast Track

 Orphan Drug

 Priority Review

 seeking Accelerated Approval

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$10

$82

$166

$262

$353

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NEUROLOGY

omaveloxolone oral Reata/Abbvie PROPOSED INDICATIONS Friedreich’s ataxia (FA)

CLINICAL OVERVIEW

Omaveloxolone activates nuclear factor erythroid 2-related factor 2 (Nrf2), a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. Part 2 of the double-blind, placebo-controlled, parallel-group, phase 2 MOXIe study (NCT02255435) evaluated omaveloxolone’s impact on neurological function in 103 patients with genetically confirmed FA. Enrolled patients were 16 to 40 years of age with a baseline modified FA Rating Scale (mFARS) between 20 and 80 points. The mFARS, the study’s primary endpoint, is a measure of neurologic severity of FA (range, 0 to 99 points) with higher scores representing worse neurological function. Patients were randomized 1:1 to omaveloxolone or placebo. After 48 weeks, there was a significant mean change from baseline in mFARS with omaveloxolone (−1.55 points) compared to placebo (0.85 points) (difference, –2.4; p=0.014). Omaveloxolone improved each component of the mFARS (bulbar, upper limb coordination, lower limb coordination, and upright stability) compared to placebo. Omaveloxolone was generally well tolerated. The most common TEAEs were headache, nausea, asymptomatic transient increases in ALT and AST, fatigue, diarrhea, and abdominal pain. The omaveloxolone dosage in part 2 of the MOXIe study was 150 mg orally once daily.

PLACE IN THERAPY

FA is a rare inherited disorder caused by a mutation in the FXN gene, leading to oxidative stress and a deficiency of the frataxin protein in the mitochondria. Progressive degeneration of spinal cord and peripheral nerves occurs, resulting in unsteady movements and impaired sensory functions. FA affects approximately 1 in 50,000 Americans. Patients with FA inherit 1 defective FXN gene from each parent. While pharmacologic and non-pharmacologic treatments exist for FA complications (e.g., diabetes, cardiac dysfunction, foot deformities, scoliosis, swallowing and speech dysfunction, hearing impairment), there is no cure or effective treatment for the neurologic effects of FA. In the MOXIe trial, omaveloxolone was well tolerated and demonstrated significant improvement in neurologic function compared to worsening neurologic function observed with placebo. If approved, omaveloxolone will be the first medication for the treatment of FA. Oral vatiquinone, by PTC Therapeutics, is also in phase 3 trials for FA. Like omaveloxolone, vatiquinone reduces oxidative stress and neuroinflammation.

FDA APPROVAL TIMELINE November 30, 2022  Fast Track

 Orphan Drug

 Priority Review

 Rare Pediatric Disease

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$44

$101

$186

$267

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CARDIOLOGY

omecamtiv mecarbil oral Cytokinetics PROPOSED INDICATIONS

Treatment of heart failure with reduced ejection fraction (HFrEF)

CLINICAL OVERVIEW

Omecamtiv mecarbil is a cardiac myosin activator. It increases the number of active actin-myosin cross bridges during systole and ultimately enhances the impaired contractility that is associated with HFrEF. Omecamtiv mecarbil was evaluated in the double-blind, placebo-controlled, phase 3 GALACTIC-HF trial (NCT02929329) in 8,256 patients with symptomatic (NYHA functional class II to IV) HFrEF and an ejection fraction (EF) < 35% within the past 12 months. When added to SOC, omecamtiv mecarbil significantly reduced the risk of the primary composite endpoint of HF event or CV death compared to placebo (HR, 0.92; p=0.025) in the overall study population. When stratified by EF, omecamtiv mecarbil-treated patients with an EF ≤ 28% experienced a 16% relative reduction in the time to first HF event or CV death (HR, 0.84; p=0.0003), while there was no significant change among those with an EF > 28% (HR, 1.04; p=0.45). In the randomized, double-blind, phase 3 METORIC-HF trial (NCT03759392), 20 weeks of treatment with omecamtiv mecarbil did not significantly impact the peak oxygen update (pVO2) compared to placebo in 276 patients with NYHA functional class II to III HFrEF and an LVEF < 35% who were also receiving SOC therapy. No benefit was demonstrated on total workload during exercise, ventilatory efficiency, or daily physical activity. The starting dose of omecamtiv mecarbil was 25 mg orally twice daily. This was titrated based on drug plasma concentrations to a maximum of 37.5 mg twice daily.

PLACE IN THERAPY

HF is a progressive condition caused by a change in cardiac function that results in a failure of the heart to deliver an adequate supply of oxygenated blood to the tissues. It is considered to be HFrEF when the EF is ≤ 40%. Coronary artery disease (CAD) is the cause of HF in nearly 70% of cases. Incidence of HF in the US is estimated to be 6.2 million individuals. Risks of HF-related hospitalization and death are higher among Blacks than Caucasians. Typical symptoms include dyspnea, fatigue, and fluid retention. An angiotensin receptor neprilysin inhibitor (ARNI) is the treatment of choice to decrease mortality and morbidity in patients with HFrEF, with ACEIs or ARBs as alternative medications in patients with NYHA class II to IV HFrEF. Other agents that may also be used are beta blockers, mineralocorticoid receptor antagonists, SGLT2 inhibitors, diuretics, ivabradine (Corlanor®), vericiguat (Verquvo®), digoxin, and isosorbide dinitrate. Omecamtiv mecarbil improves impaired systolic function, a key defect of HFrEF. In clinical trials, omecamtiv mecarbil led to greater therapeutic benefit with lower baseline EF in patients with HFrEF. If approved, it will be the first cardiac myosin activator in the US to treat HF.

FDA APPROVAL TIMELINE February 28, 2023

The Cardiovascular and Renal Drugs Advisory Committee will review omecamtiv mecarbil on December 13, 2022.  Fast Track

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$31

$90

$149

$221

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RENAL

sparsentan oral Travere/Bristol-Myers Squibb PROPOSED INDICATIONS

Immunoglobulin A (IgA) nephropathy (IgAN)

CLINICAL OVERVIEW

Sparsentan is a dual endothelin and angiotensin receptor antagonist (DEARA) designed to reduce proteinuria, protect podocytes, and prevent glomerulosclerosis and mesangial cell proliferation. The double-blind, active-controlled, phase 3 PROTECT study (NCT03762850) evaluated sparsentan in 404 adults with biopsy confirmed IgAN and persistent proteinuria despite treatment with an ACEI or ARB. Patients were randomized to sparsentan or irbesartan. At the 36-week interim analysis, sparsentan led to a significant mean reduction in proteinuria compared to irbesartan (49.8% versus 15.1%, respectively; p<0.0001). Sparsentan was generally well tolerated. Final data of the 114-week trial is expected in mid-2026. Sparsentan was administered orally as 200 mg once daily for 2 weeks; if tolerated, it was increased to 400 mg once daily.

PLACE IN THERAPY

IgAN, also known as Berger’s disease, is an autoimmune disorder characterized by the accumulation of IgA in the kidneys. This causes inflammation and damage to the glomeruli of the kidneys and may eventually lead to ESRD. IgAN is one of the most common causes of glomerulonephritis. IgAN can occur at any age, but onset is typically seen between the teen years to late 30s. The incidence is 2-fold higher in men than women. It is also more common among Asians and Caucasians. IgAN is estimated to occur in about 60,000 Americans. Pharmacologic treatment of IgAN is focused on preventing or delaying ESRD with antihypertensive medications, such as ACEIs, ARBs, beta blockers, and calcium channel blockers. Corticosteroids, immunosuppressive agents, an SGLT2 inhibitor, and/or fish oil may be added in patients with persistent proteinuria (≥ 1 g/day) who are at increased risk for progression to ESRD. Kidney transplantation is the treatment of choice in patients who progress to kidney failure; however, recurrent IgA accumulation is common. Currently, delayed-release oral budesonide (Tarpeyo™) is the only medication FDA-approved to reduce proteinuria in adults with primary IgAN. If approved, sparsentan will be the first non-immunosuppressive option to treat IgAN. In the pivotal clinical trial, it led to a significantly greater improvement in proteinuria compared to ARB therapy. Several other agents with various mechanisms of action are in phase 3 trials for IgAN. They include atrasentan (endothelin A receptor antagonist), iptacopan (factor B inhibitor of the alternative complement pathway), narsoplimab (anti-mannan-associated lectin-binding serine protease−2 monoclonal antibody), and VIS649 (anti-A proliferation-inducing ligand monoclonal antibody). Sparsentan is also in phase 3 trials for focal segmental glomerulosclerosis.

FDA APPROVAL TIMELINE November 17, 2022  Orphan Drug

 Priority Review

 seeking Accelerated Approval

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$40

$150

$258

$351

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NEUROLOGY

zavegepant intranasal Biohaven/Bristol-Myers Squibb PROPOSED INDICATIONS

Acute treatment of migraine in adults

CLINICAL OVERVIEW

Zavegepant is a calcitonin gene-related peptide (CGRP) receptor antagonist. Zavegepant was evaluated in 2 randomized, double-blind, placebo-controlled, phase 3 trials (study 1 [NCT04571060], study 2 [NCT03872453]) in adults experiencing a moderate to severe migraine attack. Patients had a history of 2 to 8 migraine attacks per month. In study 1 (n=1,269 evaluable patients), zavegepant 10 mg was superior to placebo based on the co-primary endpoints of freedom from pain 2 hours after the dose (2hPF) (23.6% versus 14.9%, respectively; p=0.0001) and freedom from the most bothersome symptom (MBS) (e.g., photophobia, phonophobia) 2 hours after the dose (2hMBSF) (39.6% versus 31.1%, respectively; p=0.0012). In addition, significantly more patients experienced pain relief with zavegepant than with placebo at 15 minutes post dose (15.9% versus 8%, respectively; p<0.0001). After the dose, more patients were able to return to normal function with zavegepant than with placebo at 30 minutes (10.5% versus 6.1%, respectively; p=0.0059) and 2 hours (35.8% versus 25.6%, respectively; p=0.0001). In study 2 (n=1,673), zavegepant 10 mg and 20 mg doses were superior to placebo based on the proportion of patients who experienced 2hPF (22.5% [p=0.112] and 23.1% [p=0.0055], respectively, versus 15.5%) and 2hMBSF (41.9% [p=0.0155] and 42.5% [p=0.0094], respectively, versus 33.7%). The most common TEAEs were mild to moderate dysgeusia, nasal discomfort, and nausea. Zavegepant was administered as a single intranasal dose to treat 1 migraine attack.

PLACE IN THERAPY

Migraine affects over 37 million men, women, and children in the US. Three times more women than men experience migraines and the majority of migraine sufferers have a family history of the condition. Migraines are considered a leading cause of disability worldwide. Episodes can be debilitating with pain that lasts hours to days. Triptans are the SOC for the acute treatment of moderate to severe migraine episodes. Oral small molecule CGRP inhibitors are also approved for acute treatment of migraine in adults and include rimegepant (Nurtec® ODT) and ubrogepant (Ubrelvy®). Several CGRP inhibitors are indicated for migraine prevention, including oral atogepant (Qulipta™), rimegepant ODT, and the injectable monoclonal antibodies eptinezumab-jjmr (Vyepti®), erenumab (Aimovig®), fremanezumab (Ajovy®), and galcanezumab (Emgality®). If approved, zavegepant will be the first intranasal CGRP inhibitor formulation for the acute treatment of migraine attacks. It will provide another option for migraine relief. In clinical trials, onset of effect was seen as early as 15 minutes and lasted for up to 48 hours in some patients. Other treatment options that bypass the GI tract are intranasal triptans (Imitrex®, Onzetral® Xsail®, Tosymra®, Zomig®), ODT rimegepant (Nurtec ODT), ODT triptans (Maxalt MLT®, Zomig ZMT®), and injectable triptans (Imitrex, Zembrace® SymTouch®). An oral formulation of zavegepant is also in phase 3 trials for migraine prevention.

FDA APPROVAL TIMELINE January to March 2023

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$39

$129

$195

$242

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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. Moreover, an interchangeable biological product is a biosimilar that is expected to produce the same clinical result as the reference product in any given patient. Switching or alternating between the reference and interchangeable products should have been evaluated and should not negatively impact the safety and efficacy of therapy. Many controversies have surrounded 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 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 focuses on providing flexibility for the efficient development of biosimilars while maintaining high scientific standards. In July 2018, the FDA finalized its guidance on labeling biosimilars. The guidance pertains to prescribing information (PI) but does not contain specific recommendations on interchangeability in the labeling. The labeling guidance provides recommendations on how to include, identify, and differentiate the biosimilar and the reference product in various sections of the PI. The basic premise remains that the originator product’s safety and effectiveness can be relied upon for HCPs to make prescribing decisions; therefore, a biosimilar should include relevant data from the originator in its PI. In May 2019, the agency released its final guidance on interchangeability. Most states have enacted biosimilar substitution laws. An interchangeable product may be substituted for the originator at the pharmacy without the involvement of the prescriber. The Purple Book is an FDA database of licensed biological products that lists biosimilar and interchangeable products. The FDA has approved 2 biosimilars for interchangeability to their reference product: insulin glargine-yfgn (Semglee®) and adalimumab-adbm (Cyltezo®). Biosimilars can receive extrapolation to gain an indication without direct trials of the biosimilar for the eligible indication(s) of the reference products without requiring additional trials. Nevertheless, as each biosimilar comes to market, it will need to be considered individually. The FDA historically regulated insulins as small molecules. However, effective March 23, 2020, drugs such as insulin and growth hormone were deemed biologics and transitioned from the drug pathway to the biologic pathway. Their licensure as biologics allows these agents to be considered in the biosimilar space and promotes competition and access.

PLACE IN THERAPY

The patents of several biologic drugs are set to expire in the next few years, opening the US market for biosimilar entry; however, patent litigation has resulted in significant launch delays of FDA-approved biosimilars. In June 2017, the US Supreme Court issued 2 landmark rulings: (1) allowing a biosimilar manufacturer to provide launch notice of commercial marketing to the originator manufacturer before or after FDA approval of the biosimilar product and (2) eliminating any federal requirement for disclosure, also known as the “patent dance.” Some states, however, mandate disclosure. These decisions may bring biosimilars to the market sooner and potentially create price competition in the marketplace. In July 2018, the FDA unveiled its Biosimilar Action Plan (BAP), a series of 11 steps to encourage biosimilar market competition, some of which were previously announced or underway. The BAP contains 4 key strategies: (1) improve the biosimilar development and approval process; (2) maximize scientific and regulatory clarity for sponsors; (3) provide effective communications for patients, clinicians, and payers; and (4) reduce 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 36 biosimilars have received FDA approval. Of these, only 24 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zaxio® (filgrastim-sndz)

Novartis/Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer/Celltrion

April 2016

Erelzi® (etanercept-szzs)

Novartis/Sandoz

August 2016

Amjevita™ (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Samsung Bioepis/ Merck

May 2017

Cyltezo (adalimumab-adbm)

Boehringer Ingelheim

August 2017

Mvasi® (bevacizumab-awwb)

Amgen

September 2017

Ixifi™ (infliximab-qbtx)†

Pfizer

December 2017

Ogivri® (trastuzumab-dkst)

Viatris

December 2017

Retacrit® (epoetin alfa-epbx)

Pfizer/Hospira

May 2018

Fulphila® (pegfilgrastim-jmdb)

Viatris

June 2018

Nivestym® (filgrastim-aafi)

Pfizer

July 2018

Hyrimoz™ (adalimumab-adaz)

Novartis/Sandoz

October 2018

Udenyca® (pegfilgrastim-cbqv)

Coherus

November 2018

Truxima® (rituximab-abbs)

Celltrion/Teva

November 2018

Herzuma® (trastuzumab-pkrb)

Celltrion/Teva

December 2018

Ontruzant® (trastuzumab-dttb)

Samsung Bioepis/ Merck

January 2019

Trazimera™ (trastuzumab-qyyp)

Pfizer

March 2019

Eticovo™ (etanercept-ykro)

Samsung Bioepis/ Merck

April 2019

Kanjinti™ (trastuzumab-anns)

Amgen

June 2019

Zirabev® (bevacizumab-bvzr)

Pfizer

June 2019

Hadlima™ (adalimumab-bwwd)

Samsung Bioepis/ Merck

July 2019

Ruxience® (rituximab-pvvr)

Pfizer

July 2019

Abrilada™ (adalimumab-afzb)

Pfizer

November 2019

Ziextenzo® (pegfilgrastim-bmez)

Novartis/Sandoz

November 2019

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Interchangeable -

Commercially Available

 

Originator (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)



Humira (Abbvie)



Neulasta (Amgen)

Rituxan (Genentech)

APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Avsola® (infliximab-axxq)

Amgen

December 2019

Nyvepria™ (pegfiltrastim-apgf)

Pfizer

June 2020

Semglee (insulin glargine-yfgn)

Viatris

July 2021

Hulio® (adalimumab-fkjp)

Viatris

July 2020

Riabni™ (rituximab-arrx)

Amgen

December 2020

Byooviz (ranibizumab-nuna)

Biogen/Samsung Bioepis

September 2021

Rezvoglar™ (insulin glargine-aglr)

Eli Lilly

December 2021

Yusimry™ (adalimumab-aqvh)

Coherus

December 2021

Releuko® (filgrastim-ayow)

Amneal

March 2022

Alymsys® (bevacizumab-maly)

Amneal

April 2022

Fylnetra® (pegfilgrastim-pbbk)

Amneal

May 2022

Interchangeable

Commercially Available



  

 

Originator (Manufacturer) Remicade (Janssen) Neulasta (Amgen) Lantus® (Sanofi-Aventis) Humira (Abbvie) Rituxan (Genentech) Lucentis® (Genentech)

Lantus (Sanofi)

Humira (Abbvie)

  -

Neupogen (Amgen) Avastin (Genentech) 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 follow-on to Sanofi’s Lantus, and Sanofi’s Admelog® insulin lispro approved as a follow-on to Eli Lilly’s Humalog®. Specialty medications, which include biologics, continue to grow and constitute a large part of drug spend. In the US, it is estimated that biosimilars will cost approximately 15% to 35% less than the originator product, although price dynamics vary. Further, the potential cost savings can vary based on the market segment where brand contracts can play a role. A host of factors will contribute to market acceptability and the potential success of biosimilars. Payers, pharmacies, prescribers, and patients each play an important role in market adoption of biosimilars. 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

IMMUNOLOGY

adalimumab SC Fresenius is seeking approval for its investigational biosimilar to Abbvie’s Humira 50 mg/mL; Alvotech, Celltrion, Samsung Bioepis/Organon, and Sandoz are seeking approval for their investigational biosimilars to Abbvie’s citratefree, high-concentration (100 mg/mL) Humira. Abbvie’s Humira is a tumor necrosis factor alpha (TNF-α) blocker indicated for the treatment of autoimmune disorders, including rheumatoid arthritis (RA), juvenile idiopathic arthritis (JIA), ankylosing spondylitis (AS), plaque psoriasis (PSO), psoriatic arthritis (PsA), Crohn’s disease (CD) in adults and children, ulcerative colitis (UC), hidradenitis suppurativa (HS), and non-infectious uveitis. Pfizer is seeking interchangeability of FDA-approved adalimumab-afzb (Abrilada) 50 mg/mL. Samsung Bioepis/ Organon and Sandoz are seeking approval for 100 mg/mL concentrations of FDA-approved adalimumab-bwwd (Hadlima) and adalimumab-adaz (Hyrimoz), respectively.

FDA APPROVAL TIMELINE

50 mg/mL • Fresenius (MSB11022) – October to December 2022 • Pfizer (Abrilada) – October to December 2022 for interchangeability 100 mg/mL • Alvotech (AVT-02) – December 2022 for initial approval and interchangeability • Celltrion (Yuflyma) – August 2022 • Samsung Bioepis/Organon (Hadlima) – August 2022 • Sandoz (Hyrimoz) – March to April 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$18,552

$10,899

$7,231

$5,527

$4,423

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

OPHTHALMOLOGY

aflibercept intravitreal Viatris/Janssen Viatris/Janssen is seeking approval of their investigational biosimilar (M710) to Regeneron’s Eylea®, a vascular endothelial growth factor (VEGF) inhibitor indicated for the treatment of neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), and diabetic retinopathy (DR).

FDA APPROVAL TIMELINE October 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$6,221

$6,303

$6,009

$5,338

$4,618

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

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BIOSIMILAR OVERVIEW continued

ONCOLOGY

bevacizumab IV Bio-Thera Solutions/Novartis, Celltrion, Centus/AstraZeneca, Samsung Bioepis/Organon, and Viatris/Biocon are seeking approval for their investigational biosimilars to Genentech’s Avastin, a vascular endothelial growth factor (VEGF)-specific angiogenesis inhibitor indicated for the treatment of metastatic colorectal cancer, nonsquamous non-small cell lung cancer, glioblastoma, metastatic renal cell carcinoma (RCC), and recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer.

FDA APPROVAL TIMELINE • • • • •

Bio-Thera Solutions/Novartis (BAT1706) – Pending Celltrion (CT-P16) – October 2022 Centus/AstraZeneca (FKB238) – Pending Samsung Bioepis/Organon (Aybintio) – Pending Viatris/Biocon (Bmab-100) – Pending

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$598

$477

$405

$362

$325

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

BLOOD MODIFIER

filgrastim IV, SC Apotex Apotex is seeking approval of their investigational biosimilar (Grastofil) to Amgen’s Neupogen, a leukocyte growth factor indicated for use in patients with nonmyeloid malignancies who are receiving myelosuppressive anti-cancer drugs; following induction or consolidation chemotherapy for acute myeloid leukemia (AML); with nonmyeloid malignancies in patients who are undergoing myeloablative chemotherapy followed by bone marrow transplantation; to mobilize autologous hematopoietic progenitor cells for collection by leukapheresis; with symptomatic congenital neutropenia‚ cyclic neutropenia‚ or idiopathic neutropenia; and in patients who are acutely exposed to myelosuppressive doses of radiation (hematopoietic syndrome of acute radiation syndrome [HSARS]).

FDA APPROVAL TIMELINE Pending

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$57

$50

$45

$40

$37

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

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BIOSIMILAR OVERVIEW continued

BLOOD MODIFIER

pegfilgrastim SC Apotex, Lupin, and Merck/Fresenius are seeking approval for their 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 • Lupin (Lupifil-P) – Pending • Merck/Fresenius (MSB-11455) – Pending

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$863

$643

$553

$486

$442

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

IMMUNOLOGY

ranibizumab intravitreal Coherus Coherus is seeking approval for their investigational biosimilar (FYB201) to Genentech’s Lucentis, a vascular endothelial growth factor (VEGF) inhibitor indicated to treat wet age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy, and myopic choroidal neovascularization (mCNV).

FDA APPROVAL TIMELINE August 2, 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$1,235

$909

$668

$516

$431

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

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BIOSIMILAR OVERVIEW continued

ONCOLOGY

trastuzumab IV Novartis and Tanvex are seeking approval for their investigational biosimilars to Genentech’s Herceptin, a HER2/neu receptor antagonist indicated for the treatment of HER2-overexpressing breast cancer and HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma.

FDA APPROVAL TIMELINE Novartis December 20, 2022 Tanvex (TX05) August 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$418

$356

$315

$284

$258

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 2026, are displayed. The financials are projected total annual US sales, reported in millions. zuranolone

adagrasib

$678

$881

Behavioral health

tiragolumab Oncology

$785

Oncology

dextromethorphan/ bupropion Behavioral health

$812

tabelecleucel

donanemab

Oncology

Neurology

$316

$1,887

resmetirom

exa-cel (CTX001)

Cardiovascular

Hematology/Gene therapy

$548

$949

nirsevimab

ﬁdanacogene elaparvovec

Infectious disease

Hematology/Gene therapy

$411

$267

mirikizumab

gantenerumab

$687

$1,836

Neurology

Immunology

lenadogene nolparvovec (GS-010)

Ophthalmology/Gene therapy

$59

giroctocogene ﬁtelparvovec Gene therapy/Hematology

$205

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

Pipeline DRUG LIST The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2023. 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

PHASE3 3 IN PHASE TRIALS TRIALS

68%

32%

38%

33%

21%

Specialty

26%

13%

17%

10 %

Traditional

Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar

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

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

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

Submitted (New Drugs) ravulizumab-cwvz (Ultomiris® SC)

AstraZeneca

Hemolytic uremic syndrome (atypical); PNH

Submitted – BLA; Orphan Drug

July 2022

spesolimab

Boehringer Ingelheim

Generalized pustular psoriasis (GPP)

IV, SC

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

July 2022

bulevirtide

Gilead

Hepatitis D infection treatment (in patients with compensated liver disease)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

Jul–Nov 2022

infliximab SC

Celltrion

IBS

Submitted – BLA

Jul–Dec 2022

roflumilast cream

Arcutis/AstraZeneca

PSO (mild–severe)

Topical

Submitted – NDA

07/29/2022

adalimumab 100 mg/mL (biosimilar to Abbvie’s Humira)

Celltrion

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

Submitted – BLA

August 2022

narsoplimab

Omeros

HSCT-associated thrombotic microangiopathy

IV, SC

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

August 2022

trastuzumab (biosimilar to Genentech’s Herceptin)

Tanvex

Breast cancer; Gastric/ gastroesophageal cancer

Submitted – BLA

August 2022

teclistamab

Janssen

Multiple myeloma (R/R)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

Aug–Dec 2022

ranibizumab (biosimilar to Genentech’s Lucentis)

Coherus

Wet AMD; Macular edema following RVO; Myopic choroidal neovascularization (mCNV)

Intravitreal

Submitted – BLA

08/02/2022

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

β-thalassemia (transfusion-dependent)

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

08/19/2022

miglustat

Amicus

Pompe disease (in combination with cipaglucosidase alfa)

Oral

Submitted – NDA

08/29/2022

microbiota suspension

Ferring

Clostridioides difficile infection (recurrent)

Rectal

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

Sep–Nov 2022

deucravacitinib

Bristol-Myers Squibb

PSO (moderate–severe)

Oral

Submitted – NDA

09/09/2022

eflapegrastim

Spectrum

Chemotherapy inducedneutropenia

Submitted – BLA

09/09/2022

linzagolix

Obseva

Uterine fibroid-related Oral heavy menstrual bleeding

Submitted – NDA

09/13/2022

dasatinib

Xspray

CML

Oral

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

09/16/2022

elivaldogene autotemcel (Lenti-D)

Bluebird Bio

Cerebral adrenoleukodystrophy (pediatrics)

Submitted – BLA; Breakthrough Therapy; Orphan Drug; Priority Review; Rare Pediatrics Disease

09/16/2022

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PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

sodium thiosulfate

Fennec

Chemotherapy-induced ototoxicity prevention

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

09/23/2022

taurolidine/citrate/heparin

Cormedix

Reduction of catheterrelated bloodstream infections (CRBSIs) related to chronic hemodialysis

Submitted – NDA; Fast Track; QIDP

09/28/2022

sodium phenylbutyrate/ tauroursodiol

Amylyx

ALS

Oral

Submitted – NDA; Orphan 09/29/2022 Drug; Priority Review

futibatinib

Otsuka

Cholangiocarcinoma (locally advanced or metastatic, prior treatment, FGFR2 gene rearrangements)

Oral

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

09/30/2022

aflibercept (biosimilar to Regeneron’s Eylea)

Viatris/Janssen

DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD

Intravitreal

Submitted – BLA

October 2022

bevacizumab (biosimilar to Genentech’s Avastin)

Celltrion

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

Submitted – BLA

October 2022

treosulfan

Medac

Allogenic-HSCT conditioning

Submitted – NDA; Orphan October 2022 Drug

adalimumab 50 mg/mL (biosimilar to Abbvie’s Humira)

Fresenius

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

Submitted – BLA

Oct–Dec 2022

tremelimumab

AstraZeneca

HCC (as single priming dose for durvalumab)

Submitted – BLA; Orphan Drug; Priority Review

Oct–Dec 2022

olipudase alfa

Sanofi

Niemann-Pick disease (acid sphingomyelinase deficiency)

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

10/03/2022

apomorphine infusion pump

Supernus

Parkinson’s disease

Submitted – NDA

10/07/2022

furosemide

scPharmaceuticals

Decompensated heart failure

Submitted – 505(b)(2) NDA

10/08/2022

cipaglucosidase alfa

Amicus

Pompe disease (in combination with oral miglustat)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

10/29/2022

etranacogene dezaparvovec

CSL Behring

Hemophilia B

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

November 2022

omidenepag isopropyl

Santen

Glaucoma/ocular hypertension

Ophthalmic

Submitted – NDA

11/04/2022

sparsentan

Travere/BristolMyers Squibb

Immunoglobulin A nephropathy (Berger’s disease)

Oral

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

11/17/2022

teplizumab

Provention Bio

T1DM (delay/prevention)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

11/17/2022

23 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

poziotinib

Spectrum

NSCLC (locally advanced/ metastatic; HER2 exon 20 insertion mutations)

Oral

Submitted – NDA; Fast Track

mirvetuximab soravtansine

Immunogen

Ovarian cancer (folate receptor alpha [FRα]high platinum-resistant, 1-3 prior systemic treatments)

Submitted – BLA; seeking 11/28/2022 Accelerated Approval; Fast Track; Orphan Drug; Priority Review

omaveloxolone

Reata/Abbvie

Friedreich’s ataxia

Oral

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

11/30/2022

omburtamab

Y-mAbs

Brain cancer (CNS/ leptomeningeal metastasis from neuroblastoma)

Intracerebroventricular

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

11/30/2022

adalimumab 100 mg/mL (biosimilar to Abbvie’s Humira)

Alvotech

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

Submitted – BLA; seeking biosimilar interchangeability

December 2022

terlipressin

Mallinckrodt

Hepatorenal syndrome

Submitted – NDA; Fast Track; Orphan Drug

December 2022

adagrasib

Mirati

NSCLC (KRAS G12C mutation, ≥ 2nd-line)

Oral

Submitted – NDA; seeking Accelerated Approval; Breakthrough Therapy; RTOR

12/14/2022

trastuzumab (biosimilar to Genentech’s Herceptin)

Novartis

Breast cancer; Gastric/ gastroesophageal cancer

Submitted – BLA

12/20/2022

toripalimab

Coherus

Nasopharyngeal cancer (advanced recurrent/ metastatic, 1st-line with chemotherapy, monotherapy for ≥ 2ndline)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

12/23/2022

ublituximab

TG Therapeutics

MS (relapsing)

Submitted – BLA

12/28/2022

mosunetuzumab

Genentech

Follicular lymphoma (3rdline)

IV, SC

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

12/29/2022

palovarotene

Ipsen

Fibrodysplasia ossificans progressiva

Oral

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

12/29/2022

sodium phenylbutyrate

Acer

Urea cycle disorders

Oral

Submitted – 505(b)(2) NDA

January 2023

budesonide/albuterol

AstraZeneca

Asthma

Inhaled

Submitted – NDA

Jan–Feb 2023

zavegepant

Biohaven/BristolMyers Squibb

Migraine treatment

Intranasal

Submitted – NDA

Jan–Mar 2023

elacestrant

Menarini

Breast cancer (ER+/HER2-, Oral advanced or metastatic)

Submitted – NDA; Fast Track

Jan–Jun 2023

momelotinib

Sierra Oncology/ Gilead

Myelofibrosis

Submitted – NDA; Fast Track; Orphan Drug

Jan–Jun 2023

24 | MAGELLANRX.COM

Oral

11/24/2022

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

lecanemab

Eisai/Biogen

Alzheimer’s disease (early)

Submitted – BLA; seeking 01/06/2023 Accelerated Approval; Breakthrough Therapy; Fast Track; Priority Review

daprodustat

GlaxoSmithKline

Anemia of CKD (dialysisdependent and -independent)

Oral

Submitted – NDA

02/01/2023

omecamtiv mecarbil

Cytokinetics

Heart failure with reduced ejection fraction (HFrEF)

Oral

Submitted – NDA; Fast Track

02/28/2023

tixagevimab/cilgavimab (Evusheld™)

AstraZeneca

COVID-19 treatment

IM, IV

Submitted – BLA

March 2023

trofinetide

Acadia

Rett syndrome (ages ≥ 2 years)

Oral

Submitted – NDA; Fast Track; Orphan Drug

3/17/2023

anthrax vaccine, adsorbed

Emergent

Anthrax infection (postexposure prophylaxis)

Submitted – BLA; Fast Track

April 2023

mirikizumab

Eli Lilly

IV, SC

Submitted – BLA

04/28/2023

(vic-)trastuzumab duocarmazine

Byondis

Breast cancer (HER2+, unresectable locally advanced or metastatic)

Submitted – BLA; Fast Track

05/12/2023

foscarbidopa/foslevodopa

Abbvie

Parkinson’s disease motor SC fluctuations

Submitted – NDA

05/20/2023

nogapendekin alfa inbakicept

Immunitybio

Bladder cancer (BCG-unresponsive, non-muscle invasive carcinoma in situ, in combination with BCG)

Intravesical

Submitted – BLA; Breakthrough Therapy; Fast Track

05/23/2023

sotagliflozin

Lexicon

Heart failure in patients with T2DM

Oral

Submitted – NDA

05/31/2023

efanesoctocog alfa

Sanofi

Hemophilia A

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

June 2023

landiolol

Eagle

Supraventricular tachycardia (short-term reduction of ventricular rate)

Submitted – NDA

06/01/2023

beremagene geperpavec

Krystal

Epidermolysis bullosa

Topical

Submitted – BLA; Fast 06/23/2023 Track; Orphan Drug; RMAT

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Submitted – NDA

06/23/2023

nirmatrelvir/ritonavir (Paxlovid)

Pfizer

COVID-19 treatment (high-risk patients)

Oral

Submitted – NDA

06/30/2023

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera Solutions/ Novartis

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Centus/AstraZeneca

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Samsung Bioepis/ Organon

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Viatris/Biocon

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

Submitted – BLA

Pending

25 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

casirivimab/imdevimab (Regen-Cov)

Regeneron

COVID-19 treatment (non-hospitalized patients); COVID-19 postexposure prophylaxis

IM, IV, SC

Submitted – BLA; Priority Review

Pending

dextromethorphan/ bupropion

Axsome

MDD

Oral

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

Pending

diazepam buccal film

Aquestive

Seizure clusters

Oral transmucosal

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

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

lenacapavir

Gilead

HIV-1 infection (heavily treatment-experienced)

Oral, SC

Submitted – NDA; Breakthrough Therapy

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Lupin

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Merck/Fresenius

Neutropenia/leukopenia

Submitted – BLA

Pending

sabizabulin

Veru

COVID-19 treatment

Oral

Submitted – EUA; Fast Track

Pending

sodium oxybate (oncenightly)

Avadel

Narcolepsy-related excessive daytime sleepiness and cataplexy

Oral

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

Pending

tislelizumab

Beigene/Novartis

Esophageal squamous cell carcinoma (unresectable, recurrent, locally advanced/ metastatic, after prior systemic therapy)

Submitted – BLA; Orphan Drug

Pending

Submitted (Supplementals) durvalumab (Imfinzi )

AstraZeneca

Biliary tract cancer (in combination with chemotherapy)

Submitted – sBLA; Orphan Drug; Priority Review

Jul–Sep 2022

fam-trastuzumab deruxtecan-nxki (Enhertu®)

Daiichi Sankyo/ AstraZeneca

NSCLC (unresectable or metastatic, HER2+, ≥ 2nd-line)

Submitted – sBLA; Breakthrough Therapy; Priority Review

Jul–Sep 2022

tocilizumab (Actemra®)

Genentech

COVID-19 treatment (hospitalized adults on systemic corticosteroids and require assisted ventilation)

Submitted – sBLA; Priority Review

Jul–Dec 2022

adalimumab-bwwd 100 mg/mL (Hadlima) (biosimilar to Abbvie’s Humira)

Samsung Bioepis/ Organon

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

Submitted – sBLA

August 2022

pimavanserin (Nuplazid®)

Acadia

Alzheimer’s diseaseOral related hallucinations and delusions

Submitted – sNDA; Breakthrough Therapy

08/04/2022

relugolix/estradiol/ norethindrone (Myfembree®)

Myovant

Endometriosis

Oral

Submitted – sNDA

08/06/2022

ustekinumab (Stelara®)

Janssen

PsA (ages ≥ 5 years)

Submitted – sBLA

08/08/2022

26 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

aprepitant (Cinvanti®)

Heron

Postoperative nausea and vomiting prophylaxis

Submitted – 505(b)(2) sNDA

09/18/2022

dupilumab (Dupixent®)

Sanofi/Regeneron

Prurigo nodularis

Submitted – sBLA; Priority Review

09/30/2022

adalimumab-afzb 50 mg/mL (Abrilada) (biosimilar to Abbvie’s Humira)

Pfizer

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

Submitted – PAS BLA for biosimilar interchangeability

Oct–Dec 2022

ibalizumab-uiyk (Trogarzo®) Theratechnologies IV push

HIV-1 treatment

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

10/03/2022

lumasiran (Oxlumo®)

Alnylam

Primary hyperoxaluria type 1 (advanced)

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

10/06/2022

darolutamide (Nubeqa®)

Bayer

Prostate cancer (metastatic, hormonesensitive, in combination with docetaxel)

Oral

Submitted – sNDA; Fast Track; Priority Review

11/03/2022

upadacitinib (Rinvoq™)

Abbvie

Axial spondyloarthritis (non-radiographic)

Oral

Submitted – sNDA

11/07/2022

pegcetacoplan (Empaveli®)

Apellis

Geographic atrophy (secondary to AMD)

Intravitreal

Submitted – sNDA; Fast Track; Priority Review

11/26/2022

asparaginase erwina Jazz chysanthemi (recombinant)rywn (Rylaze™ IM)

AML and Lymphoblastic lymphoma (3 times a week IM dosing, with chemotherapy, ages ≥ 1 month, E. coliderived asparaginase hypersensitivity)

Submitted – sBLA; Fast Track; Orphan Drug; RTOR

12/02/2022

cariprazine (Vraylar®)

Abbvie

MDD (adjunct)

Oral

Submitted – sNDA

12/22/2022

abaloparatide (Tymlos®)

Radius

Osteoporosis (men, highrisk for fracture)

Submitted – sNDA

12/23/2022

ibrutinib (Imbruvica®)

Abbvie/Janssen

GVHD treatment (ages ≥ 1 years, ≥ 2nd-line)

Oral

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

12/28/2022

lumacaftor/ivacaftor (Orkambi®)

Vertex

Cystic fibrosis (ages 12 to 23 months)

Oral

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

January 2023

vonoprazan fumarate (Takecab®)

Phathom

Erosive esophagitis; Heartburn relief

Oral

Submitted – sNDA

01/11/2023

zanubrutinib (Brukinsa®)

Beigene

CLL/SLL

Oral

Submitted – sNDA; Orphan Drug

01/20/2023

pembrolizumab (Keytruda®)

Merck

NSCLC (post surgical resection; stage 1B, II, or IIIA)

Submitted – sBLA

01/29/2023

relugolix/estradiol/ norethindrone (Myfembree)

Myovant

Uterine fibroids (premenopausal women)

Oral

Submitted – sNDA

01/29/2023

asparaginase erwina Jazz chysanthemi (recombinant)rywn (Rylaze IV)

ALL

Submitted – sBLA; Fast Track; Orphan Drug; RTOR

February 2023

aflibercept (Eylea)

Diabetic retinopathy (16-week maintenance regimen)

Intravitreal

Submitted – sBLA

2/28/2023

27 | MAGELLANRX.COM

Regeneron

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

amifampridine phosphate (Firdapse®)

Catalyst

Lambert-Eaton myasthenic syndrome (pediatric)

Oral

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

March 2023

adalimumab-adaz 100 mg/ mL (Hyrimoz) (biosimilar to Abbvie’s Humira)

Sandoz

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

Submitted – sBLA

March–April 2023

ibrexafungerp (Brexafemme®)

Scynexis

Recurrent vulvovaginal candidiasis prevention

Oral

Submitted – sNDA; Fast Track; Orphan Drug; QIDP

April 2023

durvalumab (Imfinzi)

AstraZeneca

HCC (unresectable)

Submitted – sBLA; Orphan Drug

04/30/2023

baricitinib (Olumiant®)

Eli Lilly

Atopic dermatitis (moderate–severe)

Oral

Submitted – sNDA

Pending

norgestrel (Opill®)

Perrigo

Contraception

Oral

Submitted – Rx-to-OTC

Pending

Phase 3 (New Drugs) AAV-RPGR gene therapy

Janssen

Retinitis pigmentosa (X-linked)

Intraoccular

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

abaloparatide-TD

Radius

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

abelacimab

Anthos

Venous thromboembolism

IV, SC

Phase 3 – BLA; Fast Track

TBD

acoramidis

Bridgebio/ AstraZeneca

Transthyretin amyloid cardiomyopathy (ATTRCM)

Oral

Phase 3 – NDA

TBD

adintrevimab

Adagio

COVID-19

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Alvotech

DME; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Amgen

DME; Wet-AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Sam Chun Dang

DME; Wet-AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Samsung Bioepis/ Biogen

DME; Wet-AMD

Intravitreal

Phase 3 – BLA

TBD

aflibercept (biosimilar to Regeneron’s Eylea)

Santo/Formycon

DME; Wet-AMD

Intravitreal

Phase 3 – BLA

TBD

aloradine

Vistagen

Social anxiety disorder

Intranasal

Phase 3 – NDA; Fast Track

TBD

amcenestrant

Sanofi

Breast cancer

Oral

Phase 3 – NDA

TBD

amubarvimab + romlusevimab

Brii

COVID-19

Phase 3 – BLA

TBD

anti-BDCA2 antibody (BIIB059)

Biogen

SLE

Phase 3 – BLA

TBD

anti-betv1 monoclonal antibodies (REGN-57135714-5715)

Regeneron

Birch allergy

Phase 3 – BLA

TBD

apolipoprotein A-I (human)

CSL

Atherosclerosis

Phase 3 – BLA

TBD

arfolitixorin hemisulfate

Isofol

CRC

Phase 3 – NDA; Fast Track

TBD

arimoclomol

Orphazyme

Niemann-Pick disease

Oral

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

TBD

ataluren

PTC

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

28 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells

CRISPR Therapeutics

SCD; Thalassemia

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

TBD

balstilimab

Agenus

Cervical cancer (R/R, in combination with zalifrelimab)

Phase 3 – BLA; Fast Track

TBD

bamlanivimab

Eli Lilly

COVID-19

Phase 3 – BLA

TBD

bentracimab

Phasebio/ AstraZeneca

Ticagrelor (Brilinta ) reversal

Phase 3 – BLA; Breakthrough Therapy

TBD

beroctocog alfa

Green Cross

Hemophilia A

Phase 3 – BLA

TBD

betibeglogene autotemcel (Zynteglo)

Bluebird Bio

SCD

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

TBD

bevacizumab-vikg

Outlook

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

bimekizumab

UCB

Axial spondyloarthritis; Hidradenitis suppurativa; PsA

Phase 3 – BLA

TBD

bis-choline tetrathiomolybdate

AstraZeneca

Wilson’s disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

BPR277

Lifemax/Novartis

Congenital ichthyosis

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

brensocatib

Insmed/AstraZeneca

Bronchiectasis

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

cannabidiol gel, transdermal

Zynerba

Fragile X syndrome

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

capsaicin

Centrexion

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – NDA; Fast Track

TBD

ceftobiprole medocaril

Basilea

ABSSSI; Bacteremia; CAP; HAP

Phase 3 – NDA; Fast Track; QIDP

TBD

ceftriaxone wearable micropump

scPharmaceuticals

Gram+/Gram- infection

Phase 3 – NDA

TBD

CM-AT (pancreatic enzyme)

Curemark

Autism spectrum disorders

Oral

Phase 3 – BLA; Fast Track

TBD

cobitolimod

Index/Merck

Rectal

Phase 3 – NDA; Orphan Drug

TBD

concizumab

Novo Nordisk

Hemophilia A and B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

copper histidine

Zydus Cadila

Menkes disease

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

TBD

cosibelimab

Fortress

Cutaneous squamous cell carcinoma (metastatic)

Phase 3 – BLA

TBD

COVID-19 vaccine (C19VAZ; formerly AZD1222; ChAdOx1)

AstraZeneca

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (JNJ78436735; formerly Ad26. COV2-S)

Janssen

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (MT2766)

Mitsubishi Tanabe/ GlaxoSmithKline

COVID-19

IM, SC

Phase 3 – BLA

TBD

COVID-19 vaccine (SCB2019)

Clover

COVID-19

Phase 3 – BLA

TBD

29 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

COVID-19 vaccine (SP0253)

Sanofi/ GlaxoSmithKline

COVID-19

Phase 3 – BLA

TBD

crovalimab

Genentech

Hemolytic uremic syndrome; PNH

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

dabocemagene autoficel

Castle Creek

Epidermolysis bullosa

Intradermal

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

TBD

darvadstrocel

Takeda

Phase 3 – BLA; Orphan Drug

TBD

delandistrogene moxeparvovec

Sarepta/Genentech

DMD

Phase 3 – BLA; Fast Track

TBD

dengue tetravalent vaccine

Takeda

Dengue fever

Phase 3 – BLA; Fast Track

TBD

denosumab (Biosimilar to Amgen’s Prolia®)

Biocon

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia)

Celltrion

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia)

Fresenius

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia)

Gedeon Richter

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia)

Novartis

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

denosumab (Biosimilar to Amgen’s Prolia)

Teva

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

dersimelagon

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

despropyl macitentan

Janssen

Hypertension (resistant)

Oral

Phase 3 – NDA

TBD

difluprednate XR

Sun

Ocular pain/inflammation

Ophthalmic

Phase 3 – NDA

TBD

donanemab

Eli Lilly

Alzheimer’s disease (early)

IV, SC

Phase 3 – BLA; Breakthrough Therapy

TBD

donaperminogene seltoplasmid

Helixmith

Diabetic foot ulcers (chronic non-healing)

Phase 3 – BLA

TBD

doravirine/islatravir

Merck

HIV-1 infection treatment

Oral

Phase 3 – NDA

TBD

dovitinib lactate

Allarity

Breast cancer; RCC (3rdline)

Oral

Phase 3 – NDA

TBD

durlobactam/sulbactam

Entasis

Acinetobacter baumannii infection

Phase 3 – NDA; Fast Track; QIDP

TBD

dust mite immunotherapy

Stallergenes Greer

Allergic rhinitis

Phase 3 – BLA

TBD

EB-101 (gene therapy)

Abeona

Epidermolysis bullosa

Surgical application

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

TBD

eculizumab (biosimilar to Alexion’s Soliris®)

Amgen

PNH

Phase 3 – BLA

TBD

elamipretide

Stealth

Barth syndrome

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

enmetazobactam

Allecra

UTI (complicated)

Phase 3 – NDA; Fast Track; QIDP

TBD

ensifentrine

Verona

COPD

Inhaled

Phase 3 – NDA

TBD

ensovibep

Novartis

COVID-19

Phase 3 – BLA; Fast Track

TBD

epcoritamab

Genmab

DLBCL

Phase 3 – BLA

TBD

epinephrine

Bryn

Anaphylaxis

Intranasal

Phase 3 – NDA; Fast Track

TBD

30 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

episalvan

Amryt

Epidermolysis bullosa

Topical

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

TBD

eplontersen

Ionis/AstraZeneca

Transthyretin amyloid polyneuropathy

Phase 3 – NDA; Orphan Drug

TBD

esreboxetine

Axsome/Pfizer

Fibromyalgia

Oral

Phase 3 – NDA

TBD

etanercept (biosimilar to Amgen’s Enbrel)

Lupin

RA; Polyarticular JIA; AS; PSO; PsA

Phase 3 – BLA

TBD

etavopivat

Forma

SCD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

etesevimab

Lonza/Eli Lilly

COVID-19

Phase 3 – BLA

TBD

etrasimod

Arena

Oral

Phase 3 – NDA; Orphan Drug

TBD

fasinumab

Regeneron

Osteoarthritis pain (knee)

Phase 3 – BLA

TBD

fexapotide triflutate

Nymox

BPH

Intraprostatic

Phase 3 – NDA

TBD

fidanacogene elaparvovec

Pfizer/Genentech

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

CD; UC

Oral

Phase 3 – NDA

TBD

firibastat

Quantum Genomics

Hypertension (systemic)

Oral

Phase 3 – NDA

TBD

firmacute eubacterial spores

Seres

C. difficile-associated diarrhea

Oral

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

fitusiran

Sanofi

Hemophilia A and B

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

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

Allergan

Female reproductive disorder

Phase 3 – BLA

TBD

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

Finox

Female reproductive disorder

Phase 3 – BLA

TBD

FVIII mimetic bi-specific antibody

Novo Nordisk

Hemophilia A

Phase 3 – BLA; Orphan Drug

TBD

gantenerumab

Genentech

Alzheimer’s disease (early)

Phase 3 – BLA; Breakthrough Therapy

TBD

garadacimab

CSL

HAE

Phase 3 – BLA; Orphan Drug

TBD

gavorestat

Applied Therapeutics Galactosemia

Oral

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

TBD

GBT601

Global Blood Therapeutics

SCD

Oral

Phase 3 – NDA

TBD

gepotidacin

GlaxoSmithKline

UTI (uncomplicated)

Oral

Phase 3 – NDA; QIDP

TBD

giredestrant

Genentech

Breast cancer

Oral

Phase 3 – NDA; Fast Track

TBD

giroctocogene fitelparvovec

Pfizer

Hemophilia A

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

TBD

glatiramer acetate depot

Viatris

Phase 3 – 505(b)(2) NDA

TBD

glofitamab

Genentech

DLBCL

Phase 3 – BLA

TBD

gold nanocrystal

Clene

ALS

Oral

Phase 3 – NDA; Orphan Drug

TBD

hypericin

Soligenix

Cutaneous T-cell lymphoma (CTCL)

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

idursulfase

Takeda/Sanofi

Mucopolysaccharidosis II (Hunter syndrome)

Intrathecal

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

inavolisib

Genentech

Breast cancer

Oral

Phase 3 – NDA

TBD

inclacumab

Global Blood Therapeutics

SCD

Phase 3 – BLA; Orphan Drug

TBD

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

Sanofi

T1DM; T2DM

Phase 3 – BLA

TBD

insulin glargine (biosimilar to Sanofi’s Lantus)

Gan & Lee

T1DM; T2DM

Phase 3 – BLA

TBD

insulin icodec (onceweekly)

Novo Nordisk

T2DM

Phase 3 – BLA

TBD

iodine-131 apamistamab

Actinium

AML

Phase 3 – BLA; Orphan Drug

TBD

ipatasertib

Genentech

Prostate cancer

Oral

Phase 3 – NDA

TBD

iptacopan

Novartis

Complement 3 (C3) glomerulopathy; Hemolytic uremic syndrome; Immunoglobulin A nephropathy (Berger’s disease); PNH

Oral

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

TBD

itepekimab

Regeneron/Sanofi

COPD

Phase 3 – BLA

TBD

Lactobacillus reuteri

Infant Bacterial Therapeutics

Necrotizing enterocolitis

Oral

Phase 3 – BLA; Orphan Drug

TBD

lanifibranor

Inventiva

NASH

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

lazertinib

Genosco

NSCLC

Oral

Phase 3 – NDA

TBD

lebrikizumab

Eli Lilly

Atopic dermatitis (moderate-severe)

Phase 3 – BLA; Fast Track

TBD

lenadogene nolparvovec (GS010)

Gensight

Leber’s hereditary optic neuropathy

Intravitreal

Phase 3 – BLA; Orphan Drug

TBD

leniolisib

Pharming/Novartis

Activated phosphatidylinositol3-kinase (PI3K)-delta syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

lenzilumab

Humanigen

COVID-19

Phase 3 – BLA

TBD

leriglitazone

Minoryx

Adrenoleukodystrophy

Oral

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

TBD

levodopa/carbidopa patch pump

Mitsubishi Tanabe

Parkinson’s disease

Phase 3 – 505(b)(2) NDA

TBD

ligelizumab

Novartis

Urticaria

Phase 3 – BLA; Breakthrough Therapy

TBD

linrodostat

Bristol-Myers Squibb

Bladder cancer

Oral

Phase 3 – NDA

TBD

lotilaner

Tarsus

Demodex blepharitis

Ophthalmic

Phase 3 – NDA

TBD

magrolimab

Gilead

Myelodysplastic syndrome

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

TBD

marstacimab

Pfizer

Hemophilia A and B

IV, SC

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

32 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

masitinib

AB Science

ALS; Alzheimer’s disease; Asthma (eosinophilic); Mastocytosis; MS

mavorixafor

melphalan

FDA DESISION

Phase 3 – NDA; Orphan Drug

TBD

Warts, Oral hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome

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

TBD

Delcath

Uveal melanoma (hepatic-dominant)

Percutaneous hepatic perfusion

Phase 3 – NDA

TBD

meningococcal vaccine

GlaxoSmithKline

Meningococcal immunization

Phase 3 – BLA

TBD

meningococcal vaccine

Pfizer

Meningococcal immunization

Phase 3 – BLA

TBD

metachromatic leukodystrophy gene therapy

Orchard

Metachromatic leukodystrophy

Phase 3 – BLA; Orphan Drug; RMAT

TBD

midomafetamine

Multidisciplinary Association for Psychedelic Studies

PTSD

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

minocycline

Journey

Rosacea

Oral

Phase 3 – 505(b)(2) NDA

TBD

minocycline/edetate/ethyl alcohol

Citius

Catheter-related bloodstream infection (CRBSI)

Phase 3 – NDA; Fast Track; QIDP

TBD

mirikizumab

Eli Lilly

IV, SC

Phase 3 – BLA

TBD

mitapivat

Agios

SCD; Thalassemia

Oral

Phase 3 – NDA; Orphan Drug

TBD

molnupiravir

Merck

COVID-19

Oral

Phase 3 – NDA

TBD

motixafortide

Biolinerx

Stem cell mobilization

Phase 3 – NDA; Orphan Drug

TBD

nabiximols

Jazz

MS-related spasticity

Oral transmucosal

Phase 3 – NDA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 – NDA; Fast Track

TBD

naloxone

Orexo

Opioid overdose

Intranasal

Phase 3 – 505(b)(2) NDA

TBD

naloxone hydrochloride dihydrate lotion

Elorac

Pruritus

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

narsoplimab

Omeros

Hemolytic uremic syndrome

IV, SC

Phase 3 – BLA; Fast Track

TBD

natalizumab (biosimilar to Biogen’s Tysabri®)

Novartis

Phase 3 – BLA

TBD

navitoclax

Abbvie/Genentech

Myelofibrosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

nedosiran

Novo Nordisk

Hyperoxaluria

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

TBD

nemolizumab

Galderma

Atopic dermatitis (moderate-severe); Pruritus

Phase 3 – BLA; Breakthrough Therapy

TBD

nipocalimab

Janssen

Autoimmune hemolytic anemia

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

33 | MAGELLANRX.COM

Oral

DEVELOPMENT STATUS

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

nirmatrelvir/ritonavir (Paxlovid)

Pfizer

COVID-19

Oral

Phase 3 – NDA

TBD

nirsevimab

AstraZeneca

RSV prevention

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

nomacopan

Akari

Bullous pemphigoid; Hemolytic uremic syndrome; HSCTassociated thrombotic microangiopathy; PNH

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

Novavax COVID-19 vaccine, adjuvanted

Novavax

COVID-19

Phase 3 – BLA

TBD

olezarsen

Akcea

Familial chylomicronemia syndrome

Phase 3 – NDA

TBD

oportuzumab monatox

Sesen

Bladder cancer (highrisk, BCG-unresponsive, nonmuscle invasive)

Intravesical

Phase 3 – BLA; Fast Track

TBD

OPT-302

Opthea

Wet AMD

Intravitreal

Phase 3 – BLA; Fast Track

TBD

OTL-103

Orchar

Wiskott-Aldrich syndrome IV

Phase 3 – BLA; Orphan Drug; RMAT

TBD

Oxalobacter formigenes

Oxthera

Hyperoxaluria

Oral

Phase 3 – BLA; Orphan Drug; Rare Pediatric Disease

TBD

padeliporfin

Steba

Bladder cancer

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

palopegteriparatide

Ascendis

Hypoparathyroidism

Phase 3 – BLA; Orphan Drug

TBD

pamrevlumab

Fibrogen

COVID-19; DMD; IV Idiopathic pulmonary fibrosis; Pancreatic cancer

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

TBD

pegadricase

Swedish Orphan Biovitrum

Gout

Phase 3 – BLA

TBD

pegzilarginase

Aeglea

Arginase 1 deficiency

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

TBD

perfluorohexyloctane

Bausch

DED

Ophthalmic

Phase 3 – NDA

TBD

pirtobrutinib

Eli Lilly

Mantle cell lymphoma

Oral

Phase 3 – NDA

TBD

plinabulin

Beyondspring

Chemotherapy-induced neutropenia prevention; NSCLC

Phase 3 – NDA; Breakthrough Therapy

TBD

plonmarlimab

I-Mab

COVID-19

Phase 3 – BLA

TBD

pollinex quattro grass

Allergy Therapeutics

Allergic rhinitis

Phase 3 – BLA

TBD

pollinex quattro ragweed

Allergy Therapeutics

Allergic rhinitis

Phase 3 – BLA

TBD

potassium citrate/ potassium bicarbonate

Advicenne

Renal tubular acidosis

Oral

Phase 3 – 505(b)(2) NDA

TBD

pozelimab

Regeneron

PNH; Chaple disease

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

pritelivir

Aicuris Anti-infective Cures

Herpes simplex virus treatment

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

prothrombin complex

Octapharma

Hemostasis

Phase 3 – BLA

TBD

proxalutamide

Kintor

COVID-19

Oral

Phase 3 – NDA

TBD

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

rabies monoclonal antibody cocktail

Sanofi

Rabies treatment

Phase 3 – BLA; Fast Track

TBD

ralinepag

United Therapeutics

PAH

Oral

Phase 3 – NDA; Orphan Drug

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Stada Arzneimittel/ Bausch

Wet AMD; Macular edema following RVO; Myopic choroidal neovascularization (mCNV)

Intravitreal

Phase 3 – BLA

TBD

rapamycin

Timber

Tuberous sclerosis complex-associated facial angiofibromas

Topical

Phase 3 – NDA

TBD

rapamycin (high-strength)

Palvella

Pachyonychia congenita

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

REGN1908-1909

Regeneron

Allergic rhinitis

Phase 3 – BLA

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

reproxalap

Aldeyra

Allergic conjunctivitis; DED

Ophthalmic

Phase 3 – NDA

TBD

resmetirom

Madrigal/Roche

NASH

Oral

Phase 3 – NDA; Fast Track

TBD

rezafungin

Cidara

Candidemia/invasive candidiasis

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

ridinilazole

Summit

C. difficile-associated diarrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

ritlecitinib

Pfizer

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

roflumilast cream

Arcutis/AstraZeneca

Atopic dermatitis (scalp)

Topical

Phase 3 – NDA

TBD

roluperidone

Minerva Neurosciences

Schizophrenia

Oral

Phase 3 – NDA

TBD

ropeginterferon alfa-2b

Pharmaessentia

Essential thrombocythemia

Phase 3 – BLA; Orphan Drug

TBD

roxadustat

AstraZeneca

Anemia due to cytotoxic chemotherapy

Oral

Phase 3 – NDA

TBD

rozanolixizumab

UCB

Myasthenia gravis

Phase 3 – BLA; Orphan Drug

TBD

RSV vaccine (GSK3844766A)

GlaxoSmithKline

RSV prevention

Phase 3 – BLA; Fast Track

TBD

RSV vaccine (JNJ64400141)

Janssen

RSV prevention

Phase 3 – BLA; Breakthrough Therapy

TBD

RSV vaccine (PF06928316)

Pfizer

RSV prevention

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

ruxolitinib (deuterated)

Concert

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

sabatolimab

Novartis

Myelodysplastic syndrome

Phase 3 – BLA; Fast Track

TBD

seasonal influenza nanoparticle vaccine

Novavax

Seasonal influenza prevention

Phase 3 – BLA; Fast Track

TBD

sebetralstat

Kalvista

HAE

Oral

Phase 3 – NDA; Fast Track

TBD

seladelpar

Cymabay/Janssen

Primary biliary cholangitis Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

35 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

seltorexant

Janssen

MDD

Oral

Phase 3 – NDA

TBD

sepofarsen

Proqr

Leber’s congenital amaurosis

Intravitreal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

sofpironium

Brickell

Axillary hyperhidrosis

Topical

Phase 3 – NDA

TBD

sotatercept

Merck/Bristol-Myers Squibb

PAH

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

sotrovimab

Vir

COVID-19

Phase 3 – BLA

TBD

sparsentan

Travere/BristolMyers Squibb

Focal segmental glomerulosclerosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

SPK-8011

Genentech

Hemophilia A

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

tabelecleucel

Atara

Epstein-Barr virus-associated post-transplant lymphoproliferative disease

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

TAK-755

Takeda

Thrombotic thrombocytopenic purpura (TTP)

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

tanfanercept

Hanall

DED

Ophthalmic

Phase 3 – BLA

TBD

tarcocimab tedromer

Kodiak

DME; Retinal vein occlusion-associated macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 – NDA

TBD

tiragolumab

Genentech

Esophageal cancer; NSCLC

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

tiratricol

Rare Thyroid Therapeutics

Resistance to thyroid hormone type beta (RTH-b)

Oral

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

TBD

tislelizumab

Beigene

Gastric cancer; HCC; NSCLC

Phase 3 – BLA; Orphan Drug

TBD

tixagevimab + cilgavimab (Evusheld)

AstraZeneca

COVID-19

Phase 3 – BLA

TBD

tocilizumab (biosimilar to Genentech’s Actemra)

Biogen

Phase 3 – BLA

TBD

tocilizumab (biosimilar to Genentech’s Actemra)

Fresenius/Merck

Phase 3 – BLA

TBD

tofersen

Biogen

ALS

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tominersen

Genentech

Huntington’s disease

Intrathecal

Phase 3 – NDA; Orphan Drug

TBD

tradipitant

Vanda/Eli Lilly

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

Oral

Phase 3 – NDA

TBD

travoprost implant

Glaukos

Glaucoma/ocular hypertension

Intraocular

Phase 3 – NDA

TBD

TT-173

Thrombotargets

Hemostasis

Topical

Phase 3 – BLA; Orphan Drug

TBD

tusamitamab ravtansine

Sanofi

NSCLC

Phase 3 – BLA

TBD

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

ustekinumab (biosimilar to Janssen’s Stelara)

Amgen

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Hikma

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Intas

PSO

IV, SC

Phase 3 – BLA

TBD

vadadustat

Akebia

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

Oral

Phase 3 – NDA

TBD

valoctocogene roxaparvovec

Biomarin

Hemophilia A

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

TBD

venglustat

Sanofi

Gaucher’s disease; GM2 gangliosidoses (TaySachs disease, Sandhoff disease, AB variant)

Oral

Phase 3 – NDA; Orphan Drug

TBD

verbrinacogene setparvovec

Freeline

Hemophilia B

Phase 3 – BLA; Orphan Drug; RMAT

TBD

VGX-3100 therapeutic vaccine

Inovio

Cervical dysplasia (human IM papillovirus-positive)

Phase 3 – BLA

TBD

visomitin

Mitotech

DED

Ophthalmic

Phase 3 – NDA

TBD

wilfactin

LFB

Von Willebrand disease

Phase 3 – BLA; Orphan Drug

TBD

zavegepant

Biohaven/BristolMyers Squibb

COVID-19; Migraine prevention

Intranasal

Phase 3 – NDA

TBD

zilucoplan

UCB

Myasthenia gravis

Phase 3 – NDA; Orphan Drug

TBD

zolbetuximab

Astellas

Gastric cancer

Phase 3 – BLA; Orphan Drug

TBD

zoliflodacin

Entasis

Gonorrhea

Oral

Phase 3 – NDA; Fast Track; QIDP

TBD

zuranolone

Sage

MDD; Post-partum depression

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

Phase 3 (Supplementals) adalimumab (Hulio; biosimilar to Abbvie’s Humira)

Viatris/Biocon

Hidradenitis suppurativa; Uveitis

Phase 3 – sBLA

TBD

Eli Lilly

JIA; Uveitis

Oral

Phase 3 – sNDA

TBD

benralizumab (Fasenra )

AstraZeneca

ANCA-associated vasculitis; Bronchiectasis; Bullous pemphigoid; Chronic rhinosinusitis with nasal polyps (CRSwNP); Esophagitis

Phase 3 – sBLA; Orphan Drug

TBD

brexpiprazole (Rexulti®)

Otsuka

PTSD

Oral

Phase 3 – sNDA

TBD

dupilumab (Dupixent)

Sanofi

Allergic fungal rhinosinusitis; Bullous pemphigoid; COPD; Urticaria

Phase 3 – sBLA; Orphan Drug

TBD

baricitinib (Olumiant) ®

37 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

efgartigimod (Vyvgart®)

Argenx

ITP; Myasthenia gravis; Pemphigus vulgaris

Phase 3 – sBLA; Orphan Drug

TBD

empagliflozin (Jardiance®)

Boehringer Ingelheim

Diabetic nephropathy

Oral

Phase 3 – sNDA

TBD

ferric carboxymaltose (Injectafer®)

Daiichi Sankyo

Anemia in heart failure

Phase 3 – sNDA

TBD

ferric derisomaltose (Monoferric®)

Pharmacosmos

Anemia in heart failure

Phase 3 – sNDA

TBD

fostamatinib (Tavalisse®)

Rigel

Autoimmune hemolytic anemia

Oral

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

guselkumab (Tremfya®)

Janssen

Phase 3 – sBLA

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 – sNDA

TBD

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

Octapharma

COVID-19

Phase 3 – sBLA

TBD

inebilizumab-cdon (Uplizna®)

Horizon

IgG4-related disease; Myasthenia gravis

Phase 3 – sBLA

TBD

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

Evofem

Chlamydia trachomatis infection; Neisseria gonorrhoeae infection

Intravaginal

Phase 3 – sNDA; Fast Track; QIDP

TBD

mepolizumab (Nucala®)

GlaxoSmithKline

COPD

IV, SC

Phase 3 – sBLA

TBD

meropenem/vaborbactam (Vabomere®)

Melinta

Bacteremia; HAP

Phase 3 – sNDA; QIDP

TBD

nitazoxanide (Alinia®)

Lupin

COVID-19; Influenza

Oral

Phase 3 – sNDA

TBD

obeticholic acid (Ocaliva )

Intercept

NASH

Oral

Phase 3 – sNDA; Breakthrough Therapy

TBD

odevixibat (Bylvay™)

Albireo

Alagille syndrome-related Oral cholestatic pruritus

Phase 3 – sNDA; Orphan Drug

TBD

omalizumab (Xolair®)

Genentech

Food allergies

Phase 3 – sBLA; Breakthrough Therapy

TBD

patisiran (Onpattro®)

Alnylam

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

Phase 3 – sNDA; Orphan Drug

TBD

pegylated liposomal irinotecan (Onivyde®)

Ipsen

SCLC

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

ravulizumab-cwvz (Ultomiris)

Alexion

Pachyonychia congenita

IV, SC

Phase 3 – sBLA

TBD

risankizumab-rzaa (Skyrizi®)

Abbvie

IV, SC

Phase 3 – sBLA

TBD

rituximab-arrx (biosimilar to Genentech’s Rituxan) (Riabni)

Amgen

Phase 3 – sBLA

TBD

rivaroxaban (Xarelto®)

Janssen

COVID-19

Oral

Phase 3 – sNDA

TBD

romiplostim (Nplate®)

Amgen

Chemotherapy-induced thrombocytopenia

Phase 3 – sBLA; Orphan Drug

TBD

secukinumab (Cosentyx®)

Novartis

Hidradenitis suppurativa

IV, SC

Phase 3 – sBLA

TBD

tapinarof (Vtama®)

Roivant

Atopic dermatitis

Topical

Phase 3 – sNDA

TBD

tezepelumab-ekko (Tezspire™)

AstraZeneca

Nasal polyposis

Phase 3 – sBLA

TBD

ticagrelor (Brilinta)

AstraZeneca

SCD

Oral

Phase 3 – sNDA

TBD

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PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DESISION

tirzepatide (Mounjaro™)

Eli Lilly

Obesity

Phase 3 – sNDA

TBD

upadacitinib (Rinvoq)

Abbvie

CD; Giant cell arteritis

Oral

Phase 3 – sNDA

TBD

Complete Response Letter (CRL) NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

DEVELOPMENT STATUS

FDA DECISION

acetaminophen/ibuprofen

Hyloris

Postsurgical pain

CRL

TBD

bimekizumab

UCB

PSO

IV, SC

CRL

TBD

cantharidin

Verrica

Molluscum contagiosum

Topical

CRL

TBD

meloxicam/rizatriptan

Axsome

Migraine treatment

Oral

CRL

TBD

setmelanotide (Imcivree®)

Rhythm

Alström (related obesity and hunger)

CRL

TBD

surufatinib

Hutchmed

Neuroendocrine tumors

Oral

CRL

TBD

tebipenem pivoxil

Spero

UTI (complicated)

Oral

CRL

TBD

toripalimab

Coherus

Nasopharyngeal cancer (recurrent or metastatic, 1st-line in combination with chemotherapy, 2ndline monotherapy)

CRL

TBD

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GLOSSARY 6MWT 6 Minute Walking Test

CF Cystic Fibrosis

ABSSSI Acute Bacterial Skin and Skin Structure Infection

CHF Congestive Heart Failure

ACEI Angiotensin-Converting Enzyme Inhibitor

CKD Chronic Kidney Disease

ACR20 American College of Rheumatology 20% Improvement ACR50 American College of Rheumatology 50% Improvement

CI Confidence Interval CLL Chronic Lymphocytic Leukemia CML Chronic Myeloid Leukemia CMS Centers for Medicare & Medicaid Services

ACR70 American College of Rheumatology 70% Improvement

CNS Central Nervous System

ADC Antibody-Drug Conjugate

COVID-19 Coronavirus Disease 2019

ADHD Attention Deficit Hyperactivity Disorder

CRC Colorectal Cancer

ADL Activities of Daily Living

CRL Complete Response Letter

AED Anti-Epileptic Drug

CRR Complete Response Rate

ALK Anaplastic Lymphoma Kinase

CSF Colony Stimulating Factor

ALL Acute Lymphoblastic Leukemia

CV Cardiovascular

ALS Amyotrophic Lateral Sclerosis

CVD Cardiovascular Disease

ALT Alanine Transaminase AMD Age-Related Macular Degeneration

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

AML Acute Myeloid Leukemia

DEA Drug Enforcement Administration

ANCA Antineutrophil Cytoplasmic Antibodies

DLBCL Diffuse Large B Cell Lymphoma

ANDA Abbreviated New Drug Application

DMARD Disease Modifying Antirheumatic Drug

ARB Angiotensin II Receptor Blocker

DMD Duchenne Muscular Dystrophy

ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor

DME Diabetic Macula Edema

ART Antiretroviral Therapy

DNA Deoxyribonucleic Acid

ARV Antiretroviral

DOR Duration of Response

AS Ankylosing Spondylitis

DPI Dry Powder for Inhalation

ASCVD Atherosclerotic Cardiovascular Disease

DPP-4 Dipeptidyl Peptidase 4

AST Aspartate Aminotransferase

DR Delayed-Release

BCVA Best Corrected Visual Acuity BLA Biologics License Application

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

BMI Body Mass Index

ECOG Eastern Cooperative Oncology Group

BSA Body Surface Area

EDSS Expanded Disability Status Scale

BsUFA Biosimilar User Fee Act

eGFR estimated Glomerular Filtration Rate

CABP Community Acquired Bacterial Pneumonia

EGFR Epidermal Growth Factor Receptor

CAP Community Acquired Pneumonia

ER Extended-Release

CAR T Chimeric Antigen Receptor T Cell

ESA Erythropoietin Stimulating Agent

CD Crohn's Disease

ESRD End-Stage Renal Disease

CDC Centers for Disease Control and Prevention

EUA Emergency Use Authorization

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COPD Chronic Obstructive Pulmonary Disease

DMT Disease Modifying Therapy

GLOSSARY continued FDA Food and Drug Administration

IRB Internal Review Board

FH Familial Hypercholesterolemia

ITP Immune Thrombocytopenic Purpura

FLT3 FMS-Like Tyrosine Kinase-3

ITT Intent-To-Treat

G-CSF Granulocyte Colony Stimulating Factor

IV Intravenous

GI Gastrointestinal

JIA Juvenile Idiopathic Arthritis

GIST Gastrointestinal Stromal Tumor

LDL Low-Density Lipoprotein

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

LDL-C Low-Density Lipoprotein Cholesterol

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

LVEF Left Ventricular Ejection Fraction

GVHD Graft Versus Host Disease

MACE Major Adverse Cardiovascular Events

H Half HAART Highly Active Antiretroviral Therapy HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HBV Hepatitis B Virus HCC Hepatocellular Carcinoma HCP Healthcare Professional HCV Hepatitis C Virus HER Human Epidermal Growth Factor Receptor HER2 Human Epidermal Growth Factor Receptor 2 HF Heart Failure HFA Hydrofluoroalkane HFpEF Heart Failure with preserved Ejection Fraction HIT Heparin Induced Thrombocytopenia HIV Human Immunodeficiency Virus HIV-1 Human Immunodeficiency Virus-1 HPV Human Papilloma Virus HR Hazard Ratio HSCT Hematopoietic Stem Cell Transplant HTN Hypertension IBS Irritable Bowel Syndrome IBS-C Irritable Bowel Syndrome, Constipation Predominant IGA Investigator's Global Assessment IL-12 Interleukin-12 IL-17 Interleukin-17 IL-23 Interleukin-23 IM Intramuscular

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mAb Monoclonal Antibody MADRS Montgomery – Åsberg Depression Rating Scale MAOI Monoamine Oxidase Inhibitor MDD Major Depressive Disorder MDI Metered Dose Inhaler MDR Multi-Drug Resistant mITT modified Intent-To-Treat MRI Magnetic Resonance Imaging MRSA Methicillin-Resistant Staphylococcus Aureus MS Multiple Sclerosis N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NCCN National Comprehensive Cancer Network NCT National Clinical Trials 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 NYHA New York Heart Association ODT Orally Disintegrating Tablet OR Odds Ratio ORR Overall/Objective Response Rate OS Overall Survival OTC Over-the-Counter PAH Pulmonary Arterial Hypertension PARP Poly(ADP-ribose) Polymerase PAS Prior Approval Supplement PASI Psoriasis Area and Severity Index PASI 50 Psoriasis Area and Severity Index 50% Reduction PASI 75 Psoriasis Area and Severity Index 75% Reduction

GLOSSARY continued PASI 90 Psoriasis Area and Severity Index 90% Reduction

SCLC Small Cell Lung Cancer

PASI 100 Psoriasis Area and Severity Index 100% Reduction

SGLT2 Sodium-Glucose Co-Transporter 2

PCI Percutaneous Coronary Intervention PCSK9 Proprotein Convertase Subtilisin Kexin 9 PD-1 Programmed Death Protein 1 PD-L1 Programmed Death-Ligand 1 PDUFA Prescription Drug User Fee Application PFS Progression-Free Survival PGA Physician Global Assessment PHI Primary Humoral Immunodeficiency PNH Paroxysmal Nocturnal Hemoglobinuria PsA Psoriatic Arthritis PSO Plaque Psoriasis PTCA Percutaneous Transluminal Coronary Angioplasty PTSD Post-Traumatic Stress Disorder Q Quarter QIDP Qualified Infectious Diseases Product QOL Quality of Life R/R Relapsed or Refractory R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone

SCT Stem Cell Transplant SL Sublingual SLE Systemic Lupus Erythematosus SLL Small Lymphocytic Lymphoma sNDA supplemental New Drug Application SNRI Serotonin and Norepinephrine Reuptake Inhibitor SOC Standard of Care sPGA static Physician Global Assessment SR Sustained-Release SSRI Selective Serotonin Reuptake Inhibitor SSSI Skin and Skin Structure Infection T1DM Type 1 Diabetes Mellitus T2DM Type 2 Diabetes Mellitus TBD To Be Determined TEAE Treatment-Emergent Adverse Event TNBC Triple Negative Breast Cancer TNF Tumor Necrosis Factor TNFα Tumor Necrosis Factor-alpha UA Unstable Angina UC Ulcerative Colitis

RA Rheumatoid Arthritis

US United States

RBC Red Blood Cell

UTI Urinary Tract Infection

RCC Renal Cell Carcinoma

VAS Visual Analog Scale

REMS Risk Evaluation and Mitigation Strategy

VEGF Vascular Endothelial Growth Factor

RMAT Regenerative Medicine Advanced Therapy

VTE Venous Thromboembolism

RNA Ribonucleic Acid

WBC White Blood Cell

RRR Relative Risk Reduction

WHO World Health Organization

RSV Respiratory Syncytial Virus

XR Extended-Release

RTOR Real-Time Oncology Review RVO Retinal Vein Occlusion SARS-CoV-2 Severe Acute Respiratory SyndromeAssociated Coronavirus-2 sBLA supplemental Biologics License Application SC Subcutaneous SCCHN Squamous Cell Cancer of the Head and Neck SCD Sickle Cell Disease

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MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS

JANUARY 2022