MRx Pipeline - January 2022 by Prime/Magellan Rx

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

JANUARY JANUARY2022 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

Lara Frick, PharmD, BCPS, BCPP Drug Information Pharmacist

KEEP ON YOUR RADAR

PIPELINE DRUG LIST

GLOSSARY

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Michelle Booth, PharmD Director, Specialty Clinical Solutions

Robert Greer, RPh, BCOP Vice President, Clinical Strategy and Programs Katie Lockhart Manager, Forecasting and Pharmacoeconomics Brian MacDonald, PharmD Director, Specialty Clinical Strategy 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

Despite the ongoing pandemic and its unprecedented challenges, in 2021 the US FDA approved 50 novel drugs. Notably, over half (52%) of these approvals were for rare or Orphan conditions, and almost a third (28%) were approved through the Accelerated Approval pathway – which allows for earlier drug approval for serious conditions that fill an unmet need based on a surrogate endpoint. In 2021, the agency “authorized” 2 oral antivirals for the treatment of COVID-19 and “approved” a COVID-19 vaccine. With the “approval” of another COVID-19 vaccine in 2022, there are now 2 “FDA-approved” COVID-19 vaccines available. 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 toward the approval of specialty medications and drugs for rare diseases, with 64% and 31% of approvals expected, respectively, for agents with applications submitted to the FDA. New treatment modalities using gene therapy, therapeutic options for rare hereditary diseases, and the continued growth of biosimilars are expected. This will be another critical year in combatting COVID-19 with a public health arsenal of approved and authorized vaccines, including for pediatrics. Other noteworthy pipeline trends to watch include the development of complex therapies, oncology, immunology, neurology, and an emerging trend in immunodermatology. Moreover, sprouting products for behavioral health, metabolic conditions, and hematology 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

78%

19%

11%

BIOSIMILAR

ORPHAN DRUG

63%

11%

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

HEMATOLOGY/GENE THERAPY

betibeglogene autotemcel (beti-cel) IV Bluebird Bio PROPOSED INDICATIONS

Transfusion-dependent β-thalassemia

CLINICAL OVERVIEW

Beti-cel is a gene therapy that contains autologous CD34+ hematopoietic stem cells (HSC) and progenitor cells transduced with a BB305 lentiviral vector that encodes a β-globin (βA-T87Q) gene. The single-arm, phase 3 trials NorthStar-2 (n=23) and NorthStar-3 (n=18) evaluated beti-cel in a total of 41 patients ≤ 50 years of age with transfusion-dependent β-thalassemia (≥ 100 mL/kg/year of packed RBC in the prior 2 years). All patients received a single dose of beti-cel via IV infusion following busulfan myeloablative conditioning. A total of 32 patients (89%) achieved the primary endpoint of transfusion independence (defined as the absence of RBC transfusions and average Hb ≥ 9 g/dL for ≥ 12 months) for a median duration of 25 months (range, 12.5 to 38.5), with a median weighted Hb of 11.6 g/dL (range, 9.3 to 13.7). The response was demonstrated across genotype (including β 0/β 0, non-β 0/β 0) and age. No safety signals were identified; however, 1 patient experienced serious thrombocytopenia possibly due to beti-cel. A long-term follow-up study revealed ongoing transfusion independence in 78% (40/51) of patients after a median post-infusion follow-up of 44.2 months (range, 22.9 to 86.5).

PLACE IN THERAPY

β-thalassemia is an autosomal recessive disorder caused by a mutation in one or both hemoglobin beta (HBB) genes. Symptomatic cases are estimated to occur in approximately 1 in 100,000 individuals in the general population. β-thalassemia is reported most often in Mediterranean, African, and Southeast Asian populations, with prevalence reported as high as 10%. The condition is characterized by a reduction in Hb and RBC levels, resulting in anemia. Other complications of β-thalassemia include iron overload, skeletal changes, and functional changes in the heart, liver, gall bladder, and spleen. β-thalassemia is categorized into 3 types based on the number and type of mutations present. β-thalassemia types are: major (homozygous, severe, transfusion-dependent), intermedia (varied genotypes, moderate severity), and minor (heterozygous, mild, non-transfusion dependent). Other factors may impact the patient’s clinical manifestations, such as fetal Hb expression in RBCs, coinheritance of alpha thalassemia, and coexistence of sickle cell trait. Long-term RBC transfusions and iron chelation therapy are used to manage anemia and complications associated with β-thalassemia major. Splenectomy may decrease transfusion requirements in select patients. In addition, allogeneic HSCT may be curative in some cases but carries a risk of graft rejection and GVHD. The only FDA-approved pharmacologic treatment for anemia in transfusion-dependent adults with β-thalassemia is luspatercept-aamt (Reblozyl®), an erythroid maturation agent given by SC injection every 3 weeks by a HCP. In clinical trials, luspatercept-aamt significantly reduced the need for RBC transfusions. If approved, a one-time dose of betibeglogene autotemcel (beti-cel) gene therapy that targets the underlying cause of β-thalassemia may eliminate or significantly reduce the need for RBC transfusions in transfusion-dependent patients. It may prove particularly useful in patients who are not appropriate for an HSCT transplant.

FDA APPROVAL TIMELINE August 19, 2022

 Breakthrough Therapy

Fast Track

 Orphan Drug

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$22

$41

$63

$81

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METABOLIC/GENE THERAPY

elivaldogene autotemcel (eli-cel) IV Bluebird Bio PROPOSED INDICATIONS

Cerebral adrenoleukodystrophy (CALD) in pediatric patients

CLINICAL OVERVIEW

Eli-cel is a one-time gene therapy that consists of a patient's own hematopoietic stem cells that have been modified with the Lenti-D lentiviral vector (LVV) encoding ABCD1 complementary DNA, resulting in the production of functional human adrenoleukodystrophy protein (ALDP). The open-label, phase 2/3 STARBEAM trial (n=32) evaluated the safety and efficacy of eli-cel in males ≤ 17 years of age with MRI-confirmed CALD and without neurologic dysfunction. Patients with a human leukocyte antigen (HLA)-matched sibling were excluded. Patients underwent apheresis to collect CD34+ cells that were then modified with the LLV to produce eli-cel. Following myeloablative chemotherapy (busulfan and cyclophosphamide), patients received a single dose of eli-cel via IV infusion. Most patients also received G-CSF after the dose to accelerate engraftment. Twenty-four months after the eli-cel dose, 90.6% of patients achieved the primary endpoint of major functional disability (MFD)-free survival; 1 patient died due to disease progression, and 2 patients withdrew from the study at the investigators’ discretion. MFD-free survival was reported for up to nearly 7 years. Graft failure or rejection, GVHD, and transplant-related mortality have not been reported with eli-cel. TEAEs included myelodysplastic syndrome (MDS), viral cystitis, pancytopenia, and vomiting. On August 19, 2021, the clinical program was placed on a clinical hold after data suggested that MDS was due to the study drug. Patients continue to be closely monitored. Eli-cel was administered as a single IV infusion containing 6 to 19.4 million CD34+ cells/kg body weight, with a vector copy number of 0.5 to 2.5.

PLACE IN THERAPY

Adrenoleukodystrophy (ALD) is a devastating, rare, X-linked genetic disorder caused by mutations in the ABCD1 gene. It affects an estimated 1 in 17,000 to 21,000 males born in the US. The condition is characterized by a lack of the transporter protein ALDP, leading to a toxic build-up of very long-chain fatty acids in the brain, spinal cord, and adrenal cortex. Neurological symptoms, or CALD, may become apparent at any age, but in approximately 35% of boys, onset occurs between the ages of 3 and 10 years. CALD progresses to a vegetative state and death within 2 to 3 years of symptom onset. Allogeneic HSCT may stop the progression of adrenoleukodystrophy neurological symptoms in childhood, and is the current SOC in boys with evidence of CNS involvement (on MRI) but no neurological symptoms. Experimental Lorenzo’s oil, a nutritional therapy that contains erucic acid and oleic acid, has also shown benefit in asymptomatic patients. Eli-cel is a one-time gene therapy. If approved, it will be the first treatment to target the underlying genetic cause of CALD. It could provide an alternative to allogeneic HSCT in select patients with CALD, particularly those without a matched related HSCT donor. Notably, a review of the potential association of MDS with eli-cel is ongoing and could pose a challenge to eli-cel's FDA approval. Leriglitazone, an investigational peroxisome proliferator-activated receptor (PPAR)-gamma agonist, is in phase 3 trials in adult males with X-linked adrenoleukodystrophy (X-ALD) with spinal cord involvement.

FDA APPROVAL TIMELINE September 16, 2022

 Breakthrough Therapy

 Orphan Drug

 Priority Review

 Rare Pediatric Disease

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$15

$26

$35

$43

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INFECTIOUS DISEASE

lenacapavir oral/SC Gilead PROPOSED INDICATIONS

Multi-drug resistant (MDR) human immunodeficiency 1 (HIV-1) infection

CLINICAL OVERVIEW

Lenacapavir is a long-acting capsid inhibitor that binds directly to the HIV capsid, which is located within the HIV virion and protects HIV RNA. Lenacapavir interferes with capsid-mediated viral replication in both early and late stages of the HIV lifecycle. The safety and efficacy of lenacapavir were assessed in the placebo-controlled, phase 2/3 CAPELLA study in heavily treatment-experienced patients with MDR HIV-1 infection. At enrollment, patients demonstrated ongoing viremia (HIV-1 RNA ≥ 400 copies/mL) and resistance to ≥ 2 agents from 3 ARV classes. Patients were randomized to oral lenacapavir (n=24) or placebo (n=12) as an add-on to their failing ARV regimen (functional monotherapy period). On day 14, a significantly higher proportion of patients treated with lenacapavir achieved the primary endpoint of viral load reduction of ≥ 0.5 log10 copies/mL from baseline in HIV-1 RNA compared with those treated with placebo (88% versus 17%, respectively; p<0.0001). On day 15, all patients entered a maintenance phase with SC lenacapavir plus optimized background therapy (patients originally given placebo transitioned to oral lenacapavir followed by SC lenacapavir). At week 26, 81% of patients achieved an undetectable viral load (HIV-1 RNA < 50 copies/mL). The most common side effect reported with lenacapavir was injection site reaction. No serious TEAEs were identified. Lenacapavir was initiated as oral doses of 600 mg on days 1 and 2, 300 mg on day 8, followed by maintenance with SC lenacapavir 927 mg (as 2 injections) on day 15 and every 6 months for 52 weeks.

PLACE IN THERAPY

Of the estimated 1.2 million people with HIV in the US, about 10,000 have MDR HIV. While several antiviral agents are available to treat HIV-1 infection, treatment may fail due to the virus’ ability to mutate and become resistant to available ARV agents. Patient choices among ARVs may also be limited based on tolerability and potential drug-drug interactions. Proper resistance testing, viral load monitoring, and access to care and ARV therapy may decrease acquired drug resistance and stabilize the rate of global drug-resistant HIV transmission. The use of regimens with greater potency and genetic barriers, such as integrase strand transfer inhibitors (INSTIs), provide additional options for second- and third-line regimens. Currently, oral fostemsavir (Rukobia) and IV-administered ibalizumab-uiyk (Trogarzo®; administered every 2 weeks), are available in the US to treat patients with MDR HIV-1 infection. Lenacapavir is a first-in-class capsid inhibitor. In clinical trials, it was well-tolerated and produced significant reductions in viral load when used in combination with other ARV agents. If approved, lenacapavir will provide a long-acting SC option for heavily-treated patients with MDR HIV-1 infection with a convenient twice-yearly maintenance dosing schedule and potential for self-administration.

FDA APPROVAL TIMELINE February 28, 2022

On December 21, 2021, Gilead announced a clinical hold on all studies for injectable (SC) lenacapavir, including phase 3 trials for HIV-1 pre-exposure prophylaxis, due to vial quality concerns. Dosing for oral formulations continues. The impact on the FDA decision is unknown.  Breakthrough Therapy

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$107

$223

$340

$447

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IMMUNODERMATOLOGY

roflumilast cream topical Arcutis/AstraZeneca PROPOSED INDICATIONS

Mild to severe plaque psoriasis (PSO)

CLINICAL OVERVIEW

Roflumilast is a highly potent, selective inhibitor of phosphodiesterase type 4 (PDE4), an enzyme that mediates immune cell response via cyclic adenosine monophosphate (cAMP). Safety and efficacy of roflumilast cream was evaluated in 2 identical, 8-week, randomized, double-blind, vehicle-controlled, phase 3 trials, DERMIS-1 (n=439) and DERMIS-2 (n=442), in adult and pediatric patients ≥ 2 years of age with mild to severe chronic PSO. The total BSA involvement ranged from 2% to 20% at baseline. In both trials, topical roflumilast demonstrated a significantly higher IGA success rate compared to vehicle (DERMIS-1, 42.4% versus 6.1%, respectively [p<0.0001]; DERMIS-2, 37.5% versus 6.9%, respectively [p<0.0001]). Significant improvement with roflumilast over vehicle was also observed with secondary endpoints, such as intertriginous IGA, PASI-75, and itch reduction. Topical roflumilast was well-tolerated. The most common TEAEs reported with roflumilast were diarrhea, headache, insomnia, nausea, upper respiratory tract infection, and UTI (all ≤ 3%). In the open-label, phase 2b DERMIS-OLE trial, safety and durability of response with topical roflumilast were demonstrated for up to 64 weeks. Roflumilast 0.3% cream was applied topically to affected areas once daily.

FDA APPROVAL TIMELINE July 29, 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$66

$211

$328

$557

tapinarof cream topical Roviant PROPOSED INDICATIONS

Mild to severe plaque psoriasis (PSO)

CLINICAL OVERVIEW

Tapinarof is an aryl hydrocarbon receptor modulator that regulates the expression of interleukin (IL)-17 and the skin-barrier proteins filaggrin and loricrin. Two identical, 12-week, randomized, vehicle-controlled, phase 3 trials, PSOARING-1 (n=510); PSOARING-2 (n=515), evaluated the safety and efficacy of tapinarof in adults with mild to severe PSO. The baseline total BSA involvement ranged from 3% to 20%. A significantly greater response was seen with taparinof compared to vehicle based on the primary efficacy endpoint of PGA score of 0 (clear) or 1 (almost clear) with a minimum 2-grade improvement from baseline (PSOARING-1, 35.4% versus 6%, respectively [p<0.001]; PSOARING-2, 40.2% versus 6.3%, respectively [p<0.001]). Tapinarof also demonstrated benefit based on secondary endpoints, such as PASI-75, PASI-90, percentage of patients achieving PGA of 0 or 1, and mean change in percent BSA affected. The most common TEAEs reported with tapinarof were folliculitis, nasopharyngitis, and contact dermatitis. No serious TEAEs were identified. Durable effects up to 52 weeks and an off-treatment remittive effect (~4 months) were demonstrated in a long-term, open-label, extension study (PSORARING-3, n=763). Tapinarof 1% cream was applied to affected areas once daily. 7 | MAGELLANRX.COM

IMMUNODERMATOLOGY FDA APPROVAL TIMELINE May 26, 2022

FINANCIAL FORECAST (reported in millions)

The financial forecast for tapinarof is not currently available.

PLACE IN THERAPY

Psoriasis is a chronic, multisystem, immune-mediated, inflammatory disease involving the skin and joints characterized by hyperproliferation of epidermal keratinocytes. It affects an estimated 8 million people in the US. While PSO can begin at any age, onset has 2 peak age ranges, 20 to 30 years and 50 to 60 years. Treatment of PSO is based on the severity of the condition. Topical agents are typically used to manage mild to moderate or localized PSO. These include topical corticosteroids, calcineurin inhibitors (pimecrolimus, tacrolimus), vitamin D analogs (calcipotriene), retinoids (tazarotene), salicylic acid, anthralin, coal tar, and moisturizers. For moderate to severe cases, targeted systemic immunomodulators are recommended after failure of topical therapy alone, when phototherapy is not available. Immunomodulators include injectable monoclonal antibodies that reduce the level of pathogenic cytokines (e.g., TNF-α, IL-23, IL-17) and the oral PDE4 inhibitor apremilast (Otezla®). If approved, tapinarof will be a first-in-class aryl hydrocarbon receptor (AHR) modulator and roflumilast cream will be the first topical PDE4 inhibitor for the treatment of PSO in adults; roflumilast cream was also studied in pediatric patients with PSO. Both topical agents will encompass therapy for all levels of PSO severity and can be used anywhere on the body, including the face, scalp, and intertriginous areas. They will provide convenient alternatives to existing treatments, from topical steroids for mild cases to injectable immunomodulators and oral apremilast for moderate and severe cases. Roflumilast and tapinarof creams are also in phase 3 trials for atopic dermatitis. In addition, topical roflumilast foam is in phase 2 investigation for psoriasis of the scalp and seborrheic dermatitis. AstraZeneca’s oral formulation of roflumilast (Daliresp®) is currently available in the US for COPD. It is not expected to be developed for PSO.

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IMMUNOLODERMATOLOGY

ruxolitinib (Opzelura™) topical Incyte PROPOSED INDICATIONS

Vitiligo in adults and pediatric patients ≥ 12 years of age Ruxolitinib cream is currently indicated for the short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in select patients ≥ 12 years of age.

CLINICAL OVERVIEW

Ruxolitinib inhibits Janus kinases JAK1 and JAK2, which mediate the signaling of several cytokines and growth factors important for immune function. The placebo-controlled, double-blind, phase 3 TRuE-V1 and TRuE-V2 trials evaluated the safety and efficacy of ruxolitinib cream in approximately 600 patients ≥ 12 years of age with nonsegmental vitiligo on a total BSA ≥ 10%, including facial and nonfacial areas. At week 24, a total of 29.9% of patients treated with ruxolitinib achieved ≥ 75% improvement from baseline in the primary endpoint of Facial Vitiligo Area Scoring Index (F-VASI). In addition, 51% and 15%, respectively, achieved ≥ 50% and 90% improvement in F-VASI. TEAEs were consistent with phase 2 trials that reported application site pruritus and acne. Ruxolitinib 1.5% cream was applied topically to affected areas twice daily.

PLACE IN THERAPY

Vitiligo is a chronic autoimmune disorder in which a loss of melanocytes cause well-defined depigmented patches of skin. Vitiligo is estimated to occur in up to 2% of the global population. It affects all racial and ethnic groups, although it is more pronounced in individuals with darker complexions. Vitiligo can occur at any age, but the average age at onset is in the mid-twenties. Nonsegmental (generalized) vitiligo is the most common form of the condition and occurs over multiple areas of the body and progresses over time. Segmental (localized) vitiligo often begins during childhood and is limited to one or a few areas of the body. While vitiligo does not impact physical function, it can cause emotional distress and impact QOL. Rarely, the hypopigmented patches precede cutaneous melanoma. There are no FDA-approved agents for vitiligo. Patients may camouflage depigmented areas with cosmetics or tattooing and avoid sun exposure to minimize tanning. Off-label use of oral or topical corticosteroids, topical calcineurin inhibitors, narrowband ultraviolet B phototherapy, and oral psoralen plus ultraviolet A (PUVA) may stimulate repigmentation to varying degrees. Depigmentation with topical monobenzone has also been used in patients with extensive vitiligo. In addition, surgical transplantation has been used in select patients who are unresponsive to medical intervention. Ruxolitinib cream (Opzelura) is the only topical JAK inhibitor available in the US. It is currently indicated for the short-term treatment of mild to moderate atopic dermatitis in select patients. If approved, it will be the only medication specifically indicated for the treatment of vitiligo. Moreover, it will provide a convenient option that addresses the underlying immune etiology of the condition. In clinical trials, it demonstrated repigmentation in patients with diffuse (nonsegmental) patches affecting a BSA ≥ 10%. The ruxolitinib cream (Opzelura) labeling contains boxed warnings regarding serious infection, mortality, malignancy, MACE, and thrombosis, which are reported with oral JAK inhibitors. No serious TEAEs were identified in studies of ruxolitinib cream for vitiligo.

FDA APPROVAL TIMELINE April 18, 2022

 Priority Review

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$38

$92

$155

$208

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ONCOLOGY

sintilimab IV Eli Lilly PROPOSED INDICATIONS

Metastatic nonsquamous non-small cell lung cancer (NSCLC) with no EGFR or ALK genomic tumor aberrations

CLINICAL OVERVIEW

Sintilimab is an IV immunoglobulin G4 (IgG4) monoclonal antibody that inhibits the PD-1/PD-L1 pathway. The randomized, double-blind, phase 3, China-based ORIENT-11 trial evaluated the efficacy of sintilimab in 397 patients with previously untreated locally advanced or metastatic stage IIIB/C or IV nonsquamous NSCLC with no sensitizing EGFR mutations or ALK rearrangements. Patients were ineligible for surgery or local therapy. Sintilimab or placebo was given in combination with pemetrexed and platinum chemotherapy. After a median follow-up of 14.8 months, sintilimab led to a significantly longer median PFS (primary endpoint) compared to placebo (9.2 versus 5 months, respectively; p<0.0001). Notably, a longer PFS was demonstrated with sintilimab in patients with higher PD-L1 expression (tumor proportion score [TPS] ≥ 50%) compared to those with lower PD-L1 expression (TPS < 50%) (median PFS, not reached versus 7.1 months, respectively). In addition, sintilimab was associated with a 40% reduction in death compared to placebo (46.2% versus 64.1%, respectively; HR, 0.6; p=0.0003) after a median follow-up of 22.9 months. The most common adverse effects (≥ 20%) included decreased RBC, WBC, and platelet counts and increased ALT and AST. Grade ≥ 3 adverse events occurred at similar rates with sintilimab and placebo (61.7% and 58.5%, respectively). Sintilimab 200 mg was administered IV in combination with pemetrexed and platinum chemotherapy every 3 weeks for 4 cycles, followed by maintenance therapy with sintilimab plus pemetrexed every 3 weeks for up to 24 months or until disease progression or unacceptable toxicity.

PLACE IN THERAPY

Lung cancer is the leading cause of cancer-related death in the US. NSCLC accounts for 80% to 85% of all lung cancers. Adenocarcinoma, a type of nonsquamous NSCLC, is the most common subtype and occurs in nonsmokers. Chemotherapy regimens are based on ECOG performance status (PS). For nonsquamous NSCLC, single-agent therapy includes platinum agents, taxanes, pemetrexed, and gemcitabine. For patients with a poor ECOG PS status (PS 2), platinum doublet therapy with pemetrexed or a taxane is used. A PD-1/PD-L1 inhibitor may be added to first-line therapy for advanced or metastatic disease. Sintilimab demonstrated significant benefit when added to first-line background chemotherapy in patients with advanced or metastatic stage IIIB/C or IV nonsquamous NSCLC with no EGFR mutations or ALK rearrangements. If approved, it could compete with other PD-1/PD-L1 inhibitors (e.g., atezolizumab, cemiplimab-rwlc, nivolumab, and pembrolizumab) in this setting. Sintilimab is also in phase 3 trials in China for the treatment of select patients with locally advanced or metastatic nonsquamous NSCLC that harbors EGFR mutations or with advanced or metastatic squamous NSCLC. Notably, acceptance of the BLA demonstrates that the FDA may be receptive to allowing clinical trials performed outside the US to serve as the basis for regulatory filings.

FDA APPROVAL TIMELINE

March 2022 (FDA's Oncologic Drugs Advisory Committee to review on February 10, 2022)

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$30

$68

$229

$306

$371

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INFECTIOUS DISEASE

tebipenem pivoxil hydrobromide (HBr) oral Spero PROPOSED INDICATIONS

Complicated urinary tract infection (cUTI)

CLINICAL OVERVIEW

Tebipenem pivoxil HBr is an oral broad spectrum carbapenem antimicrobial. The randomized, double-blind, double-dummy, phase 3 ADAPT-PO trial compared the safety and efficacy of oral tebipenem pivoxil HBr versus IV ertapenem in 1,372 hospitalized adults with cUTI or acute pyelonephritis. ADAPT-PO was a multinational study with locations in the US. At the test-of-cure (TOC) day 19 (± 2 days), tebipenem pivoxil HBr was statistically non-inferior to ertapenem based on the primary endpoint of overall response, a composite of clinical cure plus microbial eradication (treatment difference -3.3%; met noninferiority margin of -12.5%). Both agents had high clinical cure rates at the TOC visit (~93%) and were effective against the Enterobacterales pathogens: Escherichia coli, Klebsiella pneumoniae, Proteus mirabilis, and Escherichia cloacea, including resistant phenotypes (e.g., extended-spectrum ß-lactamase [ESBL]-producing, fluoroquinolone-resistant, and trimethoprim-sulfamethoxazole-resistant). The most common TEAEs were similar between the groups, including diarrhea, headache, and nausea. No C. difficile-associated TEAEs were observed in the tebipenem pivoxil HBr arm, but 3 cases were reported in the ertapenem arm. In the clinical trial, patients were treated with 7 to 10 days (up to 14 days for bacteremic patients) of tebipenem pivoxil HBr 600 mg orally 3 times per day or ertapenem 1,000 mg via IV infusion once daily.

PLACE IN THERAPY

UTIs may involve the bladder (cystitis) and kidneys/ureters (pyelonephritis). Complicated UTI is considered when an acute UTI extends beyond the bladder (e.g., pyelonephritis, sepsis). UTIs are most often caused by Gram-negative bacteria, with E. coli as the usual pathogen, but Klebsiella spp, Proteus spp, Pseudomonas spp, enterococci, and staphylococci may also be involved. Antimicrobial resistance of uropathogens is increasing, leading to treatment challenges. Most notably is the emergence of ESBL-producing and fluoroquinoloneresistant pathogens. Oral fluoroquinolones are commonly prescribed for UTIs, including cUTI, in the outpatient setting. They provide antimicrobial activity against most uropathogens (including Pseudomonas aeruginosa); however, increasing bacterial resistance and serious adverse effects limit their use. The IV-administered carbapenem antibiotics (ertapenem, imipenem, meropenem) are indicated for cUTI. Notably, ertapenem has a narrower antimicrobial spectrum than other IV carbapenems and, therefore, may not be appropriate for select infections/populations (e.g., pathogens, resistance patterns). In the ADAPT-PO clinical trial, oral tebipenem pivoxil HBr demonstrated non-inferiority to IV ertapenem in overall response, but it has not been compared to other carbapenems in clinical trials. If approved, tebipenem pivoxil HBr will be the first oral carbapenem antibiotic in the US. It has the potential to help transition patients from the hospital to home following IV antibiotic therapy and/or prevent hospitalization in adults with cUTI caused by select uropathogens.

FDA APPROVAL TIMELINE June 27, 2022  Fast Track

Priority Review

QIDP

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$33

$145

$324

$509

$635

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HEMATOLOGY

vadadustat oral Akebia/Otsuka PROPOSED INDICATIONS

Anemia due to chronic kidney disease (CKD) in dialysis-dependent (DD) and non-dialysis dependent (NDD) patients

CLINICAL OVERVIEW

Vadadustat is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), which stabilizes HIF and stimulates erythropoietin (EPO) and RBC production in response to decreased oxygen levels in the body. Four randomized, open-label, active-controlled, phase 3 trials compared vadadustat and darbepoetin alfa in patients with CKD. Two trials enrolled patients with NDD-CKD (PRO2TECT trials; total n=3,476), and two trials enrolled patients with DD-CKD (INNO2VATE trials; total n=3,923). The PRO2TECT and INNO2VATE studies included patients with and without prior exposure to erythropoiesis-stimulating agents (ESAs). Across all trials, vadadustat was non-inferior to darbepoetin alfa based on the primary efficacy endpoint of difference in the change in mean Hb from baseline to weeks 24 through 36 (non-inferiority margin, -0.75 g/dL), and response was maintained in weeks 40 through 52. However, mixed results were seen regarding the primary safety endpoint of time to MACE, a composite of all-cause death, nonfatal MI, and nonfatal stroke. Both INNO2VATE trials demonstrated non-inferiority regarding MACE with vadadustat compared to darbepoetin alfa (non-inferiority HR margin, 1.25) among DD-CKD patients. However, pooled data from the PRO2TECT trials revealed a 17% higher rate of MACE with vadadustat than with darbepoetin alfa in NDD-CKD patients (HR, 1.17), which appeared largely driven by higher rates of nonfatal MI and non-CV death. A higher risk of MACE was seen primarily among patients at sites outside the US where the Hb target was higher in patients with prior ESA therapy (the baseline Hb range was 8 to 11 g/dL at US sites and 9 to 12 g/dL at non-US locations). The risk of MACE was increased by 6% (HR, 1.06) in US patients and by 30% (HR, 1.3) in non-US patients. No incidence of pulmonary hypertension or cancer was reported among any of the trials. In all 4 trials, vadadustat was initiated at 300 mg orally once daily. The dose was adjusted based on Hb level to a maximum of 600 mg daily.

PLACE IN THERAPY

An estimated 37 million people in the US have CKD. Anemia is prevalent in patients with CKD and worsens as the disease progresses. Severe anemia may hasten the progression of CKD and may lead to left ventricular hypertrophy and heart failure. Anemia is managed with ESA therapy (epoetin alfa [Epoetin®, Procrit®], darbepoetin alfa [Aranesp®]) and RBC transfusions as well as iron, vitamin B12, and folic acid supplementations. The FDA recommends that HCPs consider ESAs when the Hb level falls below 10 g/dL, as higher levels in patients with CKD increase the risk of CV events and death. If approved, vadadustat will be the first orally-administered small molecule HIF-PHI to treat CKD-related anemia. However, HIF pathways impact many biologic processes; therefore, this new therapeutic class comes with concerns regarding non-erythropoietic adverse effects, including increased risk of cancer, thrombosis, CVD, and diabetic retinopathy. While vadadustat provides a more convenient oral administration and comparable efficacy in maintaining target Hb levels compared to ESAs, reports of an increased risk of MACE may hinder its FDA approval in patients with NDD-CKD. Notably, in August 2021, roxadustat, another investigational oral HIF-PHI, failed to gain FDA-approval due, at least in part, to an increased risk of thrombotic events.

FDA APPROVAL TIMELINE March 29, 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$36

$118

$197

$276

$338

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NEUROLOGY

vutrisiran SC Alnylam PROPOSED INDICATIONS

Polyneuropathy (PN) of hereditary transthyretin-mediated (hATTR) amyloidosis

CLINICAL OVERVIEW

Vutrisiran is an RNA interface (RNAi) agent designed to silence messenger RNA and block the production of wild-type and variant transthyretin (TTR) protein. The ongoing, open-label, phase 3 HELIOS-A trial evaluated the safety and efficacy of vutrisiran in 164 patients with PN of hATTR amyloidosis. Researchers included IV patisiran as an active comparator. The efficacy of vutrisiran was compared to the placebo group in the APOLLO study, a pivotal trial for patisiran. At 9 months, a statistically significant improvement in neuropathy was seen with vutrisiran, based on the primary efficacy endpoint of mean change in the modified neuropathy impairment score (mNIS+7) from baseline (-2.24, -1.41, and 14.76 points, respectively, with vutrisiran, patisiran, and placebo; vutrisiran versus placebo difference, -17 points; p=3.54x10-12). A significant improvement in QOL with vutrisiran compared to placebo was also seen (mean changes in Norfolk QOL-DN score from baseline, -3.3, 0.1, and 12.9 points, respectively, with vutrisiran, patisiran, and placebo; vutrisiran versus placebo difference, -16.2; p=5.43x10-9). Responses were observed among patients regardless of prior TTR stabilizer therapy. Two serious TEAEs, dyslipidemia and UTI, were reported and considered due to vutrisiran. Injection site reactions were mild and transient. The study dose of vutrisiran was 25 mg SC every 3 months.

PLACE IN THERAPY

PN associated with hATTR amyloidosis (formerly known as transthyretin familial amyloid polyneuropathy) is a rare, progressive, fatal neurodegenerative condition caused by mutations in the TTR gene. This leads to an accumulation of abnormal amyloid proteins in organs and tissues and ensuing sensory and motor polyneuropathy. Neurologic manifestations vary based on the specific TTR mutations present and the patient’s age at onset. CNS involvement may be observed in advanced stages. It is estimated that 1 in 100,000 people are affected by hATTR amyloidosis in the US. The onset of symptoms typically appears between 20 and 50 years of age. If left untreated, death occurs within 7 to 12 years after diagnosis. The TTR proteins are primarily produced in the liver. Prior to the advent of the TTR-directed RNAi agents inotersen (Tegsedi®) and patisiran (Onpattro®; also by Alnylam), liver transplantation was the only DMT available for hATTR amyloidosis. Other therapies focus on symptom management. If approved, vutrisiran will be the third TTR-targeting treatment for hATTR amyloidosis-associated PN. It is well tolerated and appears to be as effective as patisiran. Vutrisiran is expected to be available as a quarterly, HCP-administered SC injection, a more convenient regimen compared to every-3-week IV infusions of patisiran and weekly selfadministered SC injections of inotersen. Moreover, inotersen is associated with an increased risk of serious and life-threatening thrombocytopenia, and, therefore, is only available through a REMS program. Acoramidis and eplontersen are investigational TTR-targeting agents in phase 3 trials for PN of hATTR amyloidosis. In addition, vutrisiran, patisiran, acoramidis, and eplontersen are in phase 3 trials for cardiomyopathy of hATTR, which is an FDA-approved indication for oral tafamidis/tafamidis meglumine (Vyndamax®/Vyndaqel®).

FDA APPROVAL TIMELINE April 14, 2022  Fast Track

Orphan Drug

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$38

$108

$178

$297

$432

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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 surround biosimilars, and regulatory and litigation hurdles remain. The FDA has issued final and draft guidances. Select FDA biosimilar guidances are noted here. In January 2017, the agency issued final guidance on the nonproprietary naming of biologic products, which also applies to biosimilars. The biological products must bear a core name followed by a distinguishing 4-letter, lowercase, hyphenated suffix that is devoid of meaning. The 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 33 biosimilars have received FDA approval. Of these, only 21 have entered the market. APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Zarxio® (filgrastim-sndz)

Novartis/Sandoz

March 2015

Inflectra® (infliximab-dyyb)

Pfizer/Celltrion

April 2016

Erelzi® (etanercept-szzs)

Novartis/Sandoz

August 2016

Amjevita™ (adalimumab-atto)

Amgen

September 2016

Renflexis® (infliximab-abda)

Samsung Bioepis/ Merck

May 2017

Cyltezo (adalimumab-adbm)

Boehringer Ingelheim

August 2017

Mvasi (bevacizumab-awwb)

Amgen

September 2017

Ixifi™ (infliximab-qbtx)†

Pfizer

December 2017

Ogivri® (trastuzumab-dkst)

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) Rituxan (Genentech) Humira (Abbvie) Neulasta (Amgen)

APPROVED BIOSIMILARS continued APPROVED BIOSIMILARS Brand Name (Nonproprietary name)

Manufacturer

Approval Date

Interchangeable

Commercially Available

Originator (Manufacturer)

Avsola® (infliximab-axxq)

Amgen

December 2019

Remicade (Janssen)

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

Lantus (Sanofi)

Yusimry™ (adalimumab-aqvh)

Coherus

December 2021

Humira (Abbvie)



  



Rituxan (Genentech)

Lucentis® (Genentech)

Neulasta (Amgen) Lantus® (SanofiAventis) Humira (Abbvie)

† 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.

IMMUNOLOGY

adalimumab SC Alvotech and Celltrion are seeking approval for their investigational biosimilars to Abbvie’s Humira, 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.

FDA APPROVAL TIMELINE Alvotech (AVT-02) Pending

Celltrion (Yuflyma) August 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$16,324

$9,416

$6,655

$4,998

$3,557

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

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BIOSIMILAR OVERVIEW continued

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

$6,042

$6,177

$5,988

$5,748

$5,263

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

ONCOLOGY

bevacizumab IV Amneal, Bio-Thera Solutions, Centus/AstraZeneca, Samsung Bioepis/Merck, 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 Amneal (BEVZ92) April to June 2022

Bio-Thera Solutions (BAT1706) Pending Centus/AstraZeneca (FKB238) Pending Samsung Bioepis/Merck (Aybintio) Pending Viatris/Biocon (Bmab-100) Pending

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$759

$659

$578

$518

$466

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

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

FDA APPROVAL TIMELINE Amneal Pending

Apotex (Grastofil) Pending

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$55

$49

$45

$39

$35

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

BLOOD MODIFIER

pegfilgrastim SC Amneal, 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 Amneal (TPI-120) Pending Apotex (Lapelga) Pending Lupin (Lupifil-P) April 2022 Merck/Fresenius (MSB-11455) Pending

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$1,289

$1,100

$969

$860

$768

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

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BIOSIMILAR OVERVIEW continued

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

$1,353

$1,205

$1,054

$925

$832

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

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 July to August 2022

FINANCIAL FORECAST (reported in millions) Year

2022

2023

2024

2025

2026

Projected Total US Sales

$493

$430

$392

$367

$345

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

$1,337

$1,368

Behavioral health

tirzepatide

Oncology

CTX001

Diabetes/Metabolic

Hematology/Gene therapy

$3,360

$949

tiragolumab

deucravacitinib

$800

$1,735

Immunology

Oncology

sotrovimab

dextromethorphan/ bupropion

COVID-19

$752

Behavioral health

$1,277 Novavax COVID-19 vaccine (NVX-CoV2373)

donanemab

COVID-19

Neurology

$5,532

$4,919 gantenerumab

Moderna (Spikevax) COVID-19 vaccine

Neurology

$1,975

COVID-19

$2,054

mirikizumab Immunology

$732

lenadogene nolparvovec (GS-010)

Ophthalmology/Gene therapy

$186

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

IN PHASE PHASE 33 TRIALS TRIALS

64%

69%

36%

31%

36%

26%

Specialty

19%

14%

15%

Traditional

Orphan Drug

Priority Review

Breakthrough Therapy

Biosimilar

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

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

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

Submitted (New Drugs) adagrasib

Mirati

NSCLC (2nd-line)

Oral

Submitted – NDA; Breakthrough Therapy; RTOR

2022

lutetium 177Lu-PSMA-617

Novartis

Prostate cancer (metastatic, castrationresistant)

Submitted – NDA; Breakthrough Therapy; Priority Review

Jan-Jun 2022

penpulimab

Akeso

Nasopharyngeal cancer (metastatic, 3rd-line)

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

Jan-Jun 2022

faricimab

Genentech

DME; Wet AMD

Intravitreal

Submitted – BLA; Priority Review

01/31/2022

treprostinil DPI (Tyvaso DPI)

United Therapeutics

PAH; Idiopathic pulmonary fibrosisassociated pulmonary hypertenson

Inhaled

Submitted – NDA

February 2022

benzoyl peroxide

Sol-Gel

Rosacea

Transdermal

Submitted – 505(b)(2) NDA

Feb-Mar 2022

sutimlimab

Sanofi

Cold agglutinin diseaserelated hemolysis

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

02/04/2022

mitapivat

Agios

Pyruvate kinase deficiency

Oral

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

02/17/2022

dextroamphetamine

Hisamitsu

ADHD

Transdermal

Submitted – NDA

02/22/2022

bardoxolone methyl

Reata

Alport syndrome-related CKD

Oral

Submitted – NDA; Orphan 02/25/2022 Drug

immune globulin (human) 10%

Green Cross

PHI

Submitted – BLA

02/25/2022

ciltacabtagene autoleucel

Janssen/Legend

Multiple myeloma (relapsed/refractory)

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

02/28/2022

episalvan

Amryt

Epidermolysis bullosa

Topical

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

02/28/2022

lenacapavir

Gilead

HIV-1 infection treatment (MDR)

Oral, SC

Submitted – NDA; Breakthrough Therapy; Priority Review

02/28/2022

pacritinib

CTI

Myelofibrosis

Oral

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

02/28/2022

sintilimab

Eli Lilly

NSCLC (metastatic, nonsquamous, without EGFR or ALK mutations)

Submitted – BLA

March 2022

donepezil

Corium

Alzheimer’s disease (mild-severe)

Transdermal

Submitted – 505(b)(2) NDA

03/11/2022

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PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

ganaxolone

Marinus

Cyclin-dependent kinaselike 5 (CDKL5) deficiency disorder-related seizures

IV, Oral

Submitted – NDA; Orphan 03/18/2022 Drug; Priority Review; Rare Pediatric Disease

relatlimab/nivolumab

Bristol-Myers Squibb

Melanoma (unresectable or metastatic, ages ≥ 12 years)

Submitted – BLA; Priority Review

03/18/2022

ublituximab

CLL/SLL (in combination with umbralisib)

Submitted – BLA; Fast Track; Orphan Drug

03/25/2022

udenafil

Dong-A Socio/ Allergan

Single ventricle heart disease after Fontan palliation

Oral

Submitted – NDA; Orphan 03/25/2022 Drug

vadadustat

Akebia/Otsuka

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

Oral

Submitted – NDA

03/29/2022

benegrastim

Evive

Neutropenia/leukopenia

Submitted – BLA

03/30/2022

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Lupin

Neutropenia/leukopenia

Submitted – BLA

April 2022

risperidone ER

Teva

Schizophrenia

Submitted – 505(b)(2) NDA

Apr-May 2022

bevacizumab (biosimilar to Genentech’s Avastin)

Amneal

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

Submitted – BLA

Apr-Jun 2022

dexmedetomidine

Bioxcel

Bipolar disorder-related acute aggitation; Schizophrenia-related acute aggitation

Submitted – 505(b)(2) NDA; Fast Track

04/05/2022

casirivimab/imdevimab

Regeneron

COVID-19 treatment (outpatient setting); COVID-19 post-exposure prophylaxis

IM, IV, SC

Submitted – BLA; Priority Review

04/13/2022

vutrisiran

Alnylam

Polyneuropathy of hereditary transthyretinmediated amyloidosis

Submitted – NDA; Fast Track; Orphan Drug

04/14/2022

mavacamten

Bristol-Myers Squibb

Obstructive hypertrophic cardiomyopathy

Oral

Submitted – NDA; Breakthrough Therapy; Orphan Drug

04/28/2022

meloxicam/rizatriptan

Axsome

Migraine treatment

Oral

Submitted – 505(b)(2) NDA

04/29/2022

surufatinib

Hutchmed

Neuroendocrine tumors

Oral

Submitted – NDA; Fast Track; Orphan Drug

04/29/2022

toripalimab

Coherus/AstraZeneca Nasopharyngeal carcinoma (recurrent or metastatic)

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

04/29/2022

faricimab

Genentech

Diabetic retinopathy

Intravitreal

Submitted – BLA

May 2022

vonoprazan

Phathom

H. pylori infection (in combination with amoxicillin ± clarithromycin)

Oral

Submitted – NDA; Priority Review; QIDP

05/03/2022

edaravone

Mitsubishi Tanabe

ALS

Oral

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

05/12/2022

cantharidin

Verrica

Molluscum contagiosum

Topical

Submitted – NDA

05/24/2022

tapinarof

Roivant

PSO (mild-severe)

Topical

Submitted – NDA

05/26/2022

23 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

tirzepatide

Eli Lilly

T2DM

Submitted – NDA; Priority Review

05/30/2022

trientine tetrahydrochloride

GMP-Orphan

Wilson’s disease

Oral

Submitted – NDA; Orphan Jun-Jul 2022 Drug

trastuzumab (biosimilar to Genentech’s Herceptin)

Tanvex

Breast cancer; Gastric/ gastroesophageal cancer

Submitted – BLA

Jul-Aug 2022

teclistamab

Janssen

Multiple myeloma (R/R)

Submitted – BLA; Breakthrough Therapy

Jun-Dec 2022

spesolimab

Boehringer Ingelheim

Psoriasis (pustular flares)

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

06/01/2022

sodium phenylbutyrate

Acer

Urea cycle disorders

Oral

Submitted – 505(b)(2) NDA

06/03/2022

tebipenem pivoxil HBr

Spero

UTI (complicated)

Oral

Submitted – NDA; Fast Track; Priority Review; QIDP

06/27/2022

tauroursodeoxycholic acid/ Amylyx sodium phenylbutyrate

ALS

Oral

Submitted – NDA; Orphan 06/29/2022 Drug; Priority Review

bulevirtide

Gilead

Hepatitis D infection treatment (chronic, compensated liver disease)

Submitted – NDA; Breakthrough Therapy; Orphan Drug

Jul-Nov 2022

tislelizumab

Beigene

Esophageal cancer

Submitted – BLA; Orphan Drug

07/12/2022

cipaglucosidase alfa

Amicus

Pompe disease (in combination with miglustat)

Submitted – BLA; Breakthrough Therapy; Orphan Drug

07/29/2022

roflumilast cream

Arcutis/AstraZeneca

PSO (mild-severe)

Topical

Submitted – NDA

07/29/2022

adalimumab (biosimilar to Abbvie’s Humira)

Celltrion

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

Submitted – BLA

August 2022

poziotinib

Spectrum

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

Oral

Submitted – NDA; Fast Track

Aug-Dec 2022

ranibizumab (biosimilar to Genentech’s Lucentis)

Coherus

Wet AMD

Intravitreal

Submitted – BLA

08/02/2022

trivalent measles-mumpsrubella (MMR) vaccine

GlaxoSmithKline

Measles, mumps, and rubella immunization

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

deucravacitinib

Bristol-Myers Squibb

PSO

Oral

Submitted – NDA

09/10/2022

linzagolix

Obseva

Uterine fibroids

Oral

Submitted – NDA

09/13/2022

dasatinib

Xspray

CML

Oral

Submitted – 505(b)(2) NDA

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

ublituximab

MS (relapsing)

Submitted – BLA

09/28/2022

24 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

aflibercept (biosimilar to Regeneron’s Eylea)

Viatris/Janssen

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

Intravitreal

Submitted – BLA

October 2022

apomorphine infusion pump

Supernus

Parkinson’s disease

Submitted – NDA

10/07/2022

dovitinib

Allarity/Novartis

RCC (3rd-line)

Oral

Submitted – NDA

December 2022

trastuzumab (biosimilar to Genentech’s Herceptin)

Novartis

Breast cancer; Gastric/ gastroesophageal cancer

Submitted – BLA

12/20/2022

sotagliflozin

Lexicon

Heart failure in patients with T2DM

Oral

Submitted – NDA

12/30/2022

adalimumab (biosimilar to Abbvie’s Humira)

Alvotech

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

Submitted – BLA

Pending

bevacizumab (biosimilar to Genentech’s Avastin)

Bio-Thera Solutions

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/ Merck

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

bimekizumab

UCB

PSO

Submitted – BLA

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)

Amneal

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

filgrastim (biosimilar to Amgen’s Neupogen)

Apotex

Neutropenia/leukopenia

IV, SC

Submitted – BLA

Pending

Moderna COVID-19 vaccine (mRNA-1273)

Moderna

COVID-19 prevention (ages 12-17 years)

Submitted – EUA; Fast Track

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Amneal

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Apotex

Neutropenia/leukopenia

Submitted – BLA

Pending

pegfilgrastim (biosimilar to Amgen’s Neulasta)

Merck/Fresenius

Neutropenia/leukopenia

Submitted – BLA

Pending

sodium oxybate (once-nightly)

Avadel

Narcolepsy-related excessive daytime sleepiness and cataplexy

Oral

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

Pending

25 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

Submitted (Supplementals) olaparib (Lynparza )

AstraZeneca

Breast cancer (BRCA+, HER2-, high-risk, early disease, 2nd-line)

Oral

Submitted – sNDA; Priority Review

Jan-Mar 2022

benralizumab (Fasenra®)

AstraZeneca

Nasal polyposis

Submitted – sBLA

Jan-Apr 2022

cabotegravir/rilpivirine (Cabenuva)

Viiv

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

Submitted – sNDA

Jan-Jun 2022

risankizumab-rzaa (Skyrizi®)

Abbvie

PsA

Submitted – sBLA

February 2022

tecovirimat (Tpoxx®)

Siga

Smallpox treatment (unable to swallow Tpoxx capsule)

Submitted – sNDA; Fast Track; Orphan Drug

03/04/2022

setmelanotide (Imcivree™)

Rhythm

Alström-related obesity and hunger; BardetBiedle syndrome-related obesity and hunger

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

03/16/2022

estradiol/progesterone (Bijuva®) 0.5 mg/100 mg (low-dose)

TherapeuticsMD

Menopause-related moderate to severe vasomotor symptoms

Oral

Submitted – 505(b)(2) sNDA

03/21/2022

fenfluramine HCl (Fintepla®)

Zogenix

Lennox-Gastaut syndrome

Oral

Submitted – sNDA; Orphan Drug; Priority Review

03/25/2022

umbralisib (Ukoniq®)

CLL/SLL (in combination with ublituximab)

Oral

Submitted – sNDA; Orphan Drug

03/25/2022

luspatercept-aamt (Reblozyl)

Acceleron

β-thalassemia (non-

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

03/27/2022

pembrolizumab (Keytruda®)

Merck

Endometrial carcinoma (advanced, MSI-H or dMMR, monotherapy, ≥ 1 prior systemic therapy, curative surgery or radiation ineligible)

Submitted – sBLA

03/28/2022

semaglutide (Ozempic®) 2 mg

Novo Nordisk

T2DM

Submitted – sNDA

03/28/2022

empagliflozin (Jardiance®)

Boehringer Ingelheim

Reduce the risk of CV death and hospitalization for HF (independent of LVEF)

Oral

Submitted – sNDA; Fast Track; Priority Review

03/30/2022

copanlisib (Aliqopa®)

Bayer

NHL (indolent R/R, in combination with rituximab)

Submitted – sNDA; Fast Track; Orphan Drug

April 2022

rivaroxaban (Xarelto®)

Janssen

Venous thromboembolism (treatment ages birth to < 18 years; prophylaxis ages ≥ 2 years) with congenital heart disease after Fontan procedure

Oral

Submitted – sNDA

Apr-Jun 2022

fam-trastuzumab deruxtecan-nxki (Enhertu®)

Daiichi Sankyo

Breast cancer (HER2+, unresectable or metastatic, ≥ 1 prior antiHER2-based regimens)

Submitted – sBLA; Breakthrough Therapy; Priority Review

Apr-Jun 2022

brolucizumab-dbll (Beovu®)

Novartis

DME

Intravitreal

Submitted – sBLA

Apr-Sep 2022

26 | MAGELLANRX.COM

transfusion dependent)

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

axicabtagene ciloleucel (Yescarta®)

Gilead

Large B cell lymphoma (R/R, 2nd-line)

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

04/01/2022

pneumococcal 15-valent conjugate vaccine (Vaxneuvance™)

Merck

Invasive pneumococcal disease prevention (ages 6 weeks-17 years)

Submitted – sBLA; Breakthrough Therapy; Priority Review

04/01/2022

ruxolitinib cream (Opzelura)

Incyte

Vitiligo

Topical

Submitted – sNDA; Priority Review

04/18/2022

tisagenlecleucel-t (Kymriah®)

Novartis

Follicular lymphoma (R/R, 3rd-line)

Submitted – sBLA; Orphan Drug; Priority Review; RMAT

04/27/2022

viloxazine (Qelbree®)

Supernus

ADHD (adults)

Oral

Submitted – sNDA

04/29/2022

relugolix/estradiol/ norethindrone (Myfembree®)

Myovant

Endometriosis

Oral

Submitted – sNDA

05/06/2022

ipilimumab (Yervoy®)

Bristol-Myers Squibb

Esophageal squamous cell carcinoma (advanced or metastatic, 1st-line, in combination with nivolumab)

Submitted – sBLA

05/28/2022

nivolumab (Opdivo®)

Bristol-Myers Squibb

Esophageal squamous cell carcinoma (1stline, in combination with ipilimumab or fluoropyriidine/platinum chemotherapy)

Submitted – sBLA

05/28/2022

ravulizumab-cwvz (Ultomiris®)

Alexion

Myasthenia gravis

IV, SC

Submitted – sBLA; Priority Review

Jun-Jul 2022

upadacitinib (Rinvoq™)

Abbvie

Oral

Submitted – sNDA; Orphan Drug

July 2022

risankizumab-rzaa (Skyrizi)

Abbvie

Submitted – sBLA; Orphan Drug

07/20/2022

abacavir/dolutegravir/ lamivudine (Triumeq®) dispersible tablet

Viiv

HIV-1 treatment (pediatrics weighing 14 kg to < 40 kg)

Oral

Submitted – sNDA

August 2022

adalimumab-bwwd (Hadlima) 100 mg/mL

Samsung Bioepis/ Merck

JIA; UC

Submitted – sBLA

August 2022

bupivacaine/meloxicam (Zynrelef™)

Heron

Postsurgical pain (foot and ankle, small–to– medium open abdominal, and lower extremity total joint arthroplasty surgical procedures)

Instillation

Submitted – sNDA; Breakthrough Therapy; Fast Track

08/04/2022

ustekinumab (Stelara®)

Janssen

PsA (ages ≥ 5 years)

Submitted – sBLA

08/08/2022

aprepitant (Cinvanti )

Heron

Postoperative nausea and vomiting prophylaxis

Submitted – 505(b)(2) sNDA

09/18/2022

cemiplimab-rwlc (Libtayo®)

Regeneron

NSCLC (advanced, firstline, in combination with chemotherapy)

Submitted – sBLA

09/19/2022

ibalizumab (Trogarzo) IV push formulation

Theratechnologies

HIV-1 infection treatment

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

10/06/2022

27 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

lumasiran (Oxlumo®)

Alnylam

Primary hyperoxaluria type 1 (advanced)

Submitted – sNDA; Breakthrough Therapy; Orphan Drug

10/14/2022

Pfizer-Biontech COVID-19 vaccine (Comirnaty®)

Pfizer/Biontech

COVID-19 prevention (ages 12-15 years)

Submitted – sBLA; Fast Track

10/14/2022

upadacitinib (Rinvoq)

Abbvie

Non-radiographic axial spondyloarthritis

Oral

Submitted – sNDA

11/07/2022

axicabtagene ciloleucel (Yescarta)

Gilead

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

Submitted – sBLA; Breakthrough Therapy; Orphan Drug

Pending

baricitinib (Olumiant®)

Eli Lilly

Atopic dermatitis (moderate-severe)

Oral

Submitted – sNDA

Pending

infliximab-dyyb (Inflectra)

Celltrion

IBS

IV, SC

Submitted – sBLA

Pending

upadacitinib (Rinvoq)

Abbvie

Oral

Submitted – sNDA

Pending

Intraocular

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

Phase 3 (New Drugs) AAV-RPGR gene therapy

Janssen

Retinitis pigmentosa (X-linked)

abaloparatide-TD

Radius

Osteoporosis/osteopenia

Transdermal

Phase 3 – NDA

TBD

acoramidis

Bridgebio

Transthyretin amyloid cardiomyopathy (ATTRCM)

Oral

Phase 3 – NDA

TBD

adalimumab (biosimilar to Abbvie’s Humira)

Fresenius

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

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

amcenestrant

Sanofi

Breast cancer

Oral

Phase 3 – NDA

TBD

anthrax vaccine, adsorbed

Emergent

Anthrax infection

Phase 3 – BLA; Fast Track

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

AT-527

Atea

COVID-19

Oral

Phase 3 – NDA

TBD

ataluren

PTC

DMD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

atezolizumab (Tecentriq®)

Genentech

NSCLC (adjuvant, post surgery and platinumbased chemotherapy, PD-L1 ≥ 1%)

Phase 3 – BLA

TBD

autologous CRISPRmodified human hematopoietic stem and progenitor cells (CTX001)

Crispr Therapeutics

SCD; Thalassemia

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

TBD

autologous genetically modified human dermal fibroblasts

Castle Creek

Epidermolysis bullosa

Intradermal

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

TBD

baclofen/naltrexone/ sorbitol

Pharnext

Charcot-Marie-Tooth disease

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

balstilimab

Agenus

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

Phase 3 – BLA; Fast Track

TBD

28 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

bamlanivimab

Eli Lilly

COVID-19

Phase 3 – BLA

TBD

bentracimab

Phasebio/ AstraZeneca

Ticagrelor (Brilinta®) reversal

Phase 3 – BLA; Breakthrough Therapy

TBD

beremagene geperpavec

Krystal

Epidermolysis bullosa

Topical

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

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

DME; RVO-associated macular edema; Wet AMD

Intravitreal

Phase 3 – BLA

TBD

BIIB059

Biogen

SLE

Phase 3 – BLA

TBD

bimekizumab

UCB

AS; 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

BRII-196/BRII-198

Brii

COVID-19

Phase 3 – BLA

TBD

cannabidiol

Zynerba

Fragile X syndrome

Transdermal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

capsaicin

Concentric Analgesics

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – 505(b)(2) NDA; Fast Track

TBD

ceftobiprole medocaril

Basilea

ABSSSI; CAP; HAP; Septicemia

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 Biotech

Cutaneous squamous cell carcinoma (metastatic)

Phase 3 – BLA

TBD

COVID-19 vaccine (C19VAZ; formerly AZD-1222; ChAdOx1)

AstraZeneca

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (INO-4800)

Inovio

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (JNJ-78436735; formerly Ad26.COV2.S)

Janssen

COVID-19

Phase 3 – BLA

TBD

COVID-19 vaccine (MT-2766)

Medicago/ GlaxoSmithKline

COVID-19

Phase 3 – BLA; Fast Track

TBD

COVID-19 vaccine (NVX-CoV2373)

Novavax

COVID-19

Phase 3 – BLA; Fast Track

TBD

29 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

COVID-19 vaccine (SP0253)

Sanofi/ GlaxoSmithKline

COVID-19

Phase 3 – BLA

TBD

crovalimab

Genentech

Paroxysmal nocturnal hemoglobinuria

IV, SC

Phase 3 – BLA; Orphan Drug

TBD

dalcetrapib

Dalcor

Acute coronary syndrome (ADCY9 AA genotype)

Oral

Phase 3 – NDA

TBD

daprodustat

GlaxoSmithKline

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

Oral

Phase 3 – NDA

TBD

darvadstrocel

Takeda

Phase 3 – BLA; Orphan Drug

TBD

dengue tetravalent vaccine

Takeda

Dengue fever

Phase 3 – BLA; Fast Track

TBD

denosumab (biosimilar to Amgen’s Prolia®)

Novartis

Osteoporosis/osteopenia

Phase 3 – BLA

TBD

dersimelagon

Mitsubishi Tanabe

Porphyria

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

despropyl macitentan

Janssen

Hypertension

Oral

Phase 3 – NDA

TBD

difluprednate XR

Sun

Ocular pain/inflammation

Ophthalmic

Phase 3 – NDA

TBD

donanemab

Eli Lilly

Alzheimer’s disease (early)

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

Allarity/Novartis

Breast cancer

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

Paroxysmal nocturnal hemoglobinuria

Phase 3 – BLA

TBD

efanesoctocog alfa

Sanofi

Hemophilia A

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

efgartigimod

Argenx

ITP; Myasthenia gravis; Pemphigus vulgaris

Phase 3 – BLA; Orphan Drug

TBD

efruxifermin

Akero/Amgen

NASH

Phase 3 – BLA; Fast Track

TBD

elacestrant

Menarini

Breast cancer

Oral

Phase 3 – NDA; Fast Track

TBD

enmetazobactam

Allecra

UTI (complicated)

Phase 3 – NDA; Fast Track; QIDP

TBD

ensifentrine

Verona

COPD

Inhaled

Phase 3 – NDA

TBD

ensovibep

Molecular Partners

COVID-19

IV, SC

Phase 3 – BLA; Fast Track

TBD

EP-2101 therapeutic vaccine

OSE

NSCLC

Phase 3 – NDA; Orphan Drug

TBD

epcoritamab

Genmab/Abbvie

DLBCL

Phase 3 – BLA

TBD

epinephrine

Bryn

Anaphylaxis

Intranasal

Phase 3 – NDA; Fast Track

TBD

eplontersen

Akcea

Transthyretin amyloid polyneuropathy

Phase 3 – NDA

TBD

eprenetapopt

Aprea

Myelodysplastic syndrome

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

TBD

30 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

esreboxetine

Axsome/Pfizer

Fibromyalgia

Oral

Phase 3 – NDA

TBD

etanercept (biosimilar to Amgen’s Enbrel)

Coherus

RA; Polyarticular JIA; AS; PSO; PsA

Phase 3 – BLA

TBD

etavopivat

Forma

SCD

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

etesevimab

Eli Lilly

COVID-19

Phase 3 – BLA

TBD

etranacogene dezaparvovec

Uniqure

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

etrasimod

Arena

CD; UC

Oral

Phase 3 – NDA; Orphan Drug

TBD

etrolizumab

Genentech

Phase 3 – BLA

TBD

fasinumab

Regeneron

Osteoarthritis pain (knee)

Phase 3 – BLA

TBD

favipiravir

Dr. Reddy’s

COVID-19; Influenza

Oral

Phase 3 – NDA

TBD

fexapotide triflutate

Nymox

BPH

Intratumoral

Phase 3 – NDA

TBD

fezolinetant

Astellas

Menopause vasomotor symptoms

Oral

Phase 3 – NDA

TBD

fidanacogene elaparvovec

Pfizer

Hemophilia B

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

filgotinib

Gilead

CD; UC

Oral

Phase 3 – NDA

TBD

firmacute eubacterial spores

Seres

Clostridium difficileassociated diarrhea

Oral

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

fitusiran

Sanofi

Hemophilia A and B

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

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

Allergan

Female reproductive disorder

Phase 3 – BLA

TBD

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

Finox

Female reproductive disorder

Phase 3 – BLA

TBD

gantenerumab

Genentech

Alzheimer’s disease

Phase 3 – BLA; Breakthrough Therapy

TBD

garadacimab

CSL

Hereditary angioedema

Phase 3 – BLA; Orphan Drug

TBD

gavorestat

Applied Therapeutics Galactosemia

Oral

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

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

givinostat

Italfarmaco

DMD

Oral

Phase 3 – NDA; Orphan Drug; Rare Pediatric Disease

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

idursulfase

Takeda/Sanofi

Mucopolysaccharidosis II Intrathecal (MPS II; Hunter syndrome)

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

31 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

inavolisib

Genentech

Breast cancer

Oral

Phase 3 – NDA

TBD

inclacumab

Global Blood Therapeutics

SCD

Phase 3 – BLA

TBD

infliximab (biosimilar to Janssen’s Remicade)

Nichi-Iko

RA; AS; PSO; PsA; CD; UC

Phase 3 – BLA

TBD

ingenol disoxate

Leo

Actinic keratoses

Topical

Phase 3 – NDA

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Sanofi

T1DM; T2DM

Phase 3 – BLA

TBD

insulin glargine (biosimilar to Sanofi’s Lantus)

Gan & Lee

T1DM; T2DM

Phase 3 – BLA

TBD

iodine-131 apamistamab

Actinium

AML

Phase 3 – BLA; Orphan Drug

TBD

iptacopan

Novartis

Oral Complement 3 (C3) glomerulopathy; Immunoglobulin A nephropathy (Berger’s disease); Paroxysmal nocturnal hemoglobinuria

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

TBD

itepekimab

Regeneron

COPD

Phase 3 – BLA

TBD

KSI-301

Kodiak

DME; RVO-associated macular edema; Wet AMD

Intravitreal

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

lecanemab

Eisai

Alzheimer’s disease (early)

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

lenadogene nolparvovec (GS010)

Gensight

Leber’s dereditary optic neuropathy

Intravitreal

Phase 3 – BLA; Orphan Drug

TBD

leniolisib

Pharming/Novartis

Activated phosphoinositide 3-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

leronlimab

Cytodyn

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

Phase 3 – BLA; Fast Track

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

lorecivivint

Samumed

Osteoarthritis pain (knee)

Intraarticular

Phase 3 – NDA

TBD

32 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

lotilaner

Tarsus

Demodex blepharitis

Ophthalmic

Phase 3 – NDA

TBD

magrolimab

Forty Seven

Myelodysplastic syndrome

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

TBD

marstacimab

Pfizer

Hemophilia A and B

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

masitinib

AB Science

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

Oral

Phase 3 – NDA; Orphan Drug

TBD

mavorixafor

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

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

TBD

melphalan

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

microbiota suspension

Ferring

C. difficile infection (recurrent)

Rectal

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

TBD

minocycline/edetate/ethyl alcohol

Citius

Catheter-related bloodstream infection (CRBSI)

Phase 3 – NDA; Fast Track; QIDP

TBD

mirikizumab

Eli Lilly

CD; UC

IV, SC

Phase 3 – BLA

TBD

mirvetuximab soravtansine

Immunogen

Ovarian cancer

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

mitapivat

Agios

Thalassemia

Oral

Phase 3 – NDA; Orphan Drug

TBD

molnupiravir

Merck

COVID-19

Oral

Phase 3 – NDA

TBD

momelotinib

Sierra Oncology/ Gilead

Myelofibrosis

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

mosunetuzumab

Genentech

Follicular lymphoma (R/R)

IV, SC

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

motixafortide

Biolinerx

Stem cell mobilization

Phase 3 – NDA; Orphan Drug

TBD

nabiximols

MS-related spasticity

Oral transmucosal

Phase 3 – NDA

TBD

nalbuphine ER

Trevi

Pruritus

Oral

Phase 3 – NDA

TBD

naloxone

Orexo

Opioid overdose

Intranasal

Phase 3 – 505(b)(2) NDA

TBD

naloxone hydrochloride dihydrate

Elorac

Pruritus

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

narsoplimab

Omeros

Hemolytic uremic syndrome; HSCTassociated thrombotic microangiopathy

IV, SC

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

TBD

33 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

natalizumab (biosimilar to Biogen’s Tysabri®)

Novartis

Phase 3 – BLA

TBD

navitoclax

Abbvie/Genentech

Myelofibrosis

Oral

Phase 3 – NDA; Orphan Drug

TBD

nedosiran

Dicerna

Hyperoxaluria

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

TBD

nemolizumab

Galderma

Atopic dermatitis (moderate-severe); Pruritus

Phase 3 – BLA; Breakthrough Therapy

TBD

nipocalimab

Janssen

Autoimmune hemolytic anemia; Myasthenia gravis

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

nirmatrelvir/ritonavir (Paxlovid™)

Pfizer

COVID-19

Oral

Phase 3 – NDA

TBD

nirsevimab

AstraZeneca

RSV infection prevention

IM, IV

Phase 3 – BLA; Breakthrough Therapy; Fast Track

TBD

nomacopan

Akari

Hemolytic uremic syndrome; HSCTassociated thrombotic microangiopathy; Paroxysmal nocturnal hemoglobinuria

Phase 3 – BLA; Fast Track; Orphan Drug

TBD

olezarsen

Akcea

Familial chylomicronemia syndrome

Phase 3 – NDA

TBD

olipudase alfa

Sanofi

Niemann-Pick disease

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

omecamtiv mecarbil

Cytokinetics

Chronic heart failure (with Oral reduced ejection fraction)

Phase 3 – NDA; Fast Track

TBD

OTL-103

Orchard

Wiskott-Aldrich syndrome IV

Phase 3 – BLA; Orphan Drug; RMAT

TBD

oxalobacter formigenes

Oxthera

Hyperoxaluria

Oral

Phase 3 – BLA; Orphan Drug; 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/ BristolMyers Squibb

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

pegcetacoplan

Apellis

Dry AMD

Intravitreal

Phase 3 – NDA; Fast Track

TBD

pegzilarginase

Aeglea

Arginase 1 deficiency

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

TBD

pemafibrate

Kowa

Dyslipidemia/ hypercholesterolemia

Oral

Phase 3 – NDA

TBD

34 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

perfluorohexyloctane

Bausch

Dry eye disease (associated with meibomian gland dysfunction)

Ophthalmic

Phase 3 – NDA

TBD

plinabulin

Beyondspring

NSCLC

Phase 3 – NDA

TBD

plonmarlimab

I-Mab

COVID-19

Phase 3 – BLA

TBD

pollinex quattro grass

Allergy Therapeutics/ Allergic rhinitis GlaxoSmithKline

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

Paroxysmal nocturnal hemoglobinuria; 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

rabies monoclonal antibody cocktail

Sanofi/Janssen

Rabies treatment

Phase 3 – BLA; Fast Track

TBD

ranibizumab (biosimilar to Genentech’s Lucentis)

Stada Arzneimittel/ Bausch

Wet AMD

Intravitreal

Phase 3 – BLA

TBD

rapamycin

Timber

Tuberous sclerosis complex-associated facial angiofibromas

Topical

Phase 3 – NDA

TBD

rapamycin (high-strength)

Palvella

Pachyonychia congenita

Topical

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

regdanvimab

Celltrion

COVID-19

Phase 3 – BLA

TBD

relacorilant

Corcept

Cushing’s syndrome

Oral

Phase 3 – NDA; Orphan Drug

TBD

reproxalap

Aldeyra

Allergic conjunctivitis; Dry eye disease

Ophthalmic

Phase 3 – NDA

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

rilzabrutinib

Principia

ITP

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

ritlecitinib

Pfizer

Alopecia areata

Oral

Phase 3 – NDA; Breakthrough Therapy

TBD

rituximab (biosimilar to Genentech’s Rituxan)

Archigen

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

Phase 3 – BLA

TBD

rivipansel

Glycomimetics

SCD

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

TBD

roflumilast

Arcutis/AstraZeneca

Atopic dermatitis

Topical

Phase 3 – NDA

TBD

rogaratinib

Bayer

Bladder cancer

Oral

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

35 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

rozanolixizumab

UCB

Myasthenia gravis

Phase 3 – BLA; Orphan Drug

TBD

RSV vaccine (JNJ-64400141)

Janssen

RSV infection prevention

Phase 3 – BLA; Breakthrough Therapy

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

seladelpar

Cymabay/Janssen

Primary biliary cholangitis Oral

Phase 3 – NDA; Breakthrough Therapy; Orphan Drug

TBD

seltorexant

Janssen

MDD

Oral

Phase 3 – NDA

TBD

sepofarsen

Proqr

Leber’s congenital amaurosis

Intravitreal

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

sofpironium

Brickell

Axillary hyperhidrosis

Topical

Phase 3 – NDA

TBD

sotatercept

Acceleron/ Bristol-Myers Squibb

PAH

Phase 3 – BLA; Breakthrough Therapy; Orphan Drug

TBD

sotrovimab

Vir

COVID-19

Phase 3 – BLA

TBD

sparsentan

Travere/ Bristol-Myers Squibb

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

Oral

Phase 3 – NDA; Orphan Drug

TBD

SPK-8011

Spark

Hemophilia A

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

Dry eye disease

Ophthalmic

Phase 3 – BLA

TBD

tapinarof

Roivant

Atopic dermatitis

Topical

Phase 3 – NDA

TBD

tecarfarin

Espero

Anticoagulation

Oral

Phase 3 – NDA

TBD

timbetasin

Regentree

Dry eye disease

Ophthalmic

Phase 3 – BLA

TBD

timbetasin

Regentree

Neurotrophic keratopathy

Topical

Phase 3 – BLA; Orphan Drug

TBD

tiragolumab

Genentech

NSCLC; SCLC

Phase 3 – BLA; Breakthrough Therapy

TBD

tirzepatide

Eli Lilly

Obesity

Phase 3 – NDA

TBD

tislelizumab

Beigene/Novartis

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®)

Bio-Thera

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

36 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

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 – 505(b)(2) NDA

TBD

trofinetide

Acadia

Rett syndrome

Oral

Phase 3 – NDA; Fast Track; Orphan Drug

TBD

tusamitamab ravtansine

Sanofi

NSCLC (2nd-line, 3rd-line) IV

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Amgen

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Formycon

PSO

IV, SC

Phase 3 – BLA

TBD

ustekinumab (biosimilar to Janssen’s Stelara)

Hikma

PSO

IV, SC

Phase 3 – BLA

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

Dry eye disease

Phase 3 – NDA

TBD

vonoprazan

Phathom

Esophagitis (in combination Oral with amoxicillin ± clarithromycin)

Ophthalmic

Phase 3 – NDA

TBD

wilfactin

LFB

Von Willebrand disease

Phase 3 – BLA; Orphan Drug

TBD

zavegepant

Biohaven/ Bristol-Myers Squibb

COVID-19; Migraine treatment & 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

zolmitriptan microneedle system

Zosano

Migraine treatment; Cluster headache treament

Transdermal

Phase 3 – 505(b)(2) NDA

TBD

zuranolone

Sage

MDD

Oral

Phase 3 – NDA; Breakthrough Therapy; Fast Track

TBD

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

Viatris/Momenta

Hidradenitis suppurativa; Uveitis

Phase 3 – sBLA

TBD

anakinra (Kineret®)

Swedish Orphan Biovitrum

COVID-19

Phase 3 – sBLA

TBD

baricitinib (Olumiant)

Eli Lilly

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

Oral

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

TBD

37 | MAGELLANRX.COM

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

benralizumab (Fasenra)

AstraZeneca

ANCA-associated vasculitis; Bullous pemphigoid; Esophagitis

Phase 3 – sBLA; Orphan Drug

TBD

cariprazine (Vraylar®)

Allergan

MDD (adjunct)

Oral

Phase 3 – sNDA

TBD

dupilumab (Dupixent®)

Sanofi

Allergic fungal rhinosinusitis; Bullous Pemphigoid; COPD; Eosinophilic esophagitis; Pruritus; Urticaria

Phase 3 – sBLA; Breakthrough Therapy; Orphan Drug

TBD

efgartigimod alfa-fcab (Vyvgart™)

Argenx

ITP

Phase 3 – sBLA; Orphan Drug

TBD

empagliflozin (Jardiance)

Boehringer Ingelheim

Diabetic nephropathy

Oral

Phase 3 – sNDA

TBD

eptacog alfa (Novoseven®)

Novo Nordisk

Factor VIII intolerance

Phase 3 – sBLA

TBD

ferric carboxymaltose (Injectafer®)

Daiichi Sankyo

Anemia in heart failure; Anemia due to cytotoxic chemotherapy; Restless leg syndrome

Phase 3 – sNDA

TBD

ferric derisomaltose (Monoferric®)

Pharmacosmos

Anemia in heart failure

Phase 3 – sNDA

TBD

fostamatinib (Tavalisse®)

Rigel

Autoimmune hemolytic anemia

Oral

Phase 3 – sNDA; Fast Track; Orphan Drug

TBD

hydrogen peroxide (Eskata®)

Aclaris

Warts

Topical

Phase 3 – sNDA

TBD

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

Octapharma

Dermatomyositis

Phase 3 – sBLA; Orphan Drug

TBD

inebilizumab-cdon (Uplizna®)

Horizon

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

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

ranibizumab for intravitreal Genentech implant (Susvimo™)

DME; Diabetic retinopathy

Intravitreal

Phase 3 – sBLA

TBD

ravulizumab-cwvz (Ultomiris)

COVID-19; HSCT-TMA; Neuromyelitis optica (Devic’s syndrome)

Phase 3 – sBLA

TBD

38 | MAGELLANRX.COM

Alexion

PIPELINE DRUG LIST continued NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

risankizumab-rzaa (Skyrizi)

Abbvie

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

Phase 3 – sBLA

TBD

tezepelumab-ekko (Tezspire™)

AstraZeneca

Nasal polyposis

Phase 3 – sBLA

TBD

ticagrelor (Brilinta)

AstraZeneca

SCD

Oral

Phase 3 – sNDA

TBD

tisagenlecleucel-t (Kymriah)

Novartis

DLBCL (1st relapse)

Phase 3 sBLA; Orphan Drug

TBD

tocilizumab (Actemra)

Genentech

COVID-19

Phase 3 – sBLA

TBD

upadacitinib (Rinvoq)

Abbvie

CD; Giant cell arteritis

Oral

Phase 3 NDA

TBD

Beigene

CLL/SLL

Oral

Phase 3 sNDA; Orphan Drug

TBD

zanubrutinib (Brukinsa ) ®

–

– –

Complete Response Letter (CRL)/Withdrawn Drugs NAME

MANUFACTURER

DOSAGE FORM

CLINICAL USE

APPROVAL STATUS

FDA APPROVAL

budesonide oral suspension

Takeda

Eosinophilic esophagitis

Oral

CRL

TBD

buprenorphine ER

Braeburn

Opioid use disorder (moderate to severe)

CRL

TBD

gefapixant

Merck

Chronic cough

Oral

CRL

TBD

insulin aspart (biosimilar to Novo Nordisk’s Novolog)

Viatris/Biocon

T1DM; T2DM

CRL

TBD

omidenepag isopropyl

Santen

Glaucoma/ocular hypertension

Ophthalmic

CRL

TBD

phenylephrine/ tropicamide

Eyenovia

Pharmacologic mydriasis

Ophthalmic

CRL

TBD

plinabulin

Beyondspring

Chemotherapy-induced neutropenia prevention

CRL

TBD

sodium thiosulfate

Fennec

Chemotherapy-induced ototoxicity prevention

CRL

TBD

somatrogon

Opko/Pfizer

Growth hormone deficiency (pediatrics)

CRL

TBD

tanezumab

Pfizer

Osteoarthritis pain

CRL

TBD

39 | MAGELLANRX.COM

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 CNS Central Nervous System

ACR70 American College of Rheumatology 70% Improvement

COPD Chronic Obstructive Pulmonary Disease

ADC Antibody-Drug Conjugate

CRC Colorectal Cancer

ADHD Attention Deficit Hyperactivity Disorder

CRL Complete Response Letter

ADL Activities of Daily Living

CRR Complete Response Rate

AED Anti-Epileptic Drug

CSF Colony Stimulating Factor

ALK Anaplastic Lymphoma Kinase

CV Cardiovascular

ALL Acute Lymphoblastic Leukemia

CVD Cardiovascular Disease

ALS Amyotrophic Lateral Sclerosis ALT Alanine Transaminase

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

AMD Age-Related Macular Degeneration

DEA Drug Enforcement Administration

AML Acute Myeloid Leukemia

DLBCL Diffuse Large B Cell Lymphoma

ANCA Antineutrophil Cytoplasmic Antibodies

DMARD Disease Modifying Antirheumatic Drug

ANDA Abbreviated New Drug Application

DMD Duchenne Muscular Dystrophy

ARB Angiotensin II Receptor Blocker

DME Diabetic Macula Edema

ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor

DMT Disease Modifying Therapy

ART Antiretroviral Therapy

DOR Duration of Response

ARV Antiretroviral

DPI Dry Powder for Inhalation

AS Ankylosing Spondylitis

DPP-4 Dipeptidyl Peptidase 4

ASCVD Atherosclerotic Cardiovascular Disease

DR Delayed-Release

AST Aspartate Aminotransferase BCVA Best Corrected Visual Acuity

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

BLA Biologics License Application

ECOG Eastern Cooperative Oncology Group

BMI Body Mass Index

EDSS Expanded Disability Status Scale

BSA Body Surface Area

eGFR estimated Glomerular Filtration Rate

BsUFA Biosimilar User Fee Act

EGFR Epidermal Growth Factor Receptor

CABP Community Acquired Bacterial Pneumonia

ER Extended-Release

CAP Community Acquired Pneumonia

ESRD End-Stage Renal Disease

CAR T Chimeric Antigen Receptor T Cell

EUA Emergency Use Authorization

CD Crohn's Disease

FDA Food and Drug Administration

CDC Centers for Disease Control and Prevention

FH Familial Hypercholesterolemia

40 | MAGELLANRX.COM

COVID-19 Coronavirus Disease 2019

DNA Deoxyribonucleic Acid

GLOSSARY continued FLT3 FMS-Like Tyrosine Kinase-3

LVEF Left Ventricular Ejection Fraction

G-CSF Granulocyte Colony Stimulating Factor

mAb Monoclonal Antibody

GI Gastrointestinal

MACE Major Adverse Cardiovascular Events

GIST Gastrointestinal Stromal Tumor

MADRS Montgomery – Åsberg Depression Rating Scale

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

MAOI Monoamine Oxidase Inhibitor

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

MDD Major Depressive Disorder

GVHD Graft Versus Host Disease

MRI Magnetic Resonance Imaging

H Half HAART Highly Active Antiretroviral Therapy HAM-D Hamilton Depression Rating Scale HAP Healthcare-Associated Pneumonia Hb Hemoglobin HbA1c Hemoglobin A1c HCC Hepatocellular Carcinoma

MDI Metered Dose Inhaler MRSA Methicillin-Resistant Staphylococcus Aureus MS Multiple Sclerosis N/A Not Applicable NASH Non-Alcoholic Steatohepatitis NDA New Drug Application NHL Non-Hodgkin Lymphoma

HCP Healthcare Professional

NIAID National Institute of Allergy and Infectious Diseases

HCV Hepatitis C Virus

NSAID Non-Steroidal Anti-Inflammatory Drug

HER Human Epidermal Growth Factor Receptor

NSCLC Non-Small Cell Lung Cancer

HER2 Human Epidermal Growth Factor Receptor 2

NYHA New York Heart Association

HF Heart Failure

ODT Orally Disintegrating Tablet

HFA Hydrofluoroalkane

OR Odds Ratio

HIT Heparin Induced Thrombocytopenia

ORR Overall/Objective Response Rate

HIV Human Immunodeficiency Virus

OS Overall Survival

HIV-1 Human Immunodeficiency Virus-1

OTC Over-the-Counter

HPV Human Papilloma Virus

PAH Pulmonary Arterial Hypertension

HR Hazard Ratio

PARP Poly(ADP-ribose) Polymerase

HSCT Hematopoietic Stem Cell Transplant

PASI Psoriasis Area and Severity Index

HTN Hypertension

PASI 50 Psoriasis Area and Severity Index 50%

IBS Irritable Bowel Syndrome

PASI 75 Psoriasis Area and Severity Index 75%

IBS-C Irritable Bowel Syndrome, Constipation Predominant

PASI 90 Psoriasis Area and Severity Index 90%

IGA Investigator's Global Assessment

PCI Percutaneous Coronary Intervention

IM Intramuscular IRB Internal Review Board ITP Immune Thrombocytopenic Purpura ITT Intent-To-Treat IV Intravenous JIA Juvenile Idiopathic Arthritis LDL Low-Density Lipoprotein LDL-C Low-Density Lipoprotein Cholesterol

41 | MAGELLANRX.COM

PASI 100 Psoriasis Area and Severity Index 100% 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 PsA Psoriatic Arthritis

GLOSSARY continued PSO Plaque Psoriasis

SL Sublingual

PTCA Percutaneous Transluminal Coronary Angioplasty

SLE Systemic Lupus Erythematosus

PTSD Post-Traumatic Stress Disorder Q Quarter

SLL Small Lymphocytic Lymphoma sNDA supplemental New Drug Application

QIDP Qualified Infectious Diseases Product

SNRI Serotonin and Norepinephrine Reuptake Inhibitor

QOL Quality of Life

SOC Standard of Care

R/R Relapsed or Refractory

sPGA static Physician Global Assessment

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

SR Sustained-Release

RA Rheumatoid Arthritis RBC Red Blood Cell RCC Renal Cell Carcinoma REMS Risk Evaluation and Mitigation Strategy RMAT Regenerative Medicine Advanced Therapy RNA Ribonucleic Acid RRR Relative Risk Reduction RSV Respiratory Syncytial Virus RTOR Real-Time Oncology Review RVO Retinal Vein Occlusion sBLA supplemental Biologics License Application SARS-CoV-2 Severe Acute Respiratory SyndromeAssociated Coronavirus-2

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 US United States UTI Urinary Tract Infection

SC Subcutaneous

VAS Visual Analog Scale

SCCHN Squamous Cell Cancer of the Head and Neck

VEGF Vascular Endothelial Growth Factor

SCD Sickle Cell Disease

VTE Venous Thromboembolism

SCLC Small Cell Lung Cancer

WBC White Blood Cell

SCT Stem Cell Transplant

WHO World Health Organization

SGLT2 Sodium-Glucose Co-Transporter 2

XR Extended-Release

42 | MAGELLANRX.COM

MRx PIPELINE A VIEW INTO UPCOMING SPECIALTY & TRADITIONAL DRUGS

JANUARY 2022