Quarterly Drug Pipeline: January 2024

Clinical insights and competitive intelligence on anticipated drugs in development

Drug Pipeline

Clinical insights and competitive intelligence on anticipated drugs in development

Editor-in-chief's message

Welcome to the inaugural Prime Therapeutics Quarterly Drug Pipeline! Dive into clinical insights and competitive intelligence on anticipated drugs in development, so you are well sourced on the drug pipeline.

Methodology

The drug pipeline is complex and fluid. Our talented and committed team of clinical and analytics experts is excited to bring you this robust publication after thoughtful research. Specialty and traditional drugs that are covered under the pharmacy and medical benefits are featured. New molecular entities, pertinent new and expanded indications for existing medications, biosimilars and regenerative medicines, such as gene and cellular therapies, are also profiled.

Quarterly Pipeline details both agents submitted for FDA review and those in phase 3 study with a likelihood of being applied to the FDA. Our deep dives consider the evidence – the products’ potential to fill an unmet need or become the new standard of care and the ability to replace existing therapies.

A market-agnostic financial forecast primarily from EvaluateTM is included for select agents to assist payers with assessing the potential budgetary impact of the pipeline. Five-year projected annual U.S. sales are forecasted.

Reflection

The FDA’s 10-year novel drug approval average is 46 per year. In 2023, the FDA approved 55 novel drugs. This is over a third more than in 2022. More than half of novel approvals in 2023 were for rare or orphan diseases. Moreover, 65% of novel approvals underwent at least one of the Agency’s expedited programs to speed approval for serious conditions, with 16% as accelerated approval. This is a designation which can be challenging for payors to manage, since approval is based on a surrogate endpoint thought to predict clinical benefit. While numbers do not tell the entire story, they represent innovation for patient care and have the potential to advance health for the American public.

On the horizon

The FDA decisions for specialty medications (75%) and for Orphan Drugs (36%) continue to grow for agents with applications submitted to the FDA. One agent is seeking FDA’s Accelerated Approval.

First quarter 2024 may usher in first-time approvals. These include:

First FDA-approved treatment for NASH
New indication for Wegovy® for secondary CVD prevention in overweight/obesity
First-in-class agent that may target the underlying cause of PAH
First indication for a monoclonal antibody for food allergies
One-time gene therapies for two rare genetic conditions
First once-weekly basal insulin
First self-administered intranasal flu vaccine

We hope you enjoy the report!

Maryam Tabatabai
Vice President, Clinical Information

Editorial team

Maryam Tabatabai
Editor-In-Chief
Vice President, Clinical Information

Carole Kerzic, RPh
Executive Editor
Clinical Pharmacist, Drug Information

Nicole Kjesbo, PharmD, BCPS
Executive Editor
Clinical Program Direcotr, Pipeline

Andrea Henry, PharmD, MBA, BCPS
Specialty Drug Information Pharmacist

Katie Lockhart
Senior Manager, Analytics

Olivia Pane, PharmD, CDCES
Clinical Pharmacist, Drug Information

Michelle E. Pannone-Booth, PharmD
Senior Director, Clinical Account Services

The drug pipeline is fluid; the dates and information within this publication are subject to change. Nothing herein is or shall be construed as a promise or representation regarding past or future events and Prime Therapeutics 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 Prime Therapeutics.

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 drug names appear in magenta throughout the publication.

atidarsagene autotemcel IV

Manufacturer: Orchard

Proposed indications

Early onset metachromatic leukodystrophy (MLD)

Clinical overview

Mechanism of action
Atidarsagene autotemcel (arsa-cel) is an ex vivo autologous hematopoietic stem cell-based gene therapy that uses a lentiviral vector (LVV) to encode a corrected arylsulfatase-A (ARSA) gene into a patient’s own stem cells, allowing the edited cells to produce a functional version of the ARSA enzyme.

Clinical trials
In two phase 1/2 open-label, single-arm studies and one expanded access framework, the efficacy and safety of arsa-cel was evaluated in an integrated analysis of 39 patients compared to the natural course of disease in 49 untreated patients. The median follow-up in the integrated analysis was 6.76 years (range, 0.64 to 12.91). The composite endpoint from an integrated analysis was severe motor impairment-free survival (sMFS) (defined as the time measured from a patient’s birth to their first loss of locomotion or sitting without support or their death). Patients treated with arsa-cel had statistically significant and clinically meaningful improvement in sMFS compared to natural course of disease in the pre-symptomatic late-infantile (LI) subgroup (p<0.001) and early juvenile (EJ) MLD subgroup (p=0.042), (p=0.042) subgroups, as well as early symptomatic EJ MLD subgroups. Most patients with pre-symptomatic LI who received arsa-cel maintained the ability to walk compared to patients in the untreated LI group, who lost all ability to move by a median age of 2.6 years. In addition, most surviving patients who received arsa-cel who had pre-symptomatic EJ maintained the ability to walk (with performance normal for age) and patients with early symptomatic EJ maintained the ability to sit unassisted and/or crawl or roll compared to patients in the untreated EJ group, who all lost the ability to move by a median age of 6.4 years. Treated patients continued to have preserved motor and cognitive function through 12 years of follow-up (median, 6.76 years). Arsa-cel was well-tolerated, with no serious treatment-related adverse events and no reports of malignancy or insertional oncogenesis. Patients experienced febrile neutropenia, primarily due to busulfan conditioning. Three deaths unrelated to treatment were reported. Anti-ARSA antibodies developed in six patients that subsequently resolved with no observed impact on clinical outcomes.

Dosage and administration
Arsa-cel is given as a one-time IV infusion. Stem and progenitor cells are retrieved through mobilization and apheresis. Functional ARSA genes are then inserted into CD34+ cells outside the body using an LVV. Finally, treated cells are reinfused back into the patient. Treatment also requires myeloablation of the bone marrow and conditioning with busulfan prior to reinfusion of the engineered cells.

Place in therapy

MLD is a rare, autosomal recessive, genetic, lysosomal storage disorder. It is caused by mutations in the ARSA gene, affecting the production of the enzyme ARSA. This results in the body producing insufficient amounts of the ARSA enzyme, leading to accumulation of fatty substances (sulfatides), which are toxic to the brain and white matter (or myelin) of the nervous system, causing progressive loss of motor and cognitive function and ultimately death. Symptoms can include difficulty with speech, swallowing, and walking, as well as seizures and changes in behavior and personality. There are three general categories of MLD based on the age of onset (prevalence): late infantile (LI) (50% to 60%), juvenile (20% to 30%; subdivided into early and late juvenile), and adult (10%). Although commonly misdiagnosed, it is usually identified around the ages of 2 to 4 years. Early-onset forms of MLD are rapidly progressive. Patients usually succumb to the disease by 5 years of age for the infantile form, within 10 to 20 years following symptom onset of the juvenile form, and within 6 to 14 years with the adult onset form.

The exact prevalence of MLD is unknown but is estimated to range from 1 in 40,000 to 1 in 160,000 worldwide (about 3,600 babies born annually); the incidence is estimated at 1 case in 40,000 births in the U.S. The prevalence rate is higher in the Navajo Nation (about 1 in 2,500) and is more common among certain Middle Eastern populations compared to the general population.

MLD has no cure, and currently there are no FDA-approved treatments for the disorder. In some patients with MLD, a stem cell transplantation can be considered in pre- or minimally-symptomatic children to delay the progression of disease. Otherwise, treatment is supportive and focuses on symptomatic relief. If approved, arsa-cel will be the first and only treatment available for patients with early-onset MLD.

Arsa-cel is also being evaluated in patients with late juvenile MLD (phase 3; primary completion 2025). Notably, top-line results for Takeda’s recombinant human ARSA enzyme, TAK-611 (formerly SHP611 and HGT-1111), which is administered intrathecally, did not meet the primary and secondary endpoints for LI MLD. In addition, the replication-deficient adeno-associated virus gene transfer vector serotype rh.10 expressing a human ARSA copy DNA (AVVrh.10cuARSA) via intracerebral administration is being studied outside the U.S. for early-onset MLD.

FDA approval timeline

March 18, 2024

FDA designations: Orphan Drug, Priority Review, RPD, RMAT

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected total U.S. sales	$11	$32	$59	$89	$123

ensifentrine inhaled

Manufacturer: Verona

Proposed indications

Chronic obstructive pulmonary disease (COPD)

Clinical overview

Mechanism of action
Ensifentrine is a selective, dual inhibitor of phosphodiesterase 3 (PDE3) and phosphodiesterase 4 (PDE4) that has a combined effect on airway inflammation, bronchodilation, and ciliary beat frequency in bronchial epithelial cells.

Clinical trials
Ensifentrine was evaluated in the randomized, double-blind, parallel-group, placebo-controlled ENHANCE-1 (NCT04535986; n=763) and ENHANCE-2 (NCT04542057; n=790) trials in patients 40 to 80 years of age with moderate to severe symptomatic COPD. Eligible patients had post-bronchodilator FEV1 of 30% to 70% predicted normal, FEV1/FVC < 0.7, a modified Medical Research Council dyspnea scale score ≥ 2, and a smoking history of ≥ 10 pack-years. Enrolled patients either were on no maintenance/background therapy or were on stable maintenance LAMA or LABA therapy with or without ICS. In both trials, ensifentrine met the primary endpoint, demonstrating significant improvement from baseline to week 12 in average 12-hour FEV1 area-under-the-curve (AUC0–12 h) compared to placebo (ENHANCE-1: 87 mL; ENHANCE-2: 94 mL; p< 0.001 for both). The rate of moderate or severe exacerbations, a secondary endpoint, also decreased with ensifentrine compared to placebo over the 24-week treatment period (ENHANCE-1: rate ratio, 0.64 [p=0.05]; ENHANCE-2: rate ratio, 0.57 [p=0.009]). Respiratory symptoms and QOL were also significantly improved at week 24 with ensifentrine compared to placebo in ENHANCE-1 but not in ENHANCE-2. Hypertension was reported more often with ensifentrine compared to placebo (ENHANCE-1: 2.5% versus 1.4%, respectively; ENHANCE-2: 1% versus 0.3%, respectively).

Dosage and administration
Ensifentrine 3 mg is administered via inhalation twice daily using a nebulizer.

FDA approval timeline

Approximately 15.7 million people in the U.S. are diagnosed with COPD. COPD is characterized by chronic respiratory symptoms (e.g., dyspnea, cough, sputum production, and/or exacerbations) due to airway (bronchitis/bronchiolitis) and/or alveoli (emphysema) abnormalities that cause persistent, often progressive, airflow obstruction.

Cyclic adenosine monophosphate (cAMP) plays a key role in the functions of many cells of the airway. The PDE3 and PDE4 enzymes are highly expressed in airway smooth muscle, where they hydrolyze (inactivate) cAMP. PDE4 is also present in airway epithelial cells and inflammatory cells implicated in COPD. Inhibition of PDE3 and PDE4 enhances cAMP activity, thereby leading to bronchial smooth muscle relaxation, decreased inflammation, downregulation of neutrophils and macrophages, improved airway clearance, and inhibition of bronchial wall remodeling.

The Global Initiative for Chronic Obstructive Lung Disease (GOLD) guidelines recommend that adding the once-daily oral PDE4 inhibitor roflumilast (Daliresp®, generics) to long-acting bronchodilator therapy (with or without an ICS) may be considered in patients with severe to very severe airflow limitation, chronic bronchitis, and exacerbations. The roflumilast prescribing information reports a rate ratio reduction in moderate or severe COPD exacerbations of 0.82 and 0.85 in two placebo-controlled clinical trials.

If approved, ensifentrine will be the first dual PDE3/PDE4 inhibitor indicated for COPD. In clinical trials, it was well-tolerated and improved FEV1 when used as monotherapy or when added to LAMA or LABA therapy. Ensifentrine’s dual mechanism of action distinguishes it from roflumilast; however, these agents have not been compared in a head-to-head trial. In addition, oral roflumilast is associated with diarrhea, nausea, and weight loss (rate in clinical trials < 10%). The inhalation route of ensifentrine appears to mitigate these side effects.

Ensifentrine is also in phase 2 studies for asthma and cystic fibrosis.

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected total U.S. sales	$36	$121	$259	$442	$631

fidanacogene elaparvovec IV

Manufacturer: Pfizer/Genentech

Proposed indications

Hemophilia B in adults

Clinical overview

Mechanism of action

Fidanacogene elaparvovec is a gene therapy that contains a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-specific promoter, and factor IX Padua (FIX–R338L) transgene. Treatment with fidanacogene elaparvovec may enable patients with hemophilia B to produce adequate levels of factor IX (FIX) to avoid bleeds without regular infusions of exogenous FIX.

Clinical trials
The ongoing, open-label, single-arm, phase 3, BENEGENE-2 (NCT03587116) study is evaluating fidanacogene elaparvovec in adult males with moderately severe to severe hemophilia B, defined as having ≤ 2% of normal FIX activity. Enrollees completed ≥ 6 months of routine FIX prophylaxis during the trial’s lead-in period. Interim analysis of data from 45 males demonstrated superiority of fidanacogene elaparvovec compared to prophylactic exogenous FIX, based on the primary endpoint of annualized bleeding rate (ABR). Fidanacogene elaparvovec led to a 71% reduction in ABR, measured from 12 weeks to 15 months after the dose compared to the ABR reported during the lead-in pre-treatment period (ABR, 1.3 versus 4.43, respectively; p<0.0001). In addition, key secondary endpoints revealed a 78% reduction in treated ABR (p=0.0001) and a 92% reduction in annualized infusion rate (p<0.0001). Moreover, the mean FIX activity was 27% at 15 months and 25% at 24 months. No deaths were reported. Two serious TEAEs were reported, a duodenal ulcer hemorrhage in a patient receiving corticosteroids, and an immune-mediated liver aminotransferase elevation. No serious infusion reactions, thrombotic events, or FIX inhibitors were reported.

In the ongoing, open-label, phase 1/2 GOLD-B (NCT02484092) trial, all 15 patients who received fidanacogene elaparvovec discontinued routine infusions of FIX concentrates at a cumulative follow-up of over 18 patient years of observation (5 to 121 weeks). No participant experienced a serious adverse event or thrombotic event. In addition, FIX inhibitors were not detected.

Dosage and administration

Fidanacogene elaparvovec was administered via IV infusion as a single dose of 5e11 vector genomes per kilogram of body weight (vg/kg).

Place in therapy

Hemophilia B is an X-linked bleeding disorder caused by mutations in the FIX gene. The majority of cases are found in males (1 in 25,000 male births). However, females who carry the gene may exhibit symptoms that are usually mild. Hemophilia B severity is dependent on the FIX levels. FIX levels between 5% and 40% of normal are indicative of mild disease, FIX levels between 1% to 5% of normal indicate moderate disease, and FIX levels < 1% of normal are associated with severe disease. Patients with moderate to severe hemophilia B experience recurrent painful spontaneous bleeding into joint space and muscles. GI, kidney and CNS bleeding may also occur. Long-term damage, including arthropathy, muscle weakness, permanent neurologic damage and/or death, may occur if left untreated.

The SOC for hemophilia B is exogenous FIX infused IV as on-demand treatment for an acute bleeding episode and infused 1 to 3 times per week to prevent bleeding. Several FIX products are available; due to a lower risk of pathogen transmission, recombinant products are preferred over products derived from human plasma. The plasma derived activated prothrombin complex (Feiba®) is also indicated for control and prevention of bleeding associated with hemophilia B with inhibitors. It is administered IV every other day for routine prophylaxis and on-demand for acute treatment. In addition, the gene therapy, etranacogene dezaparvovec-drlb (Hemgenix®) was FDA approved in 2022 for use in select adults with severe congenital hemophilia B.

If approved, fidanacogene elaparvovec will be the second gene therapy for hemophilia B and will compete with Hemgenix. Both gene therapies deliver a FIX Padua transgene and are intended to be given as a single dose in a patient’s lifetime. Durability of response to etranacogene dezaparvovec-drlb has been demonstrated for up to 3 years after the dose (94% of patients were free from continuous FIX prophylaxis) and no serious TEAEs were reported. Duration of response longer than 15 months is not yet available for fidanacogene elaparvovec.

FDA approval timeline

April to June 2024

FDA designations: Breakthrough Therapy, Orphan Drug, RMAT

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected total U.S. sales	$44	$85	$128	$158	$183

insulin icodec SC

Manufacturer: Novo Nordisk

Proposed indications

Type 1 diabetes mellitus (T1DM) and type 2 diabetes mellitus (T2DM)

Clinical overview

Mechanism of action
Insulin icodec is a long-acting basal insulin with a half-life of approximately 1 week.

Clinical trials
Insulin icodec has been studied in the randomized, multicenter, phase 3 ONWARDS clinical trial program with comparisons to available insulins. Estimated treatment differences (ETDs) were based on the primary endpoint of change in HbA1c from baseline. Trials enrolled adults with T1DM or T2DM and HbA1c ranging from 7% to 11%.

ONWARDS 1 (open-label): Insulin icodec demonstrated noninferiority and superiority to once-daily insulin glargine 100 units/mL in 984 insulin-naïve adults with T2DM (ETD, -0.19 percentage points; p<0.001 for noninferiority; p=0.02 for superiority) at 52 weeks. Icodec and glargine were given concurrently with non-insulin antidiabetic treatment. The rates of combined clinically significant or severe hypoglycemia were 0.3 and 0.16 events per person-year of exposure with icodec and glargine 100 units/mL respectively, at week 52.

ONWARDS 2 (open-label): Insulin icodec demonstrated noninferiority and superiority to once-daily insulin degludec in 526 adults with T2DM uncontrolled on basal insulin (ETD, -0.22 percentage points; p<0.0001 for noninferiority; p=0.0028 for superiority) at 26 weeks. The incidence of hypoglycemia was low in both groups. The incidence of significant or severe hypoglycemia was numerically higher with icodec but did not reach statistical significance compared to degludec.

ONWARDS 3 (double-blind): Insulin icodec demonstrated non-inferiority and superiority to once-daily insulin degludec in 588 insulin-naïve adults with T2DM (ETD, −0.2 percentage points; p<0.001 for noninferiority; p=0.002 for superiority) at 26 weeks. A significantly higher incidence of combined significant or severe hypoglycemia was reported with icodec compared to degludec (0.35 versus 0.12 events per patient-year exposure; p=0.01) through week 26.

ONWARDS 4 (open-label): Insulin icodec demonstrated non-inferiority to once-daily insulin glargine 100 units/mL in 582 adults with long-standing T2DM on a basal-bolus insulin regimen (ETD, +0.02 percentage points; p<0.0001) at 26 weeks, when combined with 2 to 4 daily injections of insulin aspart. The combined incidence of significant or severe hypoglycemia was similar between the groups.

ONWARDS 5 (open-label with real-world elements): Insulin icodec titrated with a dosing guide application (app) was superior to once-daily basal insulin analogs (degludec, glargine 100 units/mL, glargine 300 units/mL) dosed per standard practice in 1,085 insulin-naïve adults with T2DM. At week 52, the estimated mean change in HbA1c from baseline with icodec was -1.68 percentage points compared to -1.31 percentage points with once-daily insulin analogs (p=0.009 for superiority). The combined incidence of significant or severe hypoglycemia was low in both groups.

ONWARDS 6 (open-label): Insulin icodec demonstrated non-inferiority to once-daily insulin degludec in 582 adults with T1DM (ETD, +0.05 percentage points; p=0.0065 for noninferiority) at 26 weeks, when each were used in adjunct to mealtime insulin aspart 100 units/mL. The incidence of significant or severe hypoglycemia was statistically significantly higher with icodec than degludec (19.9 versus 10.4 events per patient-year of exposure; p<0.0001).

Dosage and administration
Insulin icodec is administered as a SC injection once weekly, with dosing based on glycemic need.

Place in therapy

It is estimated that 38.4 million Americans have diabetes mellitus (DM). DM is responsible for increased morbidity and mortality. Adequate glycemic control is essential to minimize microvascular and macrovascular complications. The American Diabetes Association recommends that glycemic goals be tailored to individual patient needs. They advise that a reasonable HbA1c goal is < 7% for nonpregnant adults without significant hypoglycemia. Multiple insulin products are available as insulin replacement therapy. Patients with T1DM require either multiple daily insulin injections of prandial and basal insulin or continuous SC insulin infusion. Exogenous insulin may be necessary in patients with T2DM when glycemic goals are not met with non-insulin antidiabetic agents.

If approved, insulin icodec will be the first once-weekly basal insulin available for patients with T1DM or T2DM in the U.S. In clinical trials, it was shown to be noninferior in terms of HbA1c lowering to once-daily insulin degludec for the management of T1DM and superior to once-daily insulin glargine 100 units/mL and insulin degludec for T2DM, but may increase the incidence of significant or severe hypoglycemia. Once weekly insulin icodec could lessen treatment burden and has the potential to improve insulin adherence in select patients who require exogenous insulin.

FDA approval timeline

April 2024

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected total U.S. sales	$88	$180	$314	$459	$555

marnetegragene autotemcel IV

Manufacturer: Rocket

Proposed indications

Severe leukocyte adhesion deficiency type 1 (LAD-1)

Clinical overview

Mechanism of action
Marnetegragene autotemcel is a gene therapy that uses a lentiviral vector (LVV) to encode a functional copy of the integrin subunit beta 2 (ITGB2) gene into a patient’s own hematopoietic stem cells. The ITGB2 gene encodes for the cluster of differentiation 18 (CD18) subunit of β2-integrins, which allows for leukocyte migration in response to bacterial and fungal infections. Defective β2-integrin expression prevents leukocyte adherence to the endothelium and extravasation to infected areas, resulting in an insufficient innate immune response and wound healing.

Clinical trials
In a global, phase 1/2 study (NCT03812263), the efficacy and safety of marnetegragene autotemcel were evaluated in patients (n=9) with severe LAD-1, ages ≥ 3 months, who did not have availability of a medically-eligible human leukocyte antigen (HLA)-identical sibling donor transplant. The primary phase 2 endpoints were the proportion of patients alive at least one year after treatment with marnetegragene autotemcel without allogeneic HSCT and the proportion of patients alive at age 2 years without allogeneic HSCT, if they were < 1 year old at the beginning of the study. Interim analysis reported that all primary and secondary endpoints (e.g., post infusion CD18 expression, genetic correction, incidence of infection) were met. At data cutoff, marnetegragene autotemcel demonstrated a 100% survival rate at 12 months after infusion for all patients with 12 to 24 months of available follow-up. Those patients also demonstrated significant reductions in all-cause hospitalizations and severe infections. When compared to pre-treatment history, data showed restoration of wound repair capabilities and large decreases in the incidence of significant infections and skin lesions. Marnetegragene autotemcel was well tolerated, with no severe treatment related adverse events.

Dosage and administration
Treatment with marnetegragene autotemcel involves a lengthy process that includes pre-collection preparation, CD34+ hematopoietic stem and progenitor cell (HSPC) mobilization, and apheresis. This is followed by ex vivo transduction of autologous hematopoietic cells with the lentiviral vector encoding the ITGB2 gene. Patients also receive myeloablative conditioning followed by a one-time IV infusion of edited HSPCs.

Place in therapy

LAD-1 is a rare genetic disorder of the immune system (B-cell and T-cell immunodeficiency) that is estimated to impact 1 in 1,000,000 persons annually. It is caused by mutations in the ITGB2 gene that encodes for the integrin beta chain-2 protein, also known as CD18. The CD18 protein plays a key role in fighting infections by facilitating leukocyte migration to infection sites where they kill invading microbes. Pediatric patients with LAD-1 are predisposed to recurrent and fatal bacterial and fungal infections and present with infections immediately after birth. LAD-1 is an immune disorder that is invariably fatal in childhood without an allogeneic HSCT. If patients with LAD-1 survive past infancy, they suffer recurrent severe infections, such as gingival ulcers, necrotic skin ulcers, pneumonia, and septicemia; survival beyond childhood is uncommon. The hallmark sign of LAD-1 is delayed umbilical cord stump detachment and lack of pus formation at the infection site.

Currently, the only treatment option for patients with severe LAD-1 is allogeneic HSCT; however, finding a matched donor may not occur in a timely manner, and without HSCT death usually occurs by 2 years of age. Other treatments for severe LAD-1 focus on controlling and preventing the infections with the use of antimicrobial agents, as well as WBC transfusions for life-threatening infections.

If approved, marnetegragene autotemcel will be the first agent approved to treat patients with LAD-1. Marnetegragene autotemcel will provide a therapeutic option to patients who do not have an HLA-identical sibling donor or whose HLA-matched sibling donor is unable to donate stem cells (e.g., mobilization and apheresis are not feasible). The one-time gene therapy is potentially curative and will address a high unmet need. In a phase 2 clinical trial, it demonstrated a favorable safety profile, and after treatment, patients did not experience any significant disease related infections or inflammatory skin lesions.

Ustekinumab (Stelara®) is also being evaluated for LAD-1 in a phase 1/2 trial.

FDA approval timeline

March 31, 2024

FDA designations: Fast Track, Orphan Drug, Priority Review, RPD, RMAT

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected total U.S. sales	$22	$39	$78	$122	$153

respiratory syncytial virus (RSV) vaccine, mRNA-1345 IM

Manufacturer: Moderna

Proposed indications

Prevention of RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD) in adults ≥ 60 years of age

Clinical overview

Mechanism of action
mRNA-1345 is an RSV vaccine that consists of a single mRNA sequence encoding for a stabilized prefusion F glycoprotein. This protein is found on the RSV surface and allows the virus to enter the host cell.

Clinical trials

The safety and efficacy of mRNA-1345 were evaluated in the multinational, randomized, double-blinded, placebo-controlled, phase 2/3 ConquerRSV (NCT05127434) trial in adults ≥ 60 years of age. Published data reported that 35,541 adults were randomized 1:1 to receive a dose of the vaccine or placebo. At a median follow-up of 112 days, the study demonstrated a vaccine efficacy (VE) of 83.7% (p<0.0001) against RSV-LRTD with ≥ 2 symptoms and a VE of 82.4% (p=0.0078) against RSV-LRTD with ≥ 3 symptoms (co-primary endpoints). Protection against both RSV subtypes A and B was reported. mRNA-1345 was well tolerated. No safety signals were reported with the vaccine, including Guillain-Barré syndrome.

Dosage and administration
In the clinical trial, mRNA-1345 was administered as a single 50 mcg IM injection.

Place in therapy

RSV is a common contagious respiratory virus that usually causes mild, cold-like symptoms lasting 1 to 2 weeks. However, RSV can cause serious illness, particularly in infants and older adults in whom bronchiolitis and pneumonia can occur, and may require hospitalization. Older adults are at higher risk of serious illness due to weakened immunity and underlying cardiac or pulmonary conditions. In the U.S., RSV causes 60,000 to 160,000 hospitalizations and 6,000 to 10,000 deaths each year among adults ≥ 65 years of age.

Typically, the RSV season in the U.S. occurs between November and April but may vary by region. Notably, the regular RSV circulation pattern was disrupted by the COVID-19 pandemic, leading to interseasonal variability; however, the RSV circulation in the 2022-2023 season suggests a return toward prepandemic seasonality.

There is no FDA-approved medication to treat RSV infection. Treatment consists of symptom management, including antipyretics and analgesics. Notably, the RSV F protein inhibitor monoclonal antibodies, nirsevimab-alip (Beyfortus™), and palivizumab (Synagis®), are FDA-approved for the prevention of RSV-LRTD, but are only indicated for use in infants during their first RSV season and select children ≤ 24 months of age. In 2023, the FDA approved two recombinant prefusion F-based (RSVpreF) vaccines, Arexvy (GlaxoSmithKline) and Abrysvo™ (Pfizer), to prevent RSV-LRTD in adults ≥ 60 years of age. Abrysvo is also indicated for immunization of infants (≤ 6 months of age) born to pregnant persons who received the vaccine at 32 to 36 weeks gestation.

If approved, mRNA-1345 will be the first RSV vaccine that uses mRNA technology. This vaccine platform may allow for a faster manufacturing process compared to the RSVpreF vaccines, Arexvy and Abrysvo, for which mRNA-1345 will compete in the older adult population. All 3 vaccines are administered as single-dose IM injections. There are no head-to-head trials comparing the 3 vaccines. Non-comparative data in older adults revealed that VE against RSV-LRTD mid-way into a second RSV season has been demonstrated after a single dose of Abrysvo (VE, 78.6%) or Arexvy (VE, 77.3%); cumulative VE (67.2%) over 2 full seasons was also observed with Arexvy. Data over multiple RSV seasons with 1 dose of mRNA-1345 are not available; further follow-up to determine the duration of protection is ongoing. In addition, in separate phase 3 trials Guillain-Barré syndrome was reported within days of vaccination in two individuals who received Abrysvo, one individual who received Arexvy, and zero who received mRNA-1345.

The mRNA-1345 vaccine is also being evaluated in a phase 3 trial in high-risk adults (18 to < 60 years of age) and a phase 2 trial for RSV protection in infants born to mothers who were vaccinated during pregnancy.

FDA approval timeline

April 2024

FDA designations: Breakthrough Therapy, Fast Track, Priority Review

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected total U.S. sales	$60	$151	$219	$284	$370

prademagene zamikeracel topical surgical application

Manufacturer: Abeona

Proposed indications

Recessive dystrophic epidermolysis bullosa (RDEB)

Clinical overview

Mechanism of action
Prademagene zamikeracel (pz-cel) is an autologous, collagen type VII alpha 1 chain (COL7A) gene-corrected epidermal sheet. In this ex vivo cell therapy, autologous keratinocyte grafts are created from skin biopsies that are transduced with a retrovirus containing the full-length COL7A1 gene and grown into sheets.

Clinical trials
The open-label, controlled, phase 3 VIITAL (NCT04227106) study evaluated pz-cel in patients ≥ 6 years of age with RDEB and two confirmed C7 mutations. In the study, pz-cel treatment was administered to 11 patients with a total of 43 large (> 20 cm2) chronic wound pairs that had remained open for ≥ 6 months (mean, 6.2 years). Top-line data revealed that, at the 6-month assessment, a significantly greater proportion of wounds achieved ≥ 50% healing from baseline (co-primary endpoint) when treated with pz-cel (81.4%) compared to untreated control wounds (16.3%; p<0.0001). In addition, at 6 months, pz-cel resulted in significantly greater pain reduction associated with wound dressing changes compared to untreated wounds (mean pain reduction from baseline, 3.07 versus 0.9, respectively; p=0.0002; based on a 0-10 pain severity scale). Pz-cel was well tolerated. TEAEs included procedural pain, muscle spasms and pruritis. No cases of positive replication-competent retrovirus (RCR), systemic immunologic responses or squamous cell carcinoma (SCC) were reported. Patients were allowed to enroll in a long-term follow-up study.

An open-label, single-site, phase 1/2 (NCT01263379) trial included seven participants with RDEB, 18 to 45 years of age, with chronic wounds that were present for a mean of 11.2 years (range, 3 to 20 years). Each patient received six grafts with pz-cel. Patients were followed for 4 to 8 years (mean, 5.9 years). Based on investigator global assessment (IGA), at 5 years, 70% of sites treated with pz-cel achieved ≥ 50% wound healing compared to baseline. In addition, no sites with ≥ 50% wound healing were painful or pruritic, compared to 67% of sites with < 50% wound healing (p<0.001) at 5 years.

Pz-cel is also being studied in a phase 3 trial (NCT05725018) in patients with RDEB who were previously treated with pz-cel. Primary study completion is anticipated in August 2024.

Dosage and administration
In clinical trials, pz-cel was administered as a one-time surgical application on up to six chronic, RDEB wounds. Each pz-cel sheet can treat a wound area up to 40 cm2. Up to six sheets were applied to each patient, depending on the area of existing wounds. Patients received adequate anesthesia during pz-cel application.

Place in therapy

Epidermolysis bullosa (EB) is a rare, inherited, genetic skin disorder characterized by formation of painful blisters after minor friction or trauma to the skin or mucosa. EB affects an estimated 1 in every 50,000 live births. Dystrophic epidermolysis bullosa (DEB) is 1 of the 4 main types of EB. It is caused by mutation in the COL7A1 gene that is responsible for making collagen VII (C7), a protein that strengthens and stabilizes the adhesion of the epidermis and dermis. There are two major subtypes of DEB, dominant DEB (DDEB) and recessive DEB (RDEB). RDEB is typically more generalized and severe than DDEB. Common manifestations of RDEB include scarring, malnutrition, anemia, esophageal strictures, growth retardation, webbing or fusion of the fingers and toes, contractures, teeth malformation, microstomia, and corneal abrasions. Patients with severe cases are also at increased risk of aggressive squamous cell carcinoma (SCC), which typically occurs in the second decade of life and can be fatal.

There is no cure for EB. Current management for this genetic disorder includes reduction of skin trauma, nutritional support, and pain management. The gene therapy beremagene geperpavec (Vyjuvek®) was FDA approved in May 2023 for use in patients ≥ 6 months of age with DEB and confirmed COL7A1 mutation. It is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy topical gel that is applied to wounds once weekly by an HCP. In clinical trials, it resulted in complete healing of 67% of wounds compared to 22% with placebo gel (p=0.002). In addition, birch triterpene topical gel (Filsuvez®) was approved in December 2023 for the treatment of wounds associated with dystrophic and junctional EB in patients ≥ 6 months of age. The botanical product is applied at home to wound surfaces at each dressing change (every 1 to 4 days) until the wound has healed. Clinical trials demonstrated that 41.3% of patients treated with the Filsuvez achieved wound closure within 45 days compared to 28.9% who received placebo.

If approved, pz-cel will be the third treatment and second gene therapy approved for EB. Pz-cel will compete with Vyjuvek in patients with RDEB. Both gene therapies provide copies of the COL7A1 gene and are administered by an HCP. Pz-cel is intended as a one-time surgical application. It is administered with anesthesia in a hospital setting, followed by a one-week hospital stay to immobilize the grafted areas. Long-term data for pz-cel demonstrated sustained wound healing for up to 8 years after pz-cel application. In contrast, Vyjuvek is a topical, redosable gene therapy that is applied directly to EB wounds by an HCP, which may be performed in the home setting. Vyjuvek application is repeated on a weekly basis until wound closure. It is not a curative option since a wound may reopen and require retreatment.

Dabocemagene autoficel (D-Fi), an autologous dermal fibroblast genetically modified with a full-length COL7A1 gene, is also in phase 3 trials. D-Fi is injected into EB wounds during at least 2 treatment sessions.

FDA approval timeline

May 25, 2024 (no FDA advisory committee review of pz-cel is currently planned).

FDA designations: Breakthrough Therapy, Orphan Drug, RMAT, RPD, Priority Review

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected total U.S. sales	$13	$64	$129	$191	$230

**rivoceranib oral + camrelizumab IV**

Manufacturer: Elevar + Jiangsu Hengrui

Proposed indications

Combination of rivoceranib + camrelizumab for first-line treatment of unresectable hepatocellular carcinoma (HCC)

Clinical overview

Mechanism of action
Rivoceranib is a tyrosine kinase inhibitor (TKI) of vascular endothelial growth factor receptor 2 (VEGFR-2). Camrelizumab is a programmed cell death-1 (PD-1)-directed humanized monoclonal antibody.

Clinical trials

The open-label, phase 3 CARES-310 (NCT03764293) trial randomized (1:1) 543 treatment-naïve patients with unresectable or metastatic HCC to combination therapy with rivoceranib plus camrelizumab (R+C) or monotherapy with sorafenib 400 mg orally twice daily. Both co-primary endpoints of PFS and OS were significantly longer with R+C compared to sorafenib. At a median follow-up of 7.8 months, the median PFS was 5.6 months with R+C and 3.7 months with sorafenib (HR, 0.52; p<0.0001). At a median follow-up of 14.5 months, the median OS was 22.1 months with R+C and 15.2 months with sorafenib (HR, 0.62; p<0.0001). Serious TEAEs were reported more often with R+C (24%) than with sorafenib (6%). The most common grade 3 or 4 TEAEs reported with R+C were hypertension, palmar-plantar erythrodysesthesia, increased AST, and increased ALT. One death considered related to treatment was reported in each group (in the R+C group, due to multiple organ dysfunction; in the sorafenib group, due to respiratory failure and circulatory collapse).

Dosage and administration
Rivoceranib 250 mg was taken orally once daily. Camrelizumab 200 mg was administered IV every 2 weeks.

Place in therapy

It is estimated that 41,210 new cases of hepatobiliary cancer and 29,380 deaths due to the condition occured in the U.S. in 2023. HCC is the most common form of hepatobiliary cancer with the major causes being cirrhosis and chronic liver disease. Specific risk factors include HBV and/or HCV infection including co-infections with HIV, chronic alcohol consumption, non-alcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), and genetic hemochromatosis (GH).

Potential curative therapies for HCC include partial hepatectomy, in patients with a single tumor and preserved liver function, and liver transplantation, in those with inoperable disease. Locoregional therapy, such as ablation, arterially directed therapies, embolization, and radiation, may be appropriate for those who are ineligible for curative therapies or when used as a bridge to curative treatment. However, most patients with HCC have advanced disease at diagnosis and are not eligible for curative therapies. Systemic therapy is often viewed as treatment of last resort for patients with very advanced disease. Systemic treatments for HCC include orally administered kinase inhibitors and IV-administered monoclonal antibodies. Preferred 1st-line regimens for HCC include atezolizumab (Tecentriq®) + bevacizumab (Avastin®) (Child-Pugh A only) and tremelimumab-actl (Imjudo®) + durvalumab (Imfinzi®). Monotherapy with sorafenib (Nexavar®; Child-Pugh A only), lenvatinib (Lenvima®; Child-Pugh A only), or durvalumab are also recommended 1st-line options.

If approved, the combination of oral rivoceranib + IV camrelizumab will compete with the SOC regimen of IV atezolizumab + bevacizumab (median OS, 19.2 months) and the recently approved tremelimumab-actl + durvalumab IV regimen (median OS, 16.4 months).

FDA approval timeline

May 16, 2024

FDA designations: Orphan Drug (for both)

Financial forecast (reported in millions)

The financial forecasts for rivoceranib and camrelizumab are not currently available.

sotatercept SC

Manufacturer: Merck/Bristol-Myers Squibb

Proposed indications

Pulmonary arterial hypertension (PAH) (WHO Group 1) in adults

Clinical overview

Mechanism of action
Sotatercept is a fusion protein comprised of the extracellular domain of activin receptor IIa (ActRIIa) attached to the Fc portion of human IgG1. Activin ligands are involved in cell proliferation, apoptosis, and vessel wall inflammation in endothelial and smooth muscle cells of the lungs. When this activity is unbalanced, it leads to remodeling of pulmonary vasculature and PAH. Sotatercept sequesters activin ligands, thereby inhibiting activin signaling. It is proposed that this action can reverse vascular remodeling and improve vascular patency in PAH.

Clinical trials
The double-blind, placebo-controlled, phase 3 STELLAR (NCT04576988) trial evaluated sotatercept in 323 adults with PAH (WHO functional class [FC] II or III). Patients were randomized 1:1 to sotatercept or placebo as add-on to background therapy, which could be mono-, double, or triple therapy with medications for PAH. The mean patient age was 47.9 years, and mean time from diagnosis was 8.8 years among enrolled patients. At trial start, 61.3% were on triple therapy, and 39.9% were on prostacyclin infusion therapy. In the prespecified analysis of the primary endpoint, the median change from baseline in 6MWD at week 24 was +34.4 meters in the sotatercept group and +1 meter in the placebo group (p<0.001). The mean change from baseline in 6MWD at week 24 was +40.1 meters with sotatercept and -1.4 meters with placebo. Compared to placebo, there were significant improvements with sotatercept in secondary endpoints, including pulmonary vascular resistance, N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels, and WHO functional class. The risk of death or nonfatal clinical worsening events was 84% lower with sotatercept compared to placebo. Two deaths (1.2%) were reported in the sotatercept arm and 7 (4.4%) in the placebo arm; however, the study was not designed nor powered to assess the effects of sotatercept on mortality. No deaths were considered to have been caused by sotatercept. TEAEs reported with sotatercept included epistaxis, dizziness, telangiectasia, increased Hb levels, thrombocytopenia, and increased blood pressure.

The double-blind, placebo-controlled, phase 2 PULSAR (NCT03496207) trial evaluated sotatercept in 106 patients with moderate to severe PAH. Patients were randomized to sotatercept or placebo as add-on to background therapy, which could include mono-, double, or triple therapy with medications for PAH. The primary endpoint was pulmonary vascular resistance (PVR). Baseline PVR was 778.6 dyn/sec/cm−5 (normal PVR range is 100 to 200 dyn/sec/cm−5). After 24 weeks, the LS mean difference in the primary endpoint between sotatercept 0.7 mg/kg and placebo was -239.5 dyn/sec/cm−5 (p<0.001). The most common TEAEs with sotatercept were thrombocytopenia and increased Hb. One death was reported in the sotatercept 0.7 mg group due to cardiac arrest. Clinical efficacy was maintained in the open-label extension period for up to 24 months.

In addition, in the ongoing, open-label extension study SOTERIA (NCT04796337), patients either continued sotatercept or were switched from placebo to sotatercept. Top-line results revealed that among 131 patients with 1 year of treatment data (most from phase 2 PULSAR and SPECTRA trials), the changes from baseline to week 24 in 6MWD, NT-proBNP and WHO-FC were generally maintained at 1 year. Serious TEAEs were reported in 19.3% of the safety population, including 4 deaths (1%).

Dosage and administration
In the phase 3 trial, sotatercept was administered SC at a starting dose of 0.3 mg/kg, which was increased every 3 weeks to a target dose of 0.7 mg/kg. Notably, 99.4% of patients assigned to sotatercept received the target dose.

Place in therapy

PAH is a rare disease caused by progressive hyperproliferation of cells in the arterial walls in the lung. This causes narrowing and increased pressure in the pulmonary arteries, leading to increased load on the heart. WHO classifies pulmonary hypertension into five groups based on etiology. WHO Group I refers to PAH. In addition, WHO identifies PAH severity based on functional class (FC): FC-I severity represents symptom-free PAH; FC-II and FC-III convey symptoms (e.g., shortness of breath) with normal daily activities; and FC-IV indicates symptoms at rest and severe symptoms with physical activity.

It is estimated that 500 to 1,000 new cases of PAH are diagnosed in the U.S. each year, with most occurring in women 30 to 60 years of age. Approximately half of PAH cases have no known cause (idiopathic), and an estimated 6% to 20% of patients have a family history of the disease. The remaining cases of PAH are caused by drugs or toxins or are associated with other conditions, such as connective tissue disease, congenital heart disease, or pulmonary hypertension. Symptoms of PAH include dyspnea, particularly during physical activity, chest pain, and syncope. Prognosis for PAH is poor, with a 5-year mortality rate estimated at 40%. In most individuals, PAH progresses to right sided heart dysfunction and death.

There is no cure for PAH. In severe cases, lung or heart-lung transplant may be recommended. Several medications are available to treat symptoms of PAH by promoting vasodilation. They include oral, inhaled, and IV prostaglandins, oral endothelin receptor antagonists (ERAs), IV and oral phosphodiesterase type 5 (PDE5) inhibitors, an oral soluble guanylate cyclase stimulator (riociguat [Adempas®]), and an IV prostacyclin receptor agonist (selexipag [Upravi®]). Other agents, such as select calcium channel blockers, anticoagulants, and diuretics, are also used in patients with PAH.

If approved, SC sotatercept, administered every 3 weeks, will be a first-in-class activin signaling inhibitor and may be the first DMT to treat PAH. However, it has only been studied as an add-on therapy, including a majority of patients already on triple therapy. The place in therapy for sotatercept is not yet clearly defined in terms of when to add it in the treatment paradigm. The Institute for Clinical and Economic Review (ICER) published a final evidence report on sotatercept for PAH. The report concludes that the addition of sotatercept to background therapy can improve clinical outcomes with relatively few harms, and sotatercept’s SC administration is less burdensome than many other PAH treatments. Efficacy in sicker populations and in those with connective tissue disease remains uncertain, along with durability of effect.

FDA approval timeline

March 26, 2024

FDA designations: Breakthrough Therapy, Orphan Drug, Priority Review

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected total U.S. sales	$168	$417	$698	$1,011	$1,311

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 Quarterly Drug Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2028, are displayed. The financials are projected total annual U.S. sales, reported in millions.

Drug Generic Name	Therapeutic category	Projected Total U.S. sales for 2028 (dollars in millions)
afamitresgene autoleucel	Oncology/Cellular therapy	$29
aficamten	Cardiovascular	$1,085
arimoclomol	Metabolic	$78
datopotamab deruxtecan	Oncology	$1,037
donanemab	Neurology	$1,695
giroctocogene fitelparvovec	Hematology/Gene therapy	$195
imetelstat	Oncology	$552
obecabtagene autoleucel	Oncology/Cellular therapy	$145
palopegteriparatide	Endocrine	$744
relacorilant	Endocrine	$732
revumenib	Oncology	$298
seladelpar	Immunology	$662
tabelecleucel	Oncology/Cellular therapy	$353
tarlatamab	Oncology	$194
tovorafenib	Oncology	$481
xanomeline-trospium	Behavioral health	$1,822

Drug list

AThe pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2026. 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 recent Complete Response Letter (CRL) are also reported.

Gene & cellular therapies

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
lifileucel	Iovance	Melanoma (advanced unresectable or metastatic)	IV	BLA; Fast Track; Orphan Drug; Priority Review; RMAT	02/24/2024
atidarsagene autotemcel	Orchard	Metachromatic leukodystrophy (early onset)	IV	BLA; Orphan Drug; Priority Review; RMAT; RPD	03/18/2024
marnetegragene autotemcel	Rocket	Leukocyte adhesion deficiency type I (LAD-I)	IV	BLA; Fast Track; Orphan Drug; Priority Review; RMAT; RPD	03/31/2024
prademagene zamikeracel	Abeona	Epidermolysis bullosa (recessive dystrophic)	Topical	BLA; Breakthrough Therapy; Orphan Drug; Priority Review; RMAT; RPD	05/25/2024
obecabtagene autoleucel	Autolus	ALL (R/R, B cell)	IV	BLA; Orphan Drug; RMAT	11/27/2024
fidanacogene elaparvovec	Pfizer/Genentech	Hemophilia B (adults)	IV	BLA; Breakthrough Therapy; Orphan Drug; RMAT	Apr-Jun 2024
afamitresgene autoleucel	Adaptimmune	Synovial sarcoma	IV	BLA; Orphan Drug; RMAT	Aug-Dec 2024

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
lisocabtagene maraleucel (Breyanzi®)	Bristol-Myers Squibb	CLL/SLL (R/R, prior Bruton TKI and BCL-2 inhibitor)	IV	sBLA; Orphan Drug; Priority Review	03/14/2024
ciltacabtagene autoleucel (Carvykti®)	Janssen	Multiple myeloma (R/R, as earlier lines of therapy)	IV	sBLA; Breakthrough Therapy; Orphan Drug	04/05/2024

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
AAV8-ranibizumab	Abbvie	Wet-AMD	Subretinal	BLA; Orphan Drug	TBD
allogeneic adult stem cells (CAP-1002)	Nippon Shinyaku	DMD	IV	BLA; Orphan Drug; RPD; RMAT	TBD
autologous kidney cells (ReACT)	Prokidney	CKD	Renal cortex injection	BLA; RMAT	TBD
botaretigene sparoparvovec	Janssen	Retinitis pigmentosa	Subretinal	BLA; Fast Track; Orphan Drug	TBD
darvadstrocel	Takeda	CD (perianal fistulas)	IV	BLA; Orphan Drug	TBD
dirloctocogene samoparvovec	Genentech	Hemophilia A	IV	BLA; Breakthrough Therapy; Orphan Drug	TBD
giroctocogene fitelparvovec	Pfizer	Hemophilia A	IV	BLA; Fast Track; Orphan Drug; RMAT	TBD
RGX-121	Regenxbio	Mucopolysaccharidosis II (Hunter syndrome)	CNS injection	BLA; Fast Track; Orphan Drug; RPD; RMAT	TBD
tabelecleucel	Pierre Fabre	Epstein-Barr virus-associated post-transplant lymphoproliferative disease	IV	BLA; Breakthrough Therapy; Orphan Drug	TBD

None

Biosimilars

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
eculizumab (biosimilar to Alexion’s Soliris®)	Amgen	PNH; Hemolytic uremic syndrome (atypical)	IV	BLA	February 2024
adalimumab (biosimilar to Abbvie’s Humira®)	Alvotech	RA; AS; PSO; PsA; JIA; CD; UC; HS; Uveitis	SC	BLA	02/24/2024
insulin lispro (biosimilar to Eli Lilly's Humalog®)	Gan & Lee/Sandoz	T1DM; T2DM	SC	BLA	04/01/2024
insulin aspart (biosimilar to Sanofi-Aventis' Lantus®)	Gan & Lee/Sandoz	T1DM; T2DM	SC	BLA	04/14/2024
ranibizumab (biosimilar to Genentech's Lucentis®)	STADA Arzneimittel/Xbrane	Diabetic retinopathy; DME; Myopic choroidal neovascularization; Macular edema following RVO; Wet AMD	Intravitreal	BLA	04/21/2024
rituximab (biosimilar to Genentech’s Rituxan®)	Dr. Reddy's	CLL; Granulomatosis with polyangiitis/microscopic polyangiitis; NHL; Mature B-cell NHL/mature B-cell acute leukemia; Pemphigus vulgaris; RA	IV	BLA	05/10/2024
aflibercept (biosimilar to Regeneron's Eylea®)	Celltrion	DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD	Intravitreal	BLA	06/28/2024
aflibercept (biosimilar to Regeneron's Eylea)	Coherus	DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD	Intravitreal	BLA	06/28/2024
aflibercept (biosimilar to Regeneron's Eylea)	Amgen	DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD	Intravitreal	BLA	Jul-Aug 2024
ustekinumab (biosimilar to Janssen's Stelara®)	Intas	PSO; PsA; CD; UC	IV, SC	BLA	Nov-Dec 2024
ustekinumab (biosimilar to Janssen's Stelara)	Alvotech/Teva	PSO; PsA; CD; UC	IV, SC	BLA	01/08/2025
insulin aspart (biosimilar to Sanofi-Aventis' Lantus)	Amphastar	T1DM; T2DM	SC	BLA	01/10/2025
aflibercept (biosimilar to Regeneron's Eylea)	Biocon/Janssen	DME; Diabetic retinopathy; Macular edema following RVO; Wet AMD	Intravitreal	BLA	Pending
denosumab (biosimilar to Amgen’s Prolia®/Xgeva®)	Novartis	Osteoporosis/osteopenia	SC	BLA	Pending
insulin glargine (biosimilar to Sanofi's Lantus)	Gan & Lee/Sandoz	T1DM; T2DM	SC	BLA	Pending
pegfilgrastim (biosimilar to Amgen's Neulasta®)	Lupin	Neutropenia/leukopenia	SC	BLA	Pending
tocilizumab (biosimilar to Genentech’s Actemra®)	Fresenius Kabi	RA; Polyarticular JIA; Systemic JIA	IV	BLA	Pending
trastuzumab (biosimilar to Genentech’s Herceptin®)	Henlius/Accord	Breast cancer; Gastric or gastroesophageal junction adenocarcinoma	IV	BLA	Pending

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
adalimumab-bwwd 40 mg/0.4 mL (Hadlima™) (biosimilar to Abbvie’s Humira)	Organon	RA; AS; PSO; PsA; JIA; CD; UC; HS; Uveitis	SC	sBLA for interchangeability	09/06/2024

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
aflibercept (biosimilar to Regeneron’s Eylea)	Sandoz	DME; Wet-AMD	Intravitreal	BLA	TBD
bevacizumab (biosimilar to Genentech’s Avastin®)	Essex	DME; Wet-AMD	IV	BLA	TBD
ustekinumab (biosimilar to Janssen’s Stelara)	Formycon	PSO; PsA; CD; UC	IV, SC	BLA	TBD

None

Specialty

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
budesonide viscous oral suspension	Takeda	Eosinophilic esophagitis	Topical	505(b)(2) NDA; Breakthrough Therapy; Orphan Drug	Jan-Feb 2024
donanemab	Eli Lilly	Alzheimer's disease (early, symptomatic)	IV	BLA; Breakthrough Therapy	Jan-Mar 2024
glatiramer depot	Viatris	MS	IM	505(b)(2) NDA	03/08/2024
resmetirom	Madrigal	NASH (liver fibrosis)	Oral	NDA; seeking Accelerated Approval; Breakthrough Therapy; Fast Track; Priority Review	03/14/2024
givinostat	Italfarmaco	DMD	Oral	NDA; Fast Track; Orphan Drug; Priority Review; RPD	03/21/2024
sotatercept	Merck	PAH	SC	BLA; Breakthrough Therapy; Orphan Drug; Priority Review	03/26/2024
vadadustat	Akebia	Anemia due to CKD (dialysis-dependent)	Oral	NDA	03/27/2024
odronextamab	Pfizer	DLBCL (R/R); Follicular lymphoma (R/R)	IV	BLA; Fast Track; Orphan Drug; Priority Review	03/31/2024
RSV pre-fusion F protein vaccine (mRNA-1345)	Moderna	RSV-LRTD prevention (ages > 60 years)	IM	BLA; Breakthrough Therapy; Fast Track; Priority Review	April 2024
apomorphine infusion pump	Supernus	Parkinson's disease (continuous treatment of motor fluctuations)	SC infusion	NDA	04/05/2024
nogapendekin alfa inbakicept	Immunitybio	Bladder cancer (BCG-unresponsive, non-muscle-invasive, CIS, with or without Ta or T1 disease)	Intravesical	BLA; Breakthrough Therapy; Fast Track	04/23/2024
mavorixafor	X4	Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome (ages ≥ 12 years)	Oral	NDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review; RPD	04/30/2024
tovorafenib	Day One	Glioma (relapsed/progressive, low-grade, monotherapy)	Oral	NDA; Breakthrough Therapy; Orphan Drug; Priority Review; RPD	04/30/2024
palopegteriparatide	Ascendis	Hypoparathyroidism	SC	NDA; Orphan Drug	05/14/2024
camrelizumab	Jiangsu Hengrui	HCC (unresectable, 1st-line, in combination with rivoceranib)	IV	BLA; Orphan Drug	05/16/2024
rivoceranib	Elevar	HCC (unresectable, 1st-line, in combination with camrelizumab)	Oral	NDA; Orphan Drug	05/16/2024
macitentan/tadalafil	Janssen	PAH (WHO functional class II-III)	Oral	NDA; Orphan Drug	05/30/2024
elafibranor	Genfit	Primary biliary cholangitis	Oral	NDA; Breakthrough Therapy; Orphan Drug; Priority Review	06/10/2024
tarlatamab	Amgen	SCLC (advanced)	IV	BLA; Breakthrough Therapy; Orphan Drug; Priority Review; RTOR	06/12/2024
imetelstat	Geron	Myelodysplastic syndrome (transfusion-dependent anemia, low-risk disease)	IV	NDA; Fast Track; Orphan Drug	06/14/2024
pneumococcal polyvalent conjugate vaccine	Merck	Pneumococcal immunization (adults)	IM	BLA; Breakthrough Therapy; Priority Review	06/17/2024
arimoclomol	Zevra	Niemann-Pick disease type C	Oral	NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RPD	06/21/2024
patritumab deruxtecan	Merck	NSCLC ( locally advanced or metastatic, EGFR-mutated, ≥ 2 prior systemic therapies)	IV	BLA; Breakthrough Therapy; Priority Review	06/26/2024
polyspecific immunoglobulin preparation	Biotest	Primary immunodeficiencies	IV	BLA	06/29/2024
tislelizumab	Beigene	Esophageal squamous cell carcinoma (unresectable, recurrent, locally advanced or metastatic, 1st-line)	IV	BLA; Orphan Drug	July 2024
revumenib	Syndax	AML (R/R, KMT2A-rearranged)	Oral	BLA; Breakthrough Therapy; Orphan Drug; RTOR	Jul-Dec 2024
naloxone (powder-based technology)	Orexo	Opioid overdose	Intranasal	505(b)(2) NDA	07/15/2024
crovalimab	Genentech	PNH	IV, SC	BLA; Breakthrough Therapy; Orphan Drug	07/27/2024
danicopan	AstraZeneca	PNH	Oral	NDA; Breakthrough Therapy; Orphan Drug	07/27/2024
glepaglutide	Zealand	Short bowel syndrome (dependent on parenteral support)	SC	NDA; Orphan Drug	Aug-Dec 2024
midomafetamine	Lykos	PTSD	Oral	NDA; Breakthrough Therapy	Aug-Dec 2024
seladelpar	CymaBay	Primary biliary cholangitis (including pruritus, ursodeoxycholic acid inadequate response/intolerance)	Oral	NDA; Breakthrough Therapy; Orphan Drug	Aug-Dec 2024
deuruxolitinib	Sun	Alopecia areata (moderate-severe)	Oral	NDA; Breakthrough Therapy; Fast Track	08/6/2024
garadacimab	CSL	HAE	SC	BLA; Fast Track; Orphan Drug	Oct-Nov 2024
marstacimab	Pfizer	Hemophilia A and B (without factor VIII or IX inhibitors)	IV, SC	BLA; Fast Track; Orphan Drug	Oct-Dec 2024
octreotide	Novartis	Acromegaly	SC	NDA	10/21/2024
gavorestat	Applied Therapeutics	Galactosemia	Oral	NDA; Fast Track; Orphan Drug; RPD	December 2024
acoramidis	Bridgebio	Transthyretin amyloid cardiomyopathy (ATTR-CM)	Oral	NDA	12/05/2024
irinotecan liposome	CSPC	Pancreatic cancer	IV	NDA	12/18/2024
axatilimab	Syndax	Chronic GVHD (failure of ≥ 2 prior lines of systemic therapy, ages ≥ 6 years)	IV	BLA; Fast Track; Orphan Drug	12/27/2024
atezolizumab SC (Tecentriq®)	Genentech	Alveolar soft part sarcoma; Breast cancer (TNBC); HCC; Melanoma; SCLC	SC	BLA	Pending

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
coagulation factor IX, recombinant (Ixinity®)	Pediapharm	Hemophilia B (on-demand, prophylactic, and perioperative treatment, ages < 12 years)	IV	sBLA	Jan-Mar 2024
influenza vaccine, live (FluMist® Quadrivalent)	AstraZeneca	Seasonal influenza immunization (self/caregiver-administration)	Intranasal	sBLA	Jan-Mar 2024
omalizumab (Xolair®)	Genentech	Food allergies (multiple foods)	SC	sBLA; Breakthrough Therapy; Priority Review	Jan-Mar 2024
osimertinib (Tagrisso®)	AstraZeneca	NSCLC (locally advanced or metastatic, EGFR-mutated)	Oral	sNDA; Breakthrough Therapy; Fast Track; Orphan Drug; Priority Review	Jan-Mar 2024
zanubrutinib (Brukinsa®)	Beigene	Follicular lymphoma (R/R, ≥ 3rd-line, in combination with obinutuzumab)	Oral	sNDA; Fast Track; Orphan Drug	Jan-Mar 2024
tesamorelin (Egrifta®) (high concentration)	Theratechnologies	HIV lipodystrophy	SC	sBLA	01/22/2024
bupivacaine/meloxicam (Zynrelef®)	Heron	Postsurgical pain (soft tissue and orthopedic surgical procedures)	Surgical site instillation	505(b)(2) sNDA; Breakthrough Therapy; Fast Track	01/23/2024
dupilumab (Dupixent®)	Sanofi	Eosinophilic esophagitis (ages 1-11 years)	SC	sBLA; Breakthrough Therapy; Orphan Drug; Priority Review	01/31/2024
irinotecan liposomal (Onivyde®)	Ipsen	Pancreatic cancer (metastatic ductal adenocarcinoma, 1st-line, in combination with 5-fluorouracil/leucovorin/oxaliplatin)	IV	505(b)(2) sNDA; Fast Track; Orphan Drug	February 2024
nintedanib (Ofev®)	Boehringer Ingelheim	Interstitial lung disease (ages 6-17 years)	Oral	sNDA; Breakthrough Therapy	Mar-Apr 2024
tasimelteon (Hetlioz®)	Vanda	Jet lag disorder	Oral	sNDA	03/04/2024
maralixibat (Livmarli®)	Mirum	Progressive familial intrahepatic cholestasis-related pruritus (ages ≥ 2 months)	Oral	sNDA; Breakthrough Therapy; Orphan Drug	03/13/2024
nivolumab (Opdivo®)	Bristol-Myers Squibb	Urothelial carcinoma (unresectable or metastatic, 1st-line, in combination with cisplatin-based chemotherapy)	IV	sBLA; Breakthrough Therapy; Priority Review	04/05/2024
valbenazine (Ingrezza®) oral granules	Neurocrine Biosciences	Huntington's disease (chorea)	Oral	sNDA; Orphan Drug	04/30/2024
hepatitis B vaccine (recombinant), adjuvanted (Heplisav-B®)	Dynavax	Hepatitis B immunization (adults on hemodialysis)	IM	sBLA	05/13/2024
immune globulin (human), 10% (Gammagard Liquid)	Takeda	Chronic inflammatory demyelinating polyneuropathy (CIDP)	IV	sBLA	05/27/2024
sarilumab (Kevzara®)	Sanofi	JIA (polyarticular-course)	SC	sBLA	06/10/2024
amivantamab-vmjw (Rybrevant®)	Janssen	NSCLC (locally advanced or metastatic, EGFR exon 20 insertion mutation, 1st-line, in combination with carboplatin-pemetrexed)	IV	sBLA; Breakthrough; RTOR	06/25/2024
risankizumab-rzaa (Skyrizi®)	Abbvie	UC	IV, SC	sBLA	06/28/2024
vedolizumab (Entyvio®)	Takeda	CD (SC maintenance following IV induction)	SC	sBLA	July 2024
fam-trastuzumab deruxtecan-nxki (Enhertu®)	Daiichi Sankyo	Breast cancer (HER2+, 3rd-line)	IV	sBLA; Breakthrough Therapy; Fast Track	Jul-Dec 2024
amivantamab-vmjw (Rybrevant)	Janssen	NSCLC (locally advanced or metastatic, EGFR exon 19 deletions or L858R substitution, 2nd-line, after disease progression on/after osimertinib, in combination with carboplatin-pemetrexed)	IV	sBLA; Breakthrough Therapy	09/20/2024
ibalizumab (Trogarzo®)	Theratechnologies	HIV-1 infection	IM	sBLA; Breakthrough Therapy; Fast Track; Orphan Drug	11/01/2024

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
abelacimab	Anthos	Stroke prevention in atrial fibrillation; VTE	SC	BLA; Fast Track	TBD
acetylleucine	IntraBio	Niemann-Pick disease type C	Oral	NDA; Fast Track; Orphan Drug; RPD	TBD
adintrevimab	Invivyd	COVID-19	IM	BLA	TBD
aficamten	Cytokinetics	Hypertrophic cardiomyopathy	Oral	NDA; Breakthrough Therapy; Orphan Drug	TBD
amlitelimab	Sanofi	Atopic dermatitis	SC	BLA	TBD
anti-betv1 antibody (REGN-5713-5714-5715)	Regeneron	Birch allergy	SC	BLA	TBD
anti-BK polyomavirus	Memo	BK polyomavirus infection (renal transplant recipients)	IV	BLA; Fast Track	TBD
ARO-APOC3	Arrowhead	Familial chylomicronemia syndrome	SC	NDA; Fast Track; Orphan Drug	TBD
astegolimab	Genentech	COPD	IV	BLA	TBD
avutometinib	Verastem	Ovarian cancer	Oral	NDA; Breakthrough Therapy	TBD
bentracimab	SERB	Ticagrelor (Brilinta®) reversal	IV	BLA; Breakthrough Therapy	TBD
blarcamesine	Anavex	Alzheimer's disease	Oral	NDA	TBD
cannabidiol (synthetic)	Radius	Infantile spasms (West syndrome; epilepsy)	Oral	505(b)(2) NDA; Orphan Drug	TBD
cannabidiol gel	Harmony	Fragile X syndrome	Topical	NDA; Fast Track; Orphan Drug	TBD
cetuximab sarotalocan	Rakuten Medical	SCCHN	IV	BLA; Fast Track	TBD
condoliase	Ferring	Lumbar disc herniation	Intradiscal	BLA	TBD
copper histidine	Zydus	Menkes disease	SC	NDA; Breakthrough Therapy; Fast Track; Orphan Drug; RPD	TBD
crinecerfont	Neurocrine	Congenital adrenal hyperplasia	Oral	NDA; Breakthrough Therapy; Orphan Drug	TBD
crovalimab	Genentech	Hemolytic uremic syndrome	IV, SC	BLA	TBD
CTX-009	Compass	Biliary tract cancer	IV	BLA	TBD
datopotamab deruxtecan	Daiichi Sankyo	Breast cancer (HR+/HER2-); NSCLC	IV	BLA	TBD
defactinib	Verastem	Ovarian cancer	Oral	NDA; Orphan Drug	TBD
deoxythymidine/deoxycytidine	UCB	Thymidine kinase 2 (TK2) deficiency	Oral	BLA; Breakthrough Therapy; Orphan Drug	TBD
depemokimab	GlaxoSmithKline	Asthma	SC	BLA	TBD
dersimelagon	Mitsubishi Tanabe	Porphyria	Oral	NDA; Fast Track; Orphan Drug	TBD
dinutuximab beta	EUSA	Neuroendocrine tumors	IV	BLA; Orphan Drug	TBD
efruxifermin	Akero	NASH	SC	BLA; Breakthrough Therapy; Fast Track	TBD
etavopivat	Novo Nordisk	SCD	Oral	NDA; Fast Track; Orphan Drug; RPD	TBD
evobrutinib	Merck	MS	Oral	NDA	TBD
factor VIII mimetic bispecific antibody	Novo Nordisk	Hemophilia A	SC	BLA; Orphan Drug	TBD
fenebrutinib	Genentech	MS	Oral	NDA	TBD
fianlimab	Regeneron	Melanoma	IV	BLA; Fast Track	TBD
fitusiran	Sanofi	Hemophilia A and B	SC	NDA; Fast Track; Orphan Drug	TBD
garetosmab	Regeneron	Fibrodysplasia ossificans progressiva	IV	BLA; Fast Track; Orphan Drug	TBD
GBT601	Pfizer	SCD	Oral	NDA; Orphan Drug	TBD
giredestrant	Genentech	Breast cancer (HR+/HER2-)	Oral	NDA; Fast Track	TBD
gold nanocrystal	Clene	ALS	Oral	NDA; Orphan Drug	TBD
house dust mite allergen extracts	Stallergenes Greer	Allergic rhinitis (due to house dust mite)	SL	BLA	TBD
ianalumab	Novartis	Autoimmune hemolytic anemia; Sjogren's syndrome	SC	BLA; Fast Track	TBD
imlifidase	Sarepta	Kidney transplant rejection	IV	BLA; Fast Track; Orphan Drug	TBD
imsidolimab	Anaptysbio	Generalized pustular psoriasis	IV, SC	BLA; Orphan Drug	TBD
inavolisib	Genentech	Breast cancer (HR+/HER2-, 1st-line)	Oral	NDA	TBD
inclacumab	Pfizer	SCD	IV	BLA; Orphan Drug	TBD
isotretinoin	Timber	Congenital ichthyosis	Topical	505(b)(2) NDA; Breakthrough Therapy; Fast Track; Orphan Drug	TBD
itepekimab	Regeneron	COPD	SC	BLA; Fast Track	TBD
JDQ-443	Novartis	NSCLC	Oral	NDA	TBD
latozinemab	Aldeyra	Frontotemporal dementia	IV	BLA; Fast Track; Orphan Drug	TBD
lazertinib	Genosco/Janssen	NSCLC	Oral	NDA	TBD
leukocyte interleukin	CEL-SCI	SCCHN	SC	BLA; Orphan Drug	TBD
ligelizumab	Novartis	Food allergies	SC	BLA	TBD
linerixibat	GlaxoSmithKline	Primary biliary cholangitis pruritus	Oral	NDA; Orphan Drug	TBD
marzeptacog alfa	Catalyst	Hemophilia A and B (with inhibitors)	SC	BLA; Fast Track; Orphan Drug	TBD
navepegritide	Ascendis	Achondroplasia	SC	NDA; Orphan Drug	TBD
navitoclax	Abbvie	Myelofibrosis	Oral	NDA; Orphan Drug	TBD
nemolizumab	Galderma	Pruritus	SC	BLA; Breakthrough Therapy	TBD
nipocalimab	Janssen	Autoimmune hemolytic anemia	IV	BLA; Fast Track; Orphan Drug	TBD
nomacopan	Akari	HSCT-associated thrombotic microangiopathy	SC	BLA; Fast Track; Orphan Drug; RPD	TBD
ocrelizumab (Ocrevus®) SC	Genentech	MS	SC	BLA	TBD
olezarsen	Akcea	Familial chylomicronemia syndrome	SC	NDA; Fast Track	TBD
ORL-101	Orpha Labs	Leukocyte adhesion deficiency type II (LAD-II)	Oral	NDA; Fast Track; Orphan Drug	TBD
paltusotine	Crinetics	Acromegaly	Oral	NDA; Orphan Drug	TBD
paromomycin	Appili	Leishmaniasis	Topical	NDA; Orphan Drug	TBD
pegadricase	Swedish Orphan Biovitrum	Gout	IV	BLA	TBD
pegargiminase	Polaris	Mesothelioma	IM	BLA; Fast Track; Orphan Drug	TBD
pegzilarginase	Immedica	Arginase 1 deficiency	IV	BLA; Breakthrough Therapy; Fast Track; Orphan Drug; RPD	TBD
pelabresib	MorphoSys	Myelofibrosis	Oral	NDA; Fast Track; Orphan Drug	TBD
pelacarsen	Novartis	Dyslipidemia/hypercholesterolemia	SC	NDA; Fast Track	TBD
piclidenoson	Can-Fite	PSO	Oral	NDA	TBD
plinabulin	Beyondspring	Chemotherapy-induced neutropenia prevention; NSCLC	IV	NDA; Breakthrough Therapy	TBD
povorcitinib	Incyte	Hidradenitis suppurativa	Oral	NDA	TBD
QRX003	Quoin	Netherton syndrome	Topical	NDA	TBD
relacorilant	Corcept	Cushing's syndrome	Oral	NDA; Orphan Drug	TBD
remibrutinib	Novartis	MS; Urticaria	Oral	NDA	TBD
resiniferatoxin	Grunenthal	Osteoarthritis pain (knee)	Intra-articular	NDA; Breakthrough Therapy	TBD
rilzabrutinib	Sanofi	ITP	Oral	NDA; Fast Track; Orphan Drug	TBD
rusfertide	Protagonist	Polycythemia vera	SC	NDA; Fast Track; Orphan Drug	TBD
sabatolimab	Novartis	Myelodysplastic syndrome	IV	BLA; Fast Track	TBD
sebetralstat	Kalvista	HAE	Oral	NDA; Fast Track; Orphan Drug	TBD
sepiapterin	PTC	Phenylketonuria (PKU)	Oral	NDA; Orphan Drug	TBD
serplulimab	Henlius	SCLC	IV	BLA; Orphan Drug	TBD
soticlestat	Takeda	Dravet syndrome; Lennox-Gastaut syndrome	Oral	NDA; Orphan Drug	TBD
sozinibercept	Opthea	Wet-AMD	Intravitreal	BLA; Fast Track	TBD
tamibarotene	Syros	Myelodysplastic syndrome	Oral	NDA; Fast Track; Orphan Drug	TBD
telisotuzumab vedotin	Abbvie	NSCLC	IV	BLA; Breakthrough Therapy	TBD
tiragolumab	Genentech	Esophageal cancer; NSCLC	IV	BLA; Orphan Drug	TBD
tiratricol	Rare Thyroid Therapeutics	Resistance to thyroid hormone type beta (RTH-b)	Oral	NDA; Fast Track; Orphan Drug; RPD	TBD
tislelizumab	Beigene	Gastric cancer; HCC; Nasopharyngeal cancer	IV	BLA; Orphan Drug	TBD
tolebrutinib	Sanofi	MS	Oral	NDA	TBD
tramiprosate	Alzheon	Alzheimer's disease	Oral	NDA; Fast Track	TBD
venglustat	Sanofi	Gaucher's disease; GM2 gangliosidoses	Oral	NDA; Orphan Drug	TBD
viaskin peanut	DBV	Peanut allergy	Transdermal	BLA; Breakthrough Therapy; Fast Track	TBD
von Willebrand factor concentrate	LFB	von Willebrand disease	IV	BLA; Orphan Drug	TBD
vorasidenib	Servier	Brain cancer (IDH-mutant diffuse glioma)	Oral	NDA; Breakthrough Therapy; Fast Track; Orphan Drug	TBD
zanidatamab	Jazz	Gastric cancer	IV	BLA; Fast Track; Orphan Drug	TBD

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
adagrasib (Krazati®)	Mirati	CRC	Oral	sNDA; Breakthrough Therapy	TBD
alpelisib (Piqray®)	Novartis	Breast cancer (HER2+); Ovarian cancer	Oral	sNDA	TBD
atezolizumab (Tecentriq)	Genentech	SCCHN	IV	sBLA	TBD
baricitinib (Olumiant®)	Eli Lilly	JIA	Oral	sNDA	TBD
benralizumab (Fasenra®)	AstraZeneca	Eosinophilic esophagitis	SC	sBLA; Orphan Drug	TBD
bimekizumab-bkzx (Bimzelx®)	UCB	Hidradenitis suppurativa	SC	sBLA	TBD
brolucizumab-dbll (Beovu®)	Novartis	Diabetic retinopathy	Intravitreal	sBLA	TBD
canakinumab (Ilaris®)	Novartis	NSCLC (adjuvant)	SC	sBLA	TBD
cemiplimab-rwlc (Libtayo®)	Regeneron	Melanoma	IV	sBLA; Fast Track	TBD
dexmedetomidine (IgalmiI™)	BioXcel	Alzheimer’s disease-related neuropsychiatric symptoms	Oral transmucosal	sNDA; Breakthrough Therapy	TBD
dupilumab (Dupixent)	Sanofi	Bullous pemphigoid; COPD	SC	sBLA; Orphan Drug	TBD
durvalumab (Imfinzi®)	AstraZeneca	Bladder cancer (1st-line)	IV	sBLA; Breakthrough Therapy	TBD
eplontersen (Wainua™)	Ionis	Transthyretin amyloid cardiomyopathy	SC	sNDA; Orphan Drug	TBD
ferric derisomaltose (Monoferric®)	Pharmacosmos	Anemia in heart failure	IV	sNDA	TBD
fostamatinib (Tavalisse®)	Rigel	Autoimmune hemolytic anemia	Oral	sNDA; Fast Track; Orphan Drug	TBD
guselkumab (Tremfya®)	Janssen	UC	SC	sBLA	TBD
iptacopan (Fabhalta®)	Novartis	C3 glomerulopathy (C3G); Hemolytic uremic syndrome; Immunoglobulin A nephropathy (Berger's disease)	Oral	sNDA; Breakthrough Therapy; Orphan Drug; RPD	TBD
mepolizumab (Nucala®)	GlaxoSmithKline	COPD	IV, SC	sBLA	TBD
mirikizumab-mrkz (Omvoh™)	Eli Lilly	CD	IV, SC	sBLA	TBD
mitapivat (Pyrukynd®)	Agios	SCD; Thalassemia (Alpha, Beta)	Oral	sNDA; Orphan Drug	TBD
mosunetuzumab-axgb (Lunsumio™)	Genentech	DLBCL	SC	sBLA	TBD
obinutuzumab (Gazyva®)	Genentech	Lupus nephritis; Membranous nephropathy; SLE	IV	sBLA; Breakthrough Therapy	TBD
pegcetacoplan (Empaveli®)	Apellis	Autoimmune hemolytic anemia	SC	sNDA; Orphan Drug	TBD
pozelimab (Veopoz™)	Regeneron	PNH	IV, SC	sBLA; Orphan Drug	TBD
ranibizumab port delivery system (Susvimo®)	Genentech	DME	Intravitreal implant	sBLA	TBD
romiplostim (Nplate®)	Amgen	Chemotherapy-induced thrombocytopenia	SC	sBLA; Orphan Drug	TBD
satralizumab-mwge (Enspryng®)	Genentech	Myelin oligodendrocyte glycoprotein antibody-associated disease	SC	sBLA; Orphan Drug	TBD
secukinumab (Cosentyx®)	Novartis	Giant cell arteritis; Lupus nephritis	SC	sBLA	TBD
sotorasib (Lumakras®)	Amgen	CRC	Oral	sNDA; Orphan Drug	TBD
sparsentan (Filspari®)	Travere	Focal segmental glomerulosclerosis	Oral	sNDA; Orphan Drug	TBD
toripalimab-tpzi (Loqtorzi™)	Coherus	Esophageal cancer	IV	sBLA; Orphan Drug	TBD
vedolizumab (Entyvio®)	Takeda	GVHD prophylaxis	IV	sBLA; Orphan Drug	TBD
venetoclax (Venclexta®)	Abbvie/Genenetech	Myelodysplastic syndrome; Multiple myeloma	Oral	sNDA; Breakthrough Therapy; Orphan Drug	TBD
vutrisiran (Amvuttra®)	Alnylam	Transthyretin amyloid cardiomyopathy	SC	sNDA; Orphan Drug	TBD

Traditional

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
dihydroergotamine nasal powder	Shin Nippon	Migraine (acute treatment)	Intranasal	505(b)(2) NDA	January 2024
scopolamine gel	Repurposed Therapeutics	Motion sickness	Intranasal	NDA; Priority Review	01/26/2024
atropine 0.01%	Nevakar	Myopia (pediatrics)	Ophthalmic	505(b)(2) NDA	01/31/2024
cefepime/taniborbactam	Venatorx/Melinta	UTI (complicated)	IV	NDA; Fast Track; QIDP; Priority Review	02/22/2024
roluperidone	Minerva	Schizophrenia (negative symptoms)	Oral	NDA	02/26/2024
cefepime/enmetazobactam	Allecra	UTI (complicated)	IV	NDA; Fast Track; QIDP	02/27/2024
aprocitentan	Idorsia	Hypertension (resistant)	Oral	NDA	03/19/2024
insulin icodec	Novo Nordisk	T1DM; T2DM	SC	BLA	April 2024
ceftobiprole medocaril	Basilea	ABSSSI; CAP; Staphylococcus aureus bacteremia	IV	NDA; Fast Track; QIDP	04/03/2024
pivmecillinam	Utility Therapeutics	UTI (uncomplicated)	Oral	NDA; Priority Review; QIDP	04/24/2024
diazepam buccal film (Libervant™)	Aquestive	Seizure disorders (ages 2-5 years)	Oral transmucosal	sNDA; Fast Track; Orphan Drug	04/26/2024
sofpironium	Botanix	Axillary hyperhidrosis (severe)	Topical	NDA	June 2024
ensifentrine	Verona	COPD	Inhaled	NDA	06/26/2024
tradipitant	Vanda	Gastroparesis	Oral	NDA	09/18/2024
xanomeline/trospium	Karuna	Schizophrenia	Oral	NDA	09/26/2024
minocycline	Journey	Rosacea	Oral	505(b)(2) NDA	01/05/2025

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
semaglutide (Wegovy®)	Novo Nordisk	CVD (risk reduction of MACE, adults with BMI of ≥ 27 kg/m² & established CVD)	SC	sNDA; Priority Review	March 2024
fluticasone propionate (Xhance®)	Optinose	Chronic rhinosinusitis	Intranasal	sNDA	03/16/2024
bempedoic acid (Nexletol®)	Esperion	CV risk reduction	Oral	sNDA	03/29/2024
bempedoic acid/ezetimibe (Nexlizet®)	Esperion	CV risk reduction	Oral	sNDA	03/29/2024
iloperidone (Fanapt®)	Vanda	Bipolar disorder	Oral	sNDA	04/02/2024
rilpivirine (Edurant®)	Janssen	HIV-1 infection (in children weighing ≥ 10 kg)	Oral	sNDA	05/28/2024
roflumilast (Zoryve®)	Arcutis	Atopic dermatitis (adults and pediatrics ages ≥ 6 years)	Topical	sNDA	07/07/2024
vonoprazan (Takecab®)	Phathom	Gastroesophageal reflux disease (non-erosive)	Oral	sNDA	07/19/2024
anacaulase-bcdb (Nexobrid®)	Mediwound	Removal of eschar due to thermal burns (deep partial/full-thickness, pediatric)	Topical	sBLA; Orphan Drug	11/08/2024

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
acoltremon	Aerie	DED	Ophthalmic	NDA	TBD
aroxybutynin/atomoxetine	Apnimed	Sleep apnea	Oral	NDA; Fast Track	TBD
asundexian	Bayer	Stroke prevention in atrial fibrillation	Oral	NDA; Fast Track	TBD
atrasentan	Novartis	Immunoglobulin A (IgA) nephropathy (Berger's disease)	Oral	NDA	TBD
aztreonam-avibactam	Abbvie	Intra-abdominal bacterial infections	IV	NDA; Fast Track; QIDP	TBD
baclofen/naltrexone/sorbitol	Pharnext	Charcot-Marie-Tooth disease	Oral	NDA	TBD
bemnifosbuvir	Atea	COVID-19	Oral	NDA; Fast Track	TBD
brensocatib	Insmed	Bronchiectasis	Oral	NDA; Breakthrough Therapy	TBD
brilaroxazine	Reviva	Schizophrenia	Oral	NDA	TBD
cagrilintide/semaglutide	Novo Nordisk	Obesity/overweight; T2DM	SC	NDA	TBD
carbachol/brimonidine	Visus	Presbyopia	Ophthalmic	505(b)(2) NDA	TBD
chikungunya vaccine	Bavarian Nordic	Chikungunya virus prevention	IM	BLA; Breakthrough Therapy; Fast Track	TBD
colistimethate	Zambon	Bronchiectasis	Inhaled	NDA; Breakthrough Therapy; Fast Track; QIDP	TBD
COVID-19 vaccine (SP0253)	Sanofi	COVID-19	IM	BLA	TBD
cyclobenzaprine	Tonix	Fibromyalgia	SL	505(b)(2) NDA	TBD
cytisinicline	Achieve Life Sciences	Smoking cessation	Oral	NDA	TBD
dengue tetravalent vaccine, live, attenuated	Takeda	Dengue fever (ages 4-60 years)	SC	BLA; Fast Track	TBD
epinephrine	ARS	Anaphylaxis	Intranasal	505(b)(2) NDA; Fast Track	TBD
epinephrine (sublingual)	Aquestive	Anaphylaxis	SL	505(b)(2) NDA; Fast Track	TBD
esreboxetine	Axsome	Fibromyalgia	Oral	NDA	TBD
estetrol	Mithra	Menopausal vasomotor symptoms	Oral	NDA	TBD
gepotidacin	GlaxoSmithKline	UTI (uncomplicated)	Oral	NDA; QIDP	TBD
influenza nanoparticle vaccine	Novavax	Seasonal influenza prevention	IM	BLA; Fast Track	TBD
lerodalcibep	LIB	Dyslipidemia/hypercholesterolemia	SC	BLA	TBD
levodopa/carbidopa patch pump	Mitsubishi Tanabe	Parkinson's disease	SC infusion	505(b)(2) NDA	TBD
meningococcal vaccine (GSK3536819A)	GlaxoSmithKline	Meningococcal immunization	IM	BLA	TBD
molnupiravir (Lagevrio)	Merck	COVID-19	Oral	NDA	TBD
nalbuphine ER	Trevi	Pruritus	Oral	NDA; Fast Track	TBD
navacaprant	Neumora	MDD	Oral	NDA	TBD
obicetrapib	NewAmsterdam	Dyslipidemia/hypercholesterolemia	Oral	NDA	TBD
orforglipron	Eli Lilly	Obesity/overweight; T2DM	Oral	NDA	TBD
PL-9643	Palatin	DED	Ophthalmic	NDA	TBD
quadrivalent influenza mRNA vaccine (mRNA-1010)	Moderna	Seasonal influenza prevention	IM	BLA	TBD
reproxalap	Aldeyra	DED	Ophthalmic	NDA	TBD
retatrutide	Eli Lilly	Obesity/overweight; T2DM	SC	NDA	TBD
sulopenem etzadroxil/ probenicid	Iterum	UTI (uncomplicated)	Oral	NDA; Fast Track; QIDP	TBD
survodutide	Boehringer Ingelheim	Obesity/overweight; T2DM	SC	NDA	TBD
ulotaront	Sumitomo	Schizophrenia	Oral	NDA; Breakthrough Therapy	TBD
visomitin	Mitotech	DED	Ophthalmic	NDA	TBD
XEN1101	Xenon	Partial/focal seizures	Oral	NDA	TBD
zoliflodacin	Innoviva	UTI	Oral	NDA; Fast Track; QIDP	TBD

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
dextromethorphan/bupropion (Auvelity™)	Axsome	Alzheimer’s disease-related neuropsychiatric symptoms	Oral	505(b)(2) sNDA; Breakthrough Therapy; Fast Track	TBD
ibrexafungerp (Brexafemme®)	GlaxoSmithKline	Fungal infections (systemic)	Oral	sNDA; Fast Track; Orphan Drug; QIDP	TBD
lumateperone (Caplyta®)	Intra-Cellular Therapies	MDD	Oral	sNDA	TBD
phentolamine 0.75% (Ryzumvi™)	Ocuphire	Presbyopia	Ophthalmic	505(b)(2) sNDA	TBD
semaglutide (Rybelsus®)	Novo Nordisk	Obesity/overweight	Oral	sNDA	TBD
semaglutide (Wegovy®)	Novo Nordisk	Chronic HFpEF	SC	sNDA	TBD
tapinarof (Vtama®)	Dermavant	Atopic dermatitis	Topical	sNDA	TBD

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
cosibelimab	Checkpoint	Cutaneous squamous cell carcinoma (metastatic)	IV	CRL	TBD
dasiglucagon (Zegalogue®)	Novo Nordisk	Congenital hyperinsulinemia (pediatric patients ages ≥ 7 days)	SC	CRL	TBD
dupilumab (Dupixent)	Sanofi	Urticaria	SC	CRL	TBD
gefapixant	Merck	Chronic cough	Oral	CRL	TBD
reproxalap	Aldeyra	DED	Ophthalmic	CRL	TBD
sotorasib (Lumakras®)	Amgen	NSCLC (locally advanced or metastatic, KRAS G12Cmutated; ≥ 2-line)	Oral	CRL	TBD
zolbetuximab	Astellas	Gastric or gastroesophageal junction (G/GEJ) adenocarcinoma (locally advanced unresectable or metastatic, HER2-, CLDN18.2-positive, 1st-line)	IV	CRL	TBD

5-FU 5-Fluorouracil

6MWD 6 Minute Walking Distance

6MWT 6 Minute Walking Test

ABSSSI Acute Bacterial Skin and Skin Structure Infection

ACC American College of Cardiology

ACEI Angiotensin-Converting Enzyme Inhibitor

ACR20 American College of Rheumatology 20% Improvement

ACR50 American College of Rheumatology 50% Improvement

ACR70 American College of Rheumatology 70% Improvement

ADC Antibody-Drug Conjugate

ADHD Attention Deficit Hyperactivity Disorder

ADL Activities of Daily Living

ALK Anaplastic Lymphoma Kinase

ALL Acute Lymphoblastic Leukemia

ALS Amyotrophic Lateral Sclerosis

ALSFRS-R Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised

ALT Alanine Transaminase

AMD Age-Related Macular Degeneration

AML Acute Myeloid Leukemia

ANCA Antineutrophil Cytoplasmic Antibodies

ARB Angiotensin II Receptor Blocker

ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor

AS Ankylosing Spondylitis

ASCVD Atherosclerotic Cardiovascular Disease

AST Aspartate Aminotransferase

BCG Bacillus Calmette-Guérin

BCL-2 B Cell Lymphoma 2

BCVA Best Corrected Visual Acuity

BLA Biologics License Application

BMI Body Mass Index

BMT Bone Marrow Transplant

BP Blood Pressure

BPH Benign Prostatic Hyperplasia

BRAF V-raf Murine Sarcoma Viral Oncogene Homolog B1

BSA Body Surface Area

BsUFA Biosimilar User Fee Act

CABP Community Acquired Bacterial Pneumonia

CAP Community Acquired Pneumonia

CAR T Chimeric Antigen Receptor T-Cell

CD Crohn's Disease

CD3 Cluster of Differentiate 3

CD19 Cluster of Differentiate 19

CD20 Cluster of Differentiate 20

CD38 Cluster of Differentiate 38

CD79b Cluster of Differentiate 79b

CDC Centers for Disease Control and Prevention

CF Cystic Fibrosis

CHF Congestive Heart Failure

CI Confidence Interval

CKD Chronic Kidney Disease

CLL Chronic Lymphocytic Leukemia

CML Chronic Myeloid Leukemia

CMS Centers for Medicare & Medicaid Services

CNS Central Nervous System

COPD Chronic Obstructive Pulmonary Disease

COVID-19 Coronavirus Disease 2019

CRC Colorectal Cancer

CRL Complete Response Letter

CRR Complete Response Rate

CRS Cytokine Release Syndrome

CSF Colony Stimulating Factor

CTLA-4 Cytotoxic T-Lymphocyte-Associated Protein 4

CV Cardiovascular

CVD Cardiovascular Disease

CYP3A4 Cytochrome P-450 3A4

CYP450 Cytochrome P-450

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

DBP Diastolic Blood Pressure

DCR Disease Control Rate

DEA Drug Enforcement Administration

DED Dry Eye Disease

DLBCL Diffuse Large B Cell Lymphoma

DMARD Disease Modifying Antirheumatic Drug

DMD Duchenne Muscular Dystrophy

DME Diabetic Macular Edema

dMMR DNA Mismatch Repair

DMT Disease Modifying Therapy

DNA Deoxyribonucleic Acid

DOR Duration of Response

DPP-4 Dipeptidyl Peptidase 4

DR Delayed-Release

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

ECOG Eastern Cooperative Oncology Group

EDSS Expanded Disability Status Scale

eGFR estimated Glomerular Filtration Rate

EGFR Epidermal Growth Factor Receptor

ER Extended-Release

ERA Endothelin Receptor Agonist

ESA Erythropoietin Stimulating Agent

ESRD End-Stage Renal Disease

EUA Emergency Use Authorization

FDA Food and Drug Administration

FEV1 Force Expiratory Volume in 1 Second

FH Familial Hypercholesterolemia

FLT3 FMS-Like Tyrosine Kinase-3

FMS Feline McDonough Sarcoma

FVC Forced Vital Capacity

GABA-A Gamma-Aminobutyric Acid Receptor Type A

G-CSF Granulocyte Colony Stimulating Factor

GI Gastrointestinal

GIST Gastrointestinal Stromal Tumor

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

GM-CSF Granulocyte-Macrophage Colony Stimulating Factor

GVHD Graft Versus Host Disease

H Half

HAE Hereditary Angioedema

HAM-A Hamilton Anxiety Rating Scale

HAM-D Hamilton Depression Rating Scale

HAMD-17 Hamilton Depression Rating Scale

HAP Healthcare-Associated Pneumonia

Hb Hemoglobin

HbA1c Hemoglobin A1c

HBV Hepatitis B Virus

HCC Hepatocellular Carcinoma

HCP Healthcare Professional

HCV Hepatitis C Virus

HDRS-17 Hamilton Depression Rating Scale

HER Human Epidermal Growth Factor Receptor

HER2 Human Epidermal Growth Factor Receptor 2

HER2- Human Epidermal Growth Factor Receptor 2-negative

HF Heart Failure

HFA Hydrofluoroalkane

HFpEF Heart Failure with preserved Ejection Fraction

HFrEF Heart Failure with reduced Ejection Fraction

HIT Heparin Induced Thrombocytopenia

HIV Human Immunodeficiency Virus

HIV-1 Human Immunodeficiency Virus-1

HR Hazard Ratio

HR+ Hormone Receptor-positive

HS Hidradenitis Suppurativa

HSCT Hematopoietic Stem Cell Transplant

HSV Herpes Simplex Virus

HTN Hypertension

IBS Irritable Bowel Syndrome

IBS-C Irritable Bowel Syndrome, Constipation Predominant

ICS Inhaled Corticosteroid

IGA Investigator's Global Assessment

IgG Immunoglobulin G

IgG1 Immunoglobulin G1

IL-4 Interleukin-4

IL-12 Interleukin-12

IL-13 Interleukin-13

IL-17 Interleukin-17

IL-23 Interleukin-23

IM Intramuscular

IR Immediate-Release

IRB Institutional Review Board

ITP Immune Thrombocytopenic Purpura

ITT Intent-To-Treat

IV Intravenous

JAK Janus Kinase Inhibitor

JIA Juvenile Idiopathic Arthritis

KIT c-KIT Proto-Oncogene

LABA Long-Acting Beta Agonist

LAMA Long-Acting Muscarinic Antagonist

LDL-C Low-Density Lipoprotein Cholesterol

LPAD Limited Population Pathway for Antibacterial and Antifungal Drugs

LS Least Square

LVEF Left Ventricular Ejection Fraction

mAb Monoclonal Antibody

MACE Major Adverse Cardiovascular Events

MADRS Montgomery – Åsberg Depression Rating Scale

MDD Major Depressive Disorder

MDI Metered Dose Inhaler

MDR Multi-Drug Resistant

MECP2 Methyl-CpG Binding Protein 2

MEK Mitogen-Activated Extracellular Signal-Regulated Kinase

MI Myocardial Infarction

mITT modified Intent-To-Treat

MRI Magnetic Resonance Imaging

MRSA Methicillin-Resistant Staphylococcus Aureus

MS Multiple Sclerosis

MSI-H Microsatellite Instability-High

N/A Not Applicable

NAFLD Nonalcoholic Fatty Liver Disease

NASH Non-Alcoholic Steatohepatitis

NCCN National Comprehensive Cancer Network

NCT National Clinical Trials

NDA New Drug Application

NHL Non-Hodgkin Lymphoma

NIH National Institutes of Health

NRAS Neuroblastoma RAS Proto-Oncogene

NSAID Non-Steroidal Anti-Inflammatory Drug

NSCLC Non-Small Cell Lung Cancer

NTRK Neurotrophic Tyrosine Receptor Kinase

NYHA New York Heart Association

ODT Orally Disintegrating Tablet

OR Odds Ratio

ORR Objective Response Rate

OS Overall Survival

OTC Over-the-Counter

PAD Peripheral Arterial Disease

PAH Pulmonary Arterial Hypertension

PARP Poly (ADP-Ribose) Polymerase

PAS Prior Approval Supplement

PASI Psoriasis Area and Severity Index

PASI 50 Psoriasis Area and Severity Index 50% Reduction

PASI 75 Psoriasis Area and Severity Index 75% Reduction

PASI 90 Psoriasis Area and Severity Index 90% Reduction

PASI 100 Psoriasis Area and Severity Index 100% Reduction

PCI Percutaneous Coronary Intervention

PCSK9 Proprotein Convertase Subtilisin Kexin 9

PD-1 Programmed Death Protein 1

PD-L1 Programmed Death-Ligand 1

PDUFA Prescription Drug User Fee Application

PFS Progression-Free Survival

PGA Physician Global Assessment

PHI Primary Humoral Immunodeficiency

PI3K Phosphatidylinositol-3-kinase

PNH Paroxysmal Nocturnal Hemoglobinuria

PsA Psoriatic Arthritis

PSO Plaque Psoriasis

PTCA Percutaneous Transluminal Coronary Angioplasty

PTSD Post-Traumatic Stress Disorder

Q Quarter

QIDP Qualified Infectious Diseases Product

QOL Quality of Life

R/R Relapsed or Refractory

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

RA Rheumatoid Arthritis

RAS Ras Protein Superfamily

RBC Red Blood Cell

RCC Renal Cell Carcinoma

REMS Risk Evaluation and Mitigation Strategy

RMAT Regenerative Medicine Advanced Therapy

RNA Ribonucleic Acid

ROS1 ROS Proto-Oncogene 1

RPD Rare Pediatric Disease

RRR Relative Risk Reduction

RSV Respiratory Syncytial Virus

RTOR Real-Time Oncology Review

RVO Retinal Vein Occlusion

SARS-CoV-2 Severe Acute Respiratory Syndrome-Associated Coronavirus-2

sBLA supplemental Biologics License Application

SBP Systolic Blood Pressure

SC Subcutaneous

SCCHN Squamous Cell Cancer of the Head and Neck

SCD Sickle Cell Disease

SCLC Small Cell Lung Cancer

SCT Stem Cell Transplant

SGLT2 Sodium-Glucose Co-Transporter 2

SL Sublingual

SLE Systemic Lupus Erythematosus

SLL Small Lymphocytic Lymphoma

sNDA supplemental New Drug Application

SNRI Serotonin and Norepinephrine Reuptake Inhibitor

SOC Standard of Care

SOD-1 Superoxide Dismutase - Type 1

sPGA static Physician Global Assessment

SR Sustained-Release

SSRI Selective Serotonin Reuptake Inhibitor

SSSI Skin and Skin Structure Infection

T1DM Type 1 Diabetes Mellitus

T2DM Type 2 Diabetes Mellitus

TBD To Be Determined

TEAE Treatment-Emergent Adverse Event

TKI Tyrosine Kinase Inhibitor

TNBC Triple Negative Breast Cancer

TNF Tumor Necrosis Factor

TNFα Tumor Necrosis Factor-alpha

UA Unstable Angina

UC Ulcerative Colitis

U.S. United States

UTI Urinary Tract Infection

VAS Visual Analog Scale

VEGF Vascular Endothelial Growth Factor

VTE Venous Thromboembolism

WBC White Blood Cell

WHO World Health Organization

XR Extended-Release