Quarterly Drug Pipeline

January 31, 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 + Magellan Rx Quarterly 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, PharmD, Editor-In-Chief, Vice President, Clinical Information
Carole Kerzic, RPh, Executive Editor, Clinical Pharmacist, Drug Information
Nicole Kjesbo, PharmD, BCPS, Executive Editor, Clinical Program Director, Pipeline

Consultant Panel

Robert Greer, RPh, BCOP, Vice President, Clinical Strategy and Programs; 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

Nothing herein is or shall be construed as a promise or representation regarding past or future events and Prime Therapeutics/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 Prime Therapeutics/Magellan Rx Management.

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 drug names appear in magenta throughout the publication.

Gene therapy/Metabolic — atidarsagene autotemcel

atidarsagene autotemcel  IV


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 trial

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
Respiratory – ensifentrine

ensifentrine  inhaled


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 trial

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.

PLACE IN THERAPY

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.

FDA APPROVAL TIMELINE

June 26, 2024

FINANCIAL FORECAST (reported in millions)

Year 2024 2025 2026 2027 2028
Projected total U.S. sales $36 $121 $259 $442 $631
Gene therapy/Hematology – fidanacogene elaparvovec

fidanacogene elaparvovec IV


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 trial

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
Endocrine – insulin icodec

insulin icodec  SC


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 trial

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
Gene therapy/Immunology – marnetegragene autotemcel

marnetegragene autotemcel  IV


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 trial

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

Infectious disease – respiratory syncytial virus (RSV) vaccine, mRNA-1345

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


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 trial

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
Gene therapy/Dermatology – prademagene zamikeracel

prademagene zamikeracel topical surgical application


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
Oncology – rivoceranib + camrelizumab

rivoceranib oral + camrelizumab IV


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.

Cardiovascular – sotatercept

sotatercept  SC


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

Pipeline

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 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.

 

 

Table view
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

 

Pipeline

Drug list

The 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

Submitted new drugs
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
Submitted supplemental drugs
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
Phase 3 new drugs
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
Phase 3 supplemental drugs

None

Submitted new drugs

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

Submitted supplemental drugs

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

Phase 3 new drugs

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

Phase 3 supplemental drugs

None

Biosimilars

Submitted new drugs
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
Submitted supplemental drugs
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
Phase 3 new drugs
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
Phase 3 supplemental drugs

None

Submitted new drugs

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

Submitted supplemental drugs

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

Phase 3 new drugs

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

Phase 3 supplemental drugs

None

Specialty

Submitted new drugs
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
Submitted supplemental drugs
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
Phase 3 new drugs
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
Phase 3 supplemental drugs
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

Submitted new drugs

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

Submitted supplemental drugs

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

Phase 3 new drugs

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

Phase 3 supplemental drugs

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

Submitted new drugs
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
Submitted supplemental drugs
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
Phase 3 new drugs
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
Phase 3 supplemental drugs
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

Submitted new drugs

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

Submitted supplemental drugs

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

Phase 3 new drugs

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

Phase 3 supplemental drugs

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
Complete response letter
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

Glossary of terms

Glossary of terms

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

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