Perspectives
Quarterly Drug Pipeline
January 31, 2024Clinical insights and competitive intelligence on anticipated drugs in development.
Drug pipeline
Clinical insights and competitive intelligence on anticipated drugs in development.
Table of contents
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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