FDA Drug Approval Decisions Expected in March 2024

The Prescription Drug User Fee Act (PDUFA) date refers to the deadline set by the US Food and Drug Administration (FDA) for reviewing a New Drug Application (NDA) or Biologics License Application (BLA) and making a final decision on marketing approval. The typical period for review is 10 months after the drug application has been accepted by the Agency. For drugs that have Priority Review, the review period is reduced to 6 months from the time of application acceptance.

Resmetirom for Nonalcoholic Steatohepatitis (NASH) With Liver Fibrosis

PDUFA date: March 14, 2024

In patients with NASH, thyroid hormone beta activity in the liver is reduced, resulting in hepatic function impairment. Resmetirom is an oral thyroid hormone receptor (THR)-β selective agonist designed to target the underlying cause of NASH. The NDA submission included data from four phase 3 studies, including the pivotal MAESTRO-NASH trial (ClinicalTrials.gov Identifier: NCT03900429), which evaluated the efficacy and safety of resmetirom in more than 1000 patients with biopsy-proven NASH and fibrosis. Findings showed the trial met its dual primary endpoints: NASH resolution with at least a 2-point reduction in nonalcoholic fatty liver disease activity score (NAS) and with no worsening of fibrosis, or at least a 1-stage improvement in fibrosis with no worsening of NAS after 52 weeks of treatment.

Atidarsagene Autotemcel for Metachromatic Leukodystrophy

PDUFA date: March 18, 2024

Metachromatic leukodystrophy (MLD) is a rare inherited lysosomal storage disease caused by a mutation in the arylsulfatase-A gene. This results in a buildup of sulfatides in the brain and other areas of the body. Atidarsagene autotemcel is a hematopoietic stem cell-based gene therapy that was evaluated in a clinical trial that included 39 pediatric patients with early-onset MLD. At the time of analysis, treatment with the one-time gene therapy resulted in a statistically significant and clinically meaningful improvement in severe motor impairment-free survival. If approved, atidarsagene autotemcel would be the first and only treatment for early-onset MLD in the US.

Sotatercept for Pulmonary Arterial Hypertension

PDUFA date: March 26, 2024

Pulmonary arterial hypertension (PAH) is a progressive and life-threatening blood vessel disorder caused by hyperproliferation of cells in the arterial walls in the lung. The disease is characterized by the constriction of small pulmonary arteries and elevations in pulmonary arterial pressure. The BLA for sotatercept, a first-in-class activin receptor type IIA-Fc fusion protein, is supported by data from the double-blind, placebo-controlled phase 3 STELLAR trial. The study included 323 adult patients with PAH (WHO Group 1). Results showed a statistically significant and clinically meaningful improvement in 6-minute walk distance among patients treated with sotatercept compared with placebo.

Vadadustat for Anemia Due to Chronic Kidney Disease

PDUFA date: March 27, 2024

Vadadustat is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor designed to mimic the physiologic effect of altitude on oxygen availability. This results in increased red blood cell production and improved oxygen delivery to tissues. The NDA submission included data from 36 clinical trials involving over 8000 patients. Findings from the INNO2VATE program, which included 2 open-label, active-controlled phase 3 trials, showed that vadadustat was noninferior to darbepoetin alfa in adult patients on dialysis with anemia due to CKD. Vadadustat was also found to be noninferior to a long-acting erythropoiesis-stimulating agent for the maintenance treatment of anemia due to CKD, in hemodialysis patients in the phase 3 FO₂CUS study.

Omalizumab for Reducing Allergic Reactions to Multiple Foods

PDUFA date: March 2024

Omalizumab, an anti-immunoglobulin E (IgE) antibody, is under review for the reduction of allergic reactions, including anaphylaxis, that may occur with accidental exposure to 1 or more foods in adults and pediatric patients aged 1 year and older with food allergy. The BLA is supported by data from the phase 3 OUtMATCH trial, which evaluated the safety and efficacy of omalizumab in patients 1 to 55 years of age who are allergic to peanuts and at least 2 other common foods. Findings showed that compared with placebo, omalizumab significantly increased the amount of peanut (primary endpoint), milk, egg, and cashew (key secondary endpoints) participants could consume without an allergic reaction.

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