Apitegromab FDA Approval Status
Last updated by Judith Stewart, BPharm on Sep 23, 2025.
FDA Approved: No
Generic name: apitegromab
Company: Scholar Rock
Treatment for: Spinal Muscular Atrophy
Apitegromab is an investigational muscle-targeted therapy intended to improve motor function in people living with spinal muscular atrophy (SMA) who have been treated with certain existing SMA therapies.
- SMA is a rare genetic neuromuscular disease characterized by skeletal muscle weakness and atrophy. Symptoms include deterioration in mobility, swallowing, and breathing, and excessive fatigue and exhaustion.
- Apitegromab is a selective inhibitor of latent myostatin, a protein that regulates muscle mass. When myostatin is activated, it works with other growth factors and hormones to inhibit muscle growth. Apitegromab is a fully human monoclonal antibody that works to block the activation of myostatin in the muscle.
- A Biologics License Application (BLA) has been submitted to the U.S. Food and Drug Administration (FDA) for apitegromab to provide clinically meaningful improvement in motor function for people living with SMA who are receiving SMN-targeted treatments. The BLA submission is based on the Phase 3 SAPPHIRE trial that demonstrated a statistically significant improvement in motor function for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52.
- The FDA has granted apitegromab Fast Track, Orphan Drug and Rare Pediatric Disease Designations in SMA.
- On September 23, 2025, Scholar Rock announced that the FDA had issued a Complete Response Letter (CRL) for the apitegromab BLA for the treatment of patients with SMA. The CRL was related to observations identified during a routine general site inspection of Catalent Indiana LLC, a third-party fill-finish facility which was acquired by Novo Nordisk A/S in December 2024. The observations were not specific to apitegromab. The CRL did not cite any other approvability concerns, including apitegromab’s efficacy and safety data or the third-party drug substance manufacturer.
Development timeline for apitegromab
Further information
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