Business Wire·Aug 7, 2026, 4:20 PM ET

Scholar Rock Announces FDA Review of Apitegromab Biologics License Application (BLA) for Spinal Muscular Atrophy (SMA) to Progress with Second Fill-Finish Facility; Approval Decision Anticipated by September 30, 2026 Action Date

On August 7, 2026, Scholar Rock received notification that the FDA classified the April 2026 inspection of Catalent Indiana LLC's fill-finish facility as Official Action Indicated (OAI).
Scholar Rock will remove Catalent Indiana from the apitegromab Biologics License Application (BLA) under FDA guidance while collaborating closely on the review process.
FDA review of the second U.S.-based fill-finish facility is progressing, with the required data package submitted ahead of the timeline agreed upon during the March 2026 Type C meeting.
A robust commercial supply of apitegromab vials from the second facility is available at a third-party site, awaiting packaging, labeling, and FDA approval.
The apitegromab BLA remains on track for a potential FDA approval by the September 30, 2026 PDUFA action date, with a U.S. launch planned immediately upon approval.
Scholar Rock is engaging with the European Medicines Agency (EMA) to include the second fill-finish facility in the apitegromab Marketing Authorisation Application (MAA).
The second facility is in good standing with both the FDA and EMA, demonstrating a strong compliance track record through recent successful inspections.
Apitegromab is an investigational myostatin inhibitor for spinal muscular atrophy (SMA) that holds Fast Track, Orphan Drug, Rare Pediatric Disease, PRIME, and Orphan Medicinal Product designations from the FDA and EMA.