Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab and granted it Priority Review. This designation indicates that apitegromab, if approved, could offer a significant improvement in the treatment of spinal muscular atrophy (SMA).
The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025, providing a clear timeline for a potential approval decision. Concurrently, the company's Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for apitegromab has been validated, initiating the formal review process.
These regulatory milestones are based on positive efficacy and safety data from the pivotal Phase 3 SAPPHIRE trial. Scholar Rock is planning for a U.S. commercial launch upon approval in 2025, with a European launch anticipated in 2026.
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