Scholar Rock's Apitegromab Receives Priority Review from FDA for Spinal Muscular Atrophy Treatment, PDUFA Date Set for September 2025

Reuters
23 Jun
Scholar Rock's Apitegromab Receives Priority Review from FDA for Spinal Muscular Atrophy Treatment, PDUFA Date Set for September 2025

Scholar Rock Holding Corporation, a late-stage biopharmaceutical company, announced that its Biologics License Application $(BLA.AU)$ for apitegromab, a treatment for spinal muscular atrophy $(SMA)$, has been accepted under priority review by the U.S. Food and Drug Administration (FDA). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025. Additionally, the European Medicines Agency $(EMA)$ has validated Scholar Rock's Marketing Authorisation Application $(MAA)$ for apitegromab for patients with SMA. This marks a significant step forward for the company in its mission to develop and commercialize treatments for debilitating neuromuscular diseases.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Scholar Rock Holding Corporation published the original content used to generate this news brief via Business Wire (Ref. ID: 20250623065428) on June 23, 2025, and is solely responsible for the information contained therein.

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