FDA rejects Scholar Rock's apitegromab application due to facility issues

The U.S. Food and Drug Administration issued a Complete Response Letter rejecting Scholar Rock's (NASDAQ: SRRK) application for apitegromab, a treatment for spinal muscular atrophy, due to manufacturing observations at a third-party facility.

The rejection relates solely to issues identified during an inspection of Catalent Indiana LLC, a fill-finish facility acquired by Novo Nordisk A/S in December 2024. The FDA did not cite any other concerns regarding apitegromab's efficacy, safety data, or drug substance manufacturing.

Scholar Rock plans to resubmit the biologics license application once Catalent Indiana resolves the FDA's manufacturing observations. Catalent submitted a response to address the observations in August 2025 and continues corrective actions.

"We are continuing to work closely with Catalent Indiana on the FDA's manufacturing observations so that we can resubmit the apitegromab BLA as soon as possible," said David L. Hallal, Scholar Rock's chairman and chief executive officer.

The FDA had granted apitegromab several designations including Orphan Drug, Rare Pediatric Disease, Priority Review, and Fast Track status. The drug targets myostatin to improve muscle function in SMA patients.

In Europe, the marketing authorization application remains under review by the European Medicines Agency, with a decision expected near mid-2026. Scholar Rock anticipates a European launch in the second half of 2026, beginning with Germany.

Spinal muscular atrophy affects approximately 35,000 patients globally who have been treated with existing therapies that target motor neurons but do not directly address muscle function.

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