CAMBRIDGE, Mass.– Scholar Rock (NASDAQ: SRRK) said Tuesday that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for its Biologics License Application (BLA) for apitegromab, an experimental treatment for spinal muscular atrophy (SMA). The CRL is tied solely to observations identified at Catalent Indiana LLC, a third-party fill-finish facility, and does not cite any concerns about apitegromab’s safety or efficacy data.
Catalent Indiana, acquired by Novo Nordisk A/S in December 2024, was the subject of a routine FDA inspection that resulted in observations unrelated to apitegromab itself. The company submitted a comprehensive response to regulators in August and continues to take corrective action. Scholar Rock said it plans to resubmit the apitegromab application once the facility’s issues are resolved.
“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, chairman and CEO of Scholar Rock. “We remain focused on working hand-in-hand with the FDA to pursue approval of the first and only muscle-targeted treatment for people living with SMA.”
Patient advocates emphasized the urgency of bringing apitegromab to market. “While we are disappointed that the availability of a muscle-targeted treatment approach for patients with SMA has been delayed, we remain enthusiastic about the transformative potential of apitegromab,” said Kenneth Hobby, president of Cure SMA. “Muscle strength and motor function are significant unmet needs for many in the SMA community and are fundamental to independence. A gain in motor function can allow someone to participate in important activities of daily living from self-care to work and social interactions, and as such, we urgently await the availability of the first-ever treatment with the potential to address the muscular component of SMA.”
Apitegromab has received Orphan Drug, Rare Pediatric Disease, Priority Review, and Fast Track designations in the United States. Outside the U.S., the European Medicines Agency is reviewing the company’s marketing application, with a decision expected by mid-2026. Scholar Rock anticipates a European launch in the second half of 2026, starting in Germany.
