Scholar Rock recently held an in-person Type A meeting with the FDA on November 12 to discuss the Biologics License Application (BLA) for apitegromab, a potential treatment for children and adults with SMA.
Cure SMA was present and active at this FDA meeting to represent our community to ensure that the patient voice and the patient need were heard and factored into decisions. In particular stressing the urgency needed to address unmet needs across the entire age spectrum in SMA.
The meeting was constructive, with Catalent Indiana, LLC—the third-party facility referenced in the FDA’s earlier Complete Response Letter (CRL)—confirming it remains on track to be ready for reinspection by the end of this year. Importantly, the CRL did not raise any concerns regarding apitegromab’s safety or efficacy.
Scholar Rock now anticipates resubmitting the BLA in 2026, with a potential U.S. launch following approval. To help accelerate timelines, they have also added a second U.S.-based fill-finish facility, with commercial capacity expected to begin in early 2026.
"“We are unwavering in our commitment to bring apitegromab, the world’s first and only muscle-targeted treatment to improve motor function, to children and adults living with SMA. We are grateful to the FDA, Cure SMA, and our colleagues at Novo Nordisk for the positive engagement at our in-person Type A meeting earlier this week. We are encouraged by the discussion and by our shared understanding of the urgency to bring this important treatment to the SMA community as rapidly as possible." - David L. Hallal, Chairman and Chief Executive of Scholar Rock
This meeting marks an important step toward bringing a new treatment option to the SMA community, highlighting the ongoing collaboration between industry, regulators, and patient advocacy groups to accelerate therapies safely and effectively.
- Completed Type A meeting with FDA. On November 12, 2025, Scholar Rock had a Type A meeting with the U.S. Food and Drug Administration (FDA). The meeting was held in person and was constructive. Catalent Indiana, LLC (part of Novo Nordisk) joined Scholar Rock at the meeting and detailed the progress it has made in implementing the remediation plan, and affirmed that it expects the facility to be ready for reinspection by the end of this year. Resubmission of the BLA and U.S. launch following approval of apitegromab for children and adults with SMA is anticipated in 2026.
- Accelerated timelines with second fill-finish facility. Scholar Rock has accelerated timelines with an additional fill-finish facility for apitegromab. The facility is a commercially approved facility that is based in the U.S. and has a proven track record of successful site inspections. Tech transfer is underway, and commercial capacity has been reserved beginning in the first quarter of 2026.