Shares of Scholar Rock (NASDAQ: SRRK) rose about 12% on Tuesday following the companys resubmission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration for apitegromab, a treatment intended for children and adults with spinal muscular atrophy (SMA).
The updated BLA specifically includes Catalent Indiana LLC, which is identified as part of Novo Nordisk, along with a second U.S.-based fill-finish facility. Scholar Rock said the decision to proceed with resubmission before an FDA reinspection of the Catalent Indiana site was reached in consultation with the FDA after interactions in the first quarter of 2026.
In September 2025 the company received a Complete Response Letter from the FDA stemming from observations recorded during a routine inspection of the Catalent Indiana facility. Scholar Rock noted that those observations were not specific to apitegromab and that the CRL did not raise any additional approvability concerns for the drug itself.
On timing, Scholar Rock is projecting that the FDA will accept the resubmitted BLA within 30 days. The company expects the agencys review to run up to six months from the resubmission date and has stated a PDUFA action date is expected in late September 2026.
Apitegromab is described by the company as the first and only muscle-targeted therapy to show statistically significant and clinically meaningful improvements in motor function in a pivotal Phase 3 trial among patients with SMA who were receiving treatment with an SMN-targeted therapy.
The therapy has received multiple regulatory designations from the FDA, including Fast Track, Orphan Drug, Priority Review, and Rare Pediatric Disease status. Separately, the European Medicines Agency is advancing its review of the Marketing Authorisation Application for apitegromab, with a decision anticipated in mid-2026.
Clear summary
Scholar Rock resubmitted a BLA for apitegromab naming two U.S. fill-finish sites, including Catalent Indiana LLC. The company expects FDA acceptance within 30 days and a review period of up to six months, with an anticipated PDUFA action date in late September 2026. The drug has multiple special FDA designations and is undergoing review in Europe with an expected mid-2026 decision.
Market reaction
The announcement prompted a roughly 12% rise in Scholar Rocks share price on the day of the filing update.
Regulatory timeline and context
- The original September 2025 Complete Response Letter related to inspection observations at Catalent Indiana, not to apitegromab specifically.
- The resubmission was coordinated with the FDA following first quarter 2026 interactions and includes a second U.S. fill-finish facility in addition to Catalent Indiana.
- Scholar Rock projects FDA acceptance within 30 days and a review window of up to six months, targeting a PDUFA action in late September 2026.
Clinical and regulatory designations
According to the company, apitegromab is the first muscle-targeted therapy to demonstrate statistically significant and clinically meaningful motor function improvements in a pivotal Phase 3 trial among SMA patients on SMN-targeted treatment. The FDA has granted Fast Track, Orphan Drug, Priority Review, and Rare Pediatric Disease designations. The European Medicines Agencys review of the Marketing Authorisation Application is progressing with a decision expected mid-2026.
Key points
- Scholar Rock resubmitted its BLA for apitegromab, including Catalent Indiana LLC and another U.S. fill-finish site.
- The company expects FDA acceptance within 30 days and a review period of up to six months, with a PDUFA action date targeted for late September 2026.
- Regulatory designations by the FDA and parallel review at the European Medicines Agency are underway, with a mid-2026 decision expected in Europe.
Risks and uncertainties
- The Complete Response Letter issued in September 2025 stemmed from inspection observations at Catalent Indiana; while those observations were not specific to apitegromab, they represent a regulatory hurdle tied to a manufacturing site.
- The timing assumptions - acceptance within 30 days and a review of up to six months leading to a late September 2026 PDUFA date - are projections and dependent on FDA processes and review outcomes.
- The European decision anticipated in mid-2026 is still pending as the EMA review of the Marketing Authorisation Application continues.
Note: This article reports on the companys statements and regulatory timelines as provided by Scholar Rock.