Scholar Rock (NASDAQ:SRRK) announced on Tuesday, March 31, 2026, that it has officially resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for apitegromab.
The investigational therapy is positioned to become the first muscle-targeted treatment for children and adults living with spinal muscular atrophy (SMA), a rare and debilitating neuromuscular disease.
The resubmission follows a Complete Response Letter (CRL) received in September 2025, which was tied to general site inspection observations at a Catalent Indiana facility.
Crucially, the FDA did not cite any concerns specific to apitegromab itself or its clinical data.
Following a series of constructive meetings in late 2025 and early 2026, Scholar Rock moved forward with the resubmission in alignment with the FDA’s feedback on the facility's remediation progress.
To ensure a resilient launch and meet anticipated global demand, the resubmitted BLA now includes a second U.S.-based fill-finish facility.
This strategic addition follows a positive Type C meeting with the FDA earlier this month, allowing the company to accelerate its commercial supply chain timelines.
Management expects the FDA to accept the BLA within 30 days, with a standard six-month review period leading to a projected PDUFA action date in late September 2026.
Apitegromab has demonstrated statistically significant improvements in motor function in pivotal Phase 3 trials when used alongside existing SMN-targeted therapies.
The drug's potential has already earned it several prestigious regulatory statuses, including Fast Track, Orphan Drug, and Priority Review designations in the United States, as well as PRIME designation in Europe.
In addition to the U.S. progress, Scholar Rock noted that the European Medicines Agency (EMA) review of its Marketing Authorisation Application (MAA) is advancing as planned.
A regulatory decision in Europe is anticipated by mid-2026.