CAMBRIDGE, Mass. — Scholar Rock has resubmitted its Biologics License Application to the U.S. Food and Drug Administration for apitegromab, an investigational therapy for children and adults with spinal muscular atrophy.
The resubmission follows discussions with the FDA earlier this year and includes updates related to manufacturing, including the addition of Catalent Indiana LLC, part of Novo Nordisk, and a second U.S.-based fill-finish facility. The company said the filing aligns with guidance received during a March 3, 2026 Type C meeting with regulators.
Scholar Rock expects the FDA to accept the application within 30 days, followed by a review period of up to six months. A regulatory decision is anticipated in late September 2026.
Apitegromab is designed as a muscle-targeted therapy and has shown statistically significant and clinically meaningful improvements in motor function in a Phase 3 clinical trial involving patients already receiving SMN-targeted treatments.
“Our apitegromab BLA resubmission marks an important step forward in our mission to bring the world’s first muscle-targeted therapy to children and adults living with SMA,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “We continue to be encouraged by the FDA’s engagement and shared sense of urgency as we work relentlessly for the SMA community. As we execute our plans for both Catalent Indiana and our second fill-finish facility, we look forward to anticipated apitegromab approvals and launches in both the U.S. and Europe this year.”
The resubmission follows a Complete Response Letter issued by the FDA in September 2025 tied to observations from a routine inspection of the Catalent Indiana manufacturing facility. The company said the issues were not specific to apitegromab and did not involve concerns about the drug’s safety or efficacy.
Since then, Scholar Rock, Catalent, and the FDA have held multiple meetings, including an in-person Type A meeting in November 2025 and additional discussions in early 2026. The company said no further corrective actions were requested following a subsequent FDA site visit.
The updated application also includes a second fill-finish facility to support supply chain resilience and anticipated global demand. Scholar Rock said the addition was made in agreement with the FDA as part of efforts to accelerate commercial readiness.
In Europe, the company said its Marketing Authorisation Application for apitegromab is under review by the European Medicines Agency, with a decision expected in mid-2026.
The FDA has granted apitegromab several regulatory designations, including Fast Track, Orphan Drug, Priority Review, and Rare Pediatric Disease status, while European regulators have provided PRIME and Orphan Medicinal Product designations.
