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Scholar Rock Resubmits Biologics License Application (BLA) to FDA for Apitegromab for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA)
Apitegromab BLA resubmission includes Catalent Indiana LLC (part of Novo Nordisk) and second U.S.-based fill-finish facility, aligned with FDA guidance from
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n\nApitegromab BLA resubmission includes Catalent Indiana LLC (part of Novo Nordisk) and second U.S.-based fill-finish facility, aligned with FDA guidance from March 3, 2026 Type C meeting\n\n\n\nFDA and Novo Nordisk Q1 2026 interactions resulted in Scholar Rock’s alignment with FDA to resubmit apitegromab BLA prior to FDA reinspection of Catalent Indiana facility\n\n\n\nFDA and Scholar Rock Q1 2026 interactions regarding accelerated progress in qualifying a second fill-finish facility resulted in alignment with FDA to include facility in BLA resubmission\n\n\n\nCompany anticipates FDA acceptance of BLA within 30 days and a review period of up to 6 months from date of resubmission, with PDUFA action date expected in late September 2026\n\n\n\nManagement to host a conference call today at 8:00am ET\n\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today announced it has resubmitted the Biologics License Application (BLA) for apitegromab for the treatment of children and adults with SMA to the U.S. Food and Drug Administration (FDA).\n\n\nApitegromab is the first and only muscle-targeted therapy to demonstrate statistically significant and clinically meaningful improvements in motor function in a pivotal Phase 3 clinical trial in patients with SMA receiving treatment with an SMN-targeted therapy.\n\n\n“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.”\n\n\nIn September 2025, Scholar Rock received a Complete Response Letter (CRL) from the FDA for apitegromab related to observations identified during a routine general s...