Scholar Rock resubmits apitegromab BLA to FDA for spinal muscular atrophy (SMA), targeting a late September 2026 decision following prior CRL tied to manufacturing observations.
Written By: Samiksha Jadhav, BPharm
Reviewed By: Pharmacally Editorial Team
Scholar Rock has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration for apitegromab, an investigational muscle-targeted therapy for children and adults with Spinal Muscular Atrophy.
The resubmission follows a Complete Response Letter (CRL) issued in September 2025, which cited manufacturing observations at Catalent Indiana, a facility operated by Novo Nordisk. The CRL raised no concerns regarding apitegromab’s clinical efficacy or safety. Scholar Rock, the FDA, and Catalent have since held multiple interactions, including a Type A meeting and an FDA site visit, with no additional corrective actions requested.
Apitegromab is a fully human monoclonal antibody that selectively inhibits myostatin activation by targeting its precursor forms in skeletal muscle. It is the first muscle-directed therapy to demonstrate statistically significant and clinically meaningful improvements in motor function in the pivotal Phase 3 SAPPHIRE trial (NCT05156320) among patients receiving standard SMN-targeted therapies.
The updated BLA incorporates a second U.S.-based fill-finish manufacturing site, added following FDA alignment in a March 2026 Type C meeting, to strengthen supply chain resilience and support anticipated global demand.
Scholar Rock expects FDA acceptance within 30 days, initiating a six-month review period with a PDUFA target action date in late September 2026. In Europe, the European Medicines Agency is reviewing the Marketing Authorisation Application (MAA), with a decision expected mid-2026.
Apitegromab has received Fast Track, Priority Review, Orphan Drug, and Rare Pediatric Disease designations from the FDA, along with PRIME and Orphan Medicinal Product designations from the EMA, underscoring its potential to address significant unmet needs in SMA.
Reference
Scholar Rock Resubmits Biologics License Application (BLA) to FDA for Apitegromab for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA), 31 March 2026, Scholar Rock Resubmits Biologics License Application (BLA) to FDA for Apitegromab for Treatment of Children and Adults with Spinal Muscular Atrophy (SMA) – Scholar Rock, Inc.
About the Writer
Samiksha Vikram Jadhav is a B.Pharm graduate with a strong academic foundation in pharmaceutical sciences, pharmacology, and drug development. She has a keen interest in healthcare advancements, clinical research, medical writing, and emerging therapies. Her work focuses on presenting developments in the pharmaceutical and healthcare sectors through clear and accurate scientific communication.