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Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA)
Scholar Rock’s BLA submission is based on the Phase 3 SAPPHIRE trial that demonstrated a statistically significant improvement in motor function for patients
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n\nScholar Rock’s BLA submission is based on the Phase 3 SAPPHIRE trial that demonstrated a statistically significant improvement in motor function for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52\n\n\n\nThe FDA has granted apitegromab Fast Track, Orphan Drug and Rare Pediatric Disease Designations in SMA\n\n\n\nScholar Rock remains on track to submit the apitegromab Marketing Authorisation Application (MAA) to the European Medicines Agency in 1Q 2025, with PRIME and Orphan Medicinal Product Designations\n\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today announced the submission of a Biologics License Application to the U.S. Food and Drug Administration (FDA) for apitegromab, a muscle-targeted therapy designed and developed to provide clinically meaningful improvement in motor function for people living with SMA who are receiving SMN-targeted treatments. The Company remains on track to file a Marketing Authorisation Application to the European Medicines Agency in 1Q 2025.\n\n\n“We are gratified that in patients already on a SMN-targeted treatment, the SAPPHIRE trial met its primary endpoint for the main efficacy population showing a statistically significant 1.8-point improvement for patients receiving apitegromab compared to placebo, as measured by the Hammersmith Functional Motor Scale-Expanded at week 52,” said Jing Marantz, M.D., Ph.D., Chief Medical Officer of Scholar Rock. “With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA on behalf of patients and families living with SMA.”\n\n\nThe review of the apitegromab BLA submission will be conducted by the Division of Neurology Products in FDA’s Center for Drug Evaluation and Research. The submission is supported by data from the Phase 3 SAPPHIRE trial and the Phase 2 TOPAZ trial. The Company shared positive topline data from the SAPPHIRE trial in 2024, and additional data will be presented at the 2025 MDA Clinical & Scientific Conference ...