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FDA Grants Priority Review for Biologics License Application (BLA) and EMA Accepts Marketing Authorisation Application (MAA) for Apitegromab as a Treatment for Spinal Muscular Atrophy
Apitegromab remains on track to be the first and only muscle-targeted treatment for people living with Spinal Muscular Atrophy FDA to review BLA application
About this update from Scholar Rock Holding Corporation
[{"type":"text","content":"\n\nApitegromab remains on track to be the first and only muscle-targeted treatment for people living with Spinal Muscular Atrophy\n\n\n\nFDA to review BLA application under priority review, with a PDUFA date of September 22, 2025\n\n\n\nApitegromab Marketing Authorisation Application to the European Medicines Agency validated and under review\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for neuromuscular diseases, cardiometabolic disorders, and other serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment that is being developed to provide clinically meaningful improvement in motor function for people living with spinal muscular atrophy (SMA) who are receiving an SMN-targeted treatment. The FDA will review the application under priority review and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2025. The FDA priority review designation conveys that the FDA has determined that if apitegromab is approved, it could offer significant improvement in the safety or effectiveness of treatment of the serious condition of SMA.\n\nThe Company has also submitted and received validation for its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for apitegromab for the treatment of SMA. Validation confirms that the application is complete and the formal review process by EMA can begin.\n\n“We are delighted with the priority review designation for apitegromab, which is consistent with the potential of apitegromab to be a transformative therapy and the first muscle-targeted treatment for people living with SMA who continue to have decline in motor function despite receiving SMN-targeted treatments,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock. “In addition to the positive news from the FDA, we are pleased with the parallel acceptance of the MAA by the EMA and look forward to continue working with regulatory agencies to bring apitegromab to the SMA community as we prepare for our global commercial launch, starting with the U.S.”\n\nApitegromab’s regulatory submissions are based on positi...