Capricor Therapeutics Announces Key Regulatory Updates for its Duchenne Muscular Dystrophy Program

Core Insights - Capricor Therapeutics has successfully completed the FDA Pre-License Inspection for its manufacturing facility in San Diego for Deramiocel, its lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD) [1][5] - The inspection resulted in a Form 483 with several observations related to quality systems and documentation practices, but no material changes to the cGMP process were required [1][5] - The FDA Advisory Committee meeting is scheduled for July 30, 2025, with a PDUFA action date for the Biologics License Application (BLA) set for August 31, 2025 [2][5] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel being a key product in late-stage development for DMD [7] - The company has received Orphan Drug Designation from both the FDA and EMA for Deramiocel, which is supported by RMAT and ATMP designations [5][6] - Capricor has entered into an exclusive commercialization agreement for Deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [9] Product Information - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic effects in preserving muscle function in DMD [4][7] - DMD is a severe genetic disorder affecting approximately 15,000-20,000 individuals in the U.S., characterized by progressive muscle weakness and inflammation [3][4] - The pathophysiology of DMD involves impaired dystrophin production, leading to muscle cell damage and heart failure, which is the leading cause of death in DMD patients [3]