Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy

Core Insights - Capricor Therapeutics is advancing its lead cell therapy candidate, Deramiocel, for the treatment of Duchenne Muscular Dystrophy (DMD) associated cardiomyopathy, with a Biologics License Application (BLA) under Priority Review by the FDA, targeting an action date of August 31, 2025 [1][2][3] Regulatory Updates - The FDA has indicated that an Advisory Committee meeting is not required at this time, and the BLA remains under Priority Review [2][7] - A late-cycle review meeting is scheduled for mid-July 2025 [7] Clinical Data - Capricor presented four-year data from its HOPE-2 Open-Label Extension study at the 2025 Parent Project Muscular Dystrophy Conference, showing sustained cardiac function and clinical benefits from long-term Deramiocel treatment [3][7] Product Information - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have demonstrated immunomodulatory and anti-fibrotic actions in preserving cardiac and skeletal muscle function in DMD [5][8] - The product has received multiple designations, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy designation from the FDA [6][10] Market and Collaboration - Capricor has entered into an exclusive commercialization agreement for Deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [10]