Capricor Therapeutics(US:CAPR) Globenewswireยท2025-03-04 12:00Core Insights - The FDA has accepted Capricor Therapeutics' Biologics License Application (BLA) for deramiocel, targeting Duchenne muscular dystrophy (DMD) cardiomyopathy, with a PDUFA action date set for August 31, 2025 [1][2] - If approved, deramiocel would be the first therapy specifically for DMD cardiomyopathy, a condition currently lacking approved treatments [1][2] - The BLA is supported by data from Capricor's Phase 2 clinical trials, indicating the potential effectiveness of deramiocel in treating DMD cardiomyopathy [2][3] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapeutics for rare diseases, with deramiocel as its lead product candidate [7] - The company has received Orphan Drug Designation from the FDA and EMA for deramiocel, which is also recognized under the Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. [3] - Capricor has entered into an exclusive commercialization agreement for deramiocel with Nippon Shinyaku Co., Ltd. for the U.S. and Japan markets, pending regulatory approval [9] Product Details - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory, antifibrotic, and regenerative properties in clinical studies [4] - The therapy is designed to be administered quarterly and aims to provide a lifelong treatment option for patients with DMD cardiomyopathy [2][3] - Clinical trials have demonstrated deramiocel's ability to attenuate DMD cardiomyopathy, which is a leading cause of death in DMD patients [3] Disease Context - Duchenne muscular dystrophy (DMD) is a severe genetic disorder affecting approximately 15,000-20,000 individuals in the U.S., characterized by progressive muscle weakness and inflammation [5][6] - The median age of mortality for DMD patients is around 30 years, with heart failure being the leading cause of death due to the disease's impact on heart muscle cells [5][6]