Capricor Therapeutics Responds to FDA Posting of Complete Response Letter (CRL) for Deramiocel

Core Viewpoint - Capricor Therapeutics is addressing the FDA's Complete Response Letter (CRL) regarding its investigational cell therapy Deramiocel for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) and is committed to transparency and collaboration with the FDA to resolve outstanding issues [1][2][3] Company Overview - Capricor Therapeutics (NASDAQ: CAPR) is focused on developing cell and exosome-based therapeutics for rare diseases, with Deramiocel as its lead product candidate [7] - The company has received multiple designations for Deramiocel, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA [6] Product Details - Deramiocel (CAP-1002) is composed of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory and anti-fibrotic effects in preserving cardiac and skeletal muscle function in DMD [5] - The therapy has been studied in over 250 peer-reviewed publications and administered to more than 250 human subjects in clinical trials [5] Regulatory Status - The FDA's CRL was received in July 2025, and Capricor plans to make its preliminary response to the FDA publicly available for transparency [2][3] - The company is awaiting official minutes from a recent Type A meeting with the FDA, which will help define the next steps in the regulatory process [3] Disease Context - Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder affecting approximately 15,000 individuals in the U.S., primarily boys, leading to progressive muscle degeneration and heart failure [4]