Capricor Therapeutics Announces Initiation of Rolling Submission of Biologics License Application (BLA) with U.S. FDA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy

Core Viewpoint - Capricor Therapeutics has initiated a rolling submission process for a Biologics License Application (BLA) for deramiocel, aimed at treating Duchenne muscular dystrophy (DMD) cardiomyopathy, with plans to complete the submission by the end of 2024 and potential eligibility for priority review by the FDA [1][2]. Group 1: Company Overview - Capricor Therapeutics is a biotechnology company focused on developing cell and exosome-based therapeutics for rare diseases, with deramiocel (CAP-1002) as its lead product candidate [6]. - The company has entered into an exclusive commercialization agreement for deramiocel in the U.S. and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [9]. Group 2: Product Details - Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory, antifibrotic, and regenerative effects in preclinical and clinical studies [3]. - The product has received Orphan Drug Designation and is supported by the Regenerative Medicine Advanced Therapy (RMAT) designation [4]. Group 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, with a median mortality age of around 30 years [5]. - There are currently no approved treatment options for DMD cardiomyopathy, highlighting the significance of deramiocel as a potential first-in-class therapy [2][5].