FDA Accepts Biologics License Application for Duchenne Muscular Dystrophy Cardiomyopathy Treatment

Core Insights - NS Pharma, a subsidiary of Nippon Shinyaku, has received FDA acceptance for the Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy, with a target action date of August 31, 2025 [1][2] - The FDA has granted Priority Review status for deramiocel, indicating a potential expedited review process [1] - NS Pharma is exclusively responsible for the commercialization and distribution of deramiocel in the U.S. under an agreement with Capricor Therapeutics [2] About Deramiocel - Deramiocel is composed of allogeneic cardiosphere-derived cells (CDCs), which have demonstrated immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies [3] - CDCs function by secreting exosomes that target macrophages, promoting a healing phenotype rather than a pro-inflammatory one [3] - Over 100 peer-reviewed publications have focused on CDCs, which have been administered to more than 200 human subjects in various clinical trials [3] About Duchenne Muscular Dystrophy - Duchenne muscular dystrophy primarily affects males and leads to progressive weakness and loss of skeletal, cardiac, and respiratory muscles [4] - Early symptoms include delayed motor skills, with significant mobility loss often requiring wheelchair use by adolescence [4] - Cardiac and respiratory complications typically arise in the teenage years, posing serious health risks [4] About Capricor Therapeutics - Capricor Therapeutics is a biotechnology company focused on cell and exosome-based therapeutics for rare diseases [5] - The company is developing its proprietary StealthX™ platform for targeted delivery of therapeutics, including oligonucleotides and proteins [5] About NS Pharma - NS Pharma is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd., dedicated to advancing treatments for rare diseases [6]