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FDA Accepts Biologics License Application for Duchenne Muscular Dystrophy Cardiomyopathy Treatment
PARAMUS, N.J., March 10, 2025 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that
About this update from Capricor Therapeutics, Inc.
[{"type":"text","content":"PARAMUS, N.J., March 10, 2025 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), announced today that acceptance has been received by Capricor Therapeutics, Inc. (Headquarters: California, USA, CEO: Linda Marbán, NASDAQ: CAPR) from the U.S. Food and Drug Administration (FDA) for the Biologics License Application (BLA) filing for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (\"DMD\") cardiomyopathy. The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (\"PDUFA\") target action date of August 31, 2025, and at this time, the FDA has not identified any potential review issues.\n\n \n \n \n \n \n \n\n \n\"Deramiocel has the potential to address a clear, unmet medical need for patients diagnosed with DMD.\"Nippon Shinyaku and Capricor entered into an exclusive distribution agreement for deramiocel for the U.S. in January 2022. NS Pharma will be exclusively responsible for commercialization and distribution of deramiocel in the U.S.\n\"Deramiocel has the potential to address a clear, unmet medical need for patients diagnosed with DMD,\" said NS Pharma President, Yukiteru Sugiyama, Ph.D. \"We are excited for the possibility to bring additional treatment options and renewed hope to families of the rare disease community.\"\nFor more details, please see the press release from Capricor.https://www.capricor.com/investors/news-events/press-releases/detail/305/capricor-therapeutics-announces-fda-acceptance-and-priority\nAbout DeramiocelDeramiocel consists of allogeneic cardiosphere-derived cells (\"CDCs\"), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory, antifibrotic and regenerative actions in dystrophinopathy and heart failure. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.\nAbout Duchenne Muscular Dystrophy (Duchenne)Duchenne is a form of muscular dystrophy that occurs primarily in males. It causes progressive we...