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Capricor Therapeutics Gains Orphan Drug Status for Becker Muscular Dystrophy, Advances Duchenne Program
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Capricor Therapeutics has secured Orphan Drug Designation from the FDA for Deramiocel, its leading cell therapy candidate, specifically for the treatment of Becker Muscular Dystrophy (BMD). This is a crucial step for the company as BMD, like the more severe Duchenne Muscular Dystrophy (DMD), causes progressive deterioration of both skeletal and cardiac muscle, with heart complications being a major cause of death. Deramiocel is designed to address these issues, and its promising results in DMD, for which it's already under priority review with an August 31, 2025 PDUFA date, suggest it could also benefit BMD patients due to the shared disease pathology. This designation not only broadens the potential market for Deramiocel but also strengthens Capricor's strategic position in developing therapies for a wider range of neuromuscular disorders.