FDA Grants Orphan Drug Designation to Capricor’s Deramiocel for Becker Muscular Dystrophy

Capricor Therapeutics has received Orphan Drug Designation from the U.S. Food and Drug Administration for its lead cell therapy candidate, Deramiocel, aimed at treating Becker Muscular Dystrophy (BMD). This designation broadens the potential applications of Deramiocel beyond Duchenne Muscular Dystrophy (DMD), targeting both cardiac and skeletal muscle complications common to these progressive X-linked neuromuscular disorders. Deramiocel has demonstrated potential to address cardiomyopathy, a major cause of morbidity and mortality in both BMD and DMD patients. The company recently completed a successful Pre-License Inspection for its Biologics License Application for DMD, with a priority review and a PDUFA target action date set for August 31, 2025. Capricor's CEO, Linda Marbán, emphasized that the designation is a significant milestone that expands the therapy’s reach and underscores the company’s commitment to delivering impactful treatments for a broader spectrum of neuromuscular diseases. Financially, Capricor remains stable, with positive market performance and optimistic revenue growth forecasts supporting its ongoing development and potential commercial launch of Deramiocel.