Capricor Therapeutics, a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Deramiocel (CAP-1002), the company’s lead cell therapy candidate, for the potential treatment of Becker Muscular Dystrophy (BMD). This designation strengthens Capricor’s strategic position as it advances a fully integrated platform targeting the cardiac and skeletal complications of muscular dystrophy and expands the commercial potential of its lead asset. – What is Deramiocel? What Does CAP-1002 Do? –
Capricor Therapeutics Proceeds with Confident Steps
- U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s focus in neuromuscular diseases
- Capricor remains on track for the August 31, 2025, The Prescription Drug User Fee Act (PDUFA) date for Deramiocel in Duchenne Muscular Dystrophy following successful FDA Pre-License Inspection
Becker Muscular Dystrophy, like Duchenne Muscular Dystrophy (DMD), is a progressive X-linked neuromuscular disorder that results in significant skeletal and cardiac muscle deterioration over time. Deramiocel is being developed to address both aspects of disease pathology, including cardiomyopathy, a primary contributor to morbidity and mortality in patients with both BMD and DMD.
Capricor’s Biologics License Application (BLA) for Deramiocel in DMD remains under priority review, with a PDUFA target action date of August 31, 2025.
Learn More: Capricor Therapeutics Announces Completion of Review Meeting
