The FDA granted Orphan Drug Designation to Capricor Therapeutics’ Deramiocel for treating Becker Muscular Dystrophy (BMD). This designation expands Deramiocel’s potential use beyond Duchenne Muscular Dystrophy (DMD), for which it is currently under priority review with a PDUFA date of August 31, 2025. This follows a successful Pre-License Inspection by the FDA, keeping Deramiocel’s DMD BLA on track.
This FDA decision is crucial because it recognizes the potential of Deramiocel to address a significant unmet medical need in BMD. While DMD and BMD are distinct conditions, they share similar disease mechanisms, particularly the development of cardiomyopathy, a leading cause of death in both diseases. Deramiocel’s potential to address both the cardiac and skeletal muscle deterioration seen in these dystrophies positions it as a potentially impactful therapy for a broader patient population within the neuromuscular disease spectrum. This expansion could significantly increase the market potential of Deramiocel.
Deramiocel is an allogeneic cell therapy derived from cardiosphere-derived cells (CDCs). These cells release exosomes which appear to promote a healing response in damaged muscle tissue. The Orphan Drug Designation for BMD, along with previous designations for DMD (including Orphan Drug, RMAT, ATMP, and Rare Pediatric Disease), underscores the innovative nature of this cell therapy and its potential to address serious, life-threatening complications of these rare diseases. Capricor has a commercialization agreement with Nippon Shinyaku for Deramiocel in the United States and Japan for DMD, pending regulatory approval.
This recent designation strengthens Capricor’s position in the rare disease space. A positive outcome for Deramiocel in DMD could pave the way for its development and potential approval in BMD, offering a new treatment option for patients with this debilitating condition. It also validates Capricor’s platform technology and sets the stage for further exploration of CDC-based therapies in other neuromuscular diseases. Looking ahead, Capricor is focused on preparing for the potential commercial launch of Deramiocel for DMD and continuing research on its exosome technology platform.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
