Capricor Therapeutics Receives FDA Orphan Drug Designation For Becker Muscular Dystrophy And Reports Regulatory Advancement In Duchenne Muscular Dystrophy Program

Capricor Therapeutics, a biotechnology company focused on developing cell and exosome-based therapies for rare diseases, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its lead investigational therapy, Deramiocel, for the potential treatment of Becker Muscular Dystrophy (BMD). This designation is expected to provide several regulatory and commercial benefits, further supporting Capricor’s strategy as it builds an integrated platform to treat both cardiac and skeletal complications associated with muscular dystrophies.

Becker Muscular Dystrophy is a rare, progressive neuromuscular condition caused by mutations in the dystrophin gene. It leads to gradual weakening and deterioration of both skeletal and cardiac muscles, similar to Duchenne Muscular Dystrophy (DMD), but often with a later onset and slower progression. One of the major challenges in treating both conditions is cardiomyopathy, which significantly contributes to illness and death.

Linda Marbán, Ph.D., CEO of Capricor Therapeutics, said in a statement, “The granting of Orphan Drug Designation for Becker Muscular Dystrophy is a significant milestone that expands the potential reach of Deramiocel and reinforces our vision to deliver impactful therapies across a broader spectrum of neuromuscular diseases. We believe Deramiocel holds promise for patients with Becker, particularly given the overlap in disease pathology with Duchenne. Deramiocel has demonstrated potential to treat the serious cardiac and skeletal muscle complications of Duchenne Muscular Dystrophy, and based on the overlap in disease pathology, may also offer benefit to patients with Becker Muscular Dystrophy.”

Dr. Marbán also stated, “In addition, Capricor successfully completed its Pre-License Inspection, an important regulatory milestone for approval of its Biologics License Application (BLA) for DMD and we believe all review activities remain on track as we approach our PDUFA date. With key milestones aligning, we continue to prepare for the potential commercial launch of Deramiocel and the opportunity to deliver meaningful benefits to patients with Duchenne.”

Deramiocel is being developed to address the underlying disease mechanisms affecting both the heart and skeletal muscles in BMD patients. In addition to its development for BMD, Deramiocel is currently under priority review by the FDA for the treatment of Duchenne Muscular Dystrophy, with a target action date for the Biologics License Application (BLA) set for August 31, 2025. This latest regulatory milestone marks a key step for Capricor as it works to expand the reach and impact of its lead therapy in treating severe, life-limiting neuromuscular disorders.