Capricor Therapeutics (CAPR) announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to Deramiocel.
Deramiocel is the lead cell therapy candidate developed for the potential treatment of Becker Muscular Dystrophy (BMD).
This designation strengthens Capricor Therapeutics’ strategic position as it advances a fully integrated platform targeting the cardiac and skeletal complications of muscular dystrophy.
It also expands the commercial potential of its lead asset.
Capricor Therapeutics has its Biologics License Application (BLA) for Deramiocel in Duchenne Muscular Dystrophy (DMD) currently under priority review.
The target action date set by the FDA under the Prescription Drug User Fee Act (PDUFA) is August 31, 2025.