Capricor Says FDA Grants Orphan Drug Designation For Deramiocel To Treat Becker Muscular Dystrophy

(RTTNews) - Biotechnology company Capricor Therapeutics, Inc. (CAPR) announced Tuesday that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Deramiocel, 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.

The company said 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.

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.