FDA Skips Advisory Committee For Capricor's Duchenne Cell Therapy

Capricor Therapeutics Inc CAPR on Tuesday provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy candidate for the treatment of Duchenne Muscular Dystrophy (DMD)—associated cardiomyopathy.

DMD is an X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles.

It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys.

Also Read: Vinay Prasad’s Appointment To FDA’s CBER Triggers Questions Over Future Of Cell And Gene Therapy Regulation

As part of the FDA’s ongoing review, the company has been informed that an Advisory Committee meeting is not indicated at this time.

The BLA remains under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of August 31, 2025.

As per some media reports, Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research (CBER), canceled the advisory committee meeting for the company’s cell therapy for Duchenne muscular dystrophy.

The FDA informed Capricor of its intent to hold the Advisory Committee meeting on July 30, 2025, although that date is pending confirmation by the FDA. At the time of the mid-cycle review, no significant issues or major deficiencies were noted.

Linda Marbán, CEO of Capricor, says the company has hit all key regulatory milestones as planned, including a successful pre-license inspection and a smooth mid-cycle review. With the application still under Priority Review, she believes Capricor is in a strong position heading into the PDUFA date.

Capricor recently presented four-year data from its HOPE-2 Open-Label Extension (OLE) study at the 2025 Parent Project Muscular Dystrophy (PPMD) Conference, representing one of the longest-running treatment datasets in DMD, including measures of both cardiac and skeletal muscle function.

Price Action: CAPR stock is up 20% at $9.22 at the last check on Tuesday

Read Next: