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FDA Advisory Committee to Review Capricor's Duchenne Muscular Dystrophy Therapy

Capricor Therapeutics said the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee plans to meet on July 29, 2026, to review the company’s biologics license application for deramiocel, its investigational cell therapy for Duchenne muscular dystrophy.

The company’s PDUFA target action date remains Aug. 22, 2026.

Capricor said the application is backed by the Phase 2 HOPE-2 trial, long-term data from HOPE-2-OLE, and Phase 3 HOPE-3 results. In HOPE-3, the company said deramiocel hit statistical significance on its primary endpoint, PUL v2.0, the key secondary cardiac endpoint, LVEF, and all other type I error-controlled secondary endpoints.

The advisory committee meeting comes about six weeks before the FDA’s decision deadline. Capricor said the meeting will be livestreamed.

The company said Duchenne muscular dystrophy affects about 15,000 people in the U.S. and that heart failure is the leading cause of death in the disease. Deramiocel has been studied in more than 250 peer-reviewed publications and administered to over 250 human subjects across multiple clinical trials. The market has reacted to these announcements by moving the company's shares -13.03% to a price of $26.44. Check out the company's full 8-K submission here.

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