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Capricor Therapeutics Announces FDA Advisory Committee Meeting to Review BLA for Deramiocel for the Treatment of Duchenne Muscular Dystrophy

CAPR
  • Capricor Therapeutics (NASDAQ: CAPR) announced a Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) meeting scheduled for July 29, 2026, to discuss its Biologics License Application (BLA) for Deramiocel.
  • The FDA's PDUFA target action date for the Deramiocel BLA is set for August 22, 2026.
  • The BLA seeks approval of Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), supported by Phase 2 HOPE-2, long-term HOPE-2-OLE, and Phase 3 HOPE-3 trial data.
  • The Phase 3 HOPE-3 trial achieved statistical significance on its primary endpoint (PUL v2.0), the key secondary cardiac endpoint (LVEF), and all other Type I error-controlled secondary endpoints.
  • Deramiocel consists of allogeneic cardiosphere-derived cells (CDCs) that secrete exosomes to exert immunomodulatory & anti-fibrotic effects.
  • The therapy holds Orphan Drug Designation from the U.S. FDA & European Medicines Agency, Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, & Rare Pediatric Disease Designation from the FDA.
  • The CTGTAC meeting will be available for live streaming.
  • No cure currently exists for DMD, which affects approximately 15,000 individuals in the U.S. & primarily impacts boys.