(RTTNews) - Capricor Therapeutics, Inc. (CAPR) announced that the U.S. Food and Drug Administration's Cellular, Tissue, and Gene Therapies Advisory Committee will meet to discuss the company's Biologics License Application seeking approval of Deramiocel in the treatment of Duchenne muscular dystrophy.
The BLA is supported by data from the Phase 2 HOPE-2 trial, long-term follow-up data from the HOPE-2 open-label extension (OLE) study, and positive results from the Phase 3 HOPE-3 trial, which met its primary endpoint of Performance of Upper Limb (PUL) v2.0 and key secondary cardiac endpoint of left ventricular ejection fraction (LVEF).
The Advisory Committee meeting is scheduled for July 29, 2026, while the final decision on the Biologics License Application remains on track for an August 22, 2026.
Duchenne muscular dystrophy (DMD) is a rare X-linked progressive neuromuscular disorder caused by mutations in the DMD gene. The condition results in the complete absence of dystrophin, a protein required to maintain muscle structure and function.
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a population of cardiac cells that, in preclinical and clinical studies, has demonstrated immunomodulatory and anti-fibrotic effects aimed at preserving cardiac and skeletal muscle function in muscular dystrophies such as DMD.
Deramiocel has received Orphan Drug Designation for the treatment of DMD from both the U.S. FDA and the European Medicines Agency (EMA).
In addition, the therapy has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which could make Capricor eligible for a Priority Review Voucher upon approval.
CAPR is currently trading down 12.11% to $26.72.
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