San Diego – October 19, 2026 -- The U.S. Food and Drug Administration's Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) will meet on July 29, 2026, to review Capricor Therapeutics' Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD), less than four weeks before the agency's August 22, 2026 PDUFA target action date.
Panel meeting precedes the August 22 PDUFA decision date
Capricor (NASDAQ: CAPR) said its BLA remains on track against the August 22 deadline. The advisory meeting will be livestreamed. The company pointed to the Federal Register and its own website for meeting details.
BLA rests on three clinical datasets including a Phase 3 readout
The application is supported by the Phase 2 HOPE-2 trial and long-term outcomes from the HOPE-2-OLE trial. It also draws on results from the Phase 3 HOPE-3 trial, which Capricor says met statistical significance on its primary endpoint (PUL v2.0), the key secondary cardiac endpoint (LVEF), and all other Type I error-controlled secondary endpoints.
CEO Linda Marbán said the company has confidence in the totality of the evidence, citing statistically significant skeletal and cardiac benefits with a consistent safety profile across multiple studies.
Regulatory designations could yield a priority review voucher
Deramiocel (CAP-1002) is an allogeneic cell therapy made from cardiosphere-derived cells. It holds Orphan Drug Designation for DMD from both the FDA and the European Medicines Agency. It also carries Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and FDA Rare Pediatric Disease Designation. The last of these may qualify Capricor for a Priority Review Voucher upon approval.
DMD patient population and unmet need
DMD affects approximately 15,000 people in the United States, primarily boys. Heart failure from cardiomyopathy is the leading cause of death. There is no cure, and treatment options remain limited.
Nippon Shinyaku litigation remains a disclosed risk
Capricor's forward-looking statements flag its litigation with Nippon Shinyaku Co., Ltd. and NS Pharma, Inc. The company cites its ability to commercialize Deramiocel independently of its existing distribution agreement as a factor tied to that dispute. It also warns that required regulatory inspections could be delayed or unsuccessful, which would delay or prevent approval.
Deramiocel has not been approved for commercial use in any indication.