SAN DIEGO – October 19, 2026 -- The FDA's Cellular, Tissue and Gene Therapies Advisory Committee voted 9-3, with no abstentions, that available evidence did not support the effectiveness of Capricor Therapeutics' Deramiocel for cardiomyopathy in patients with Duchenne muscular dystrophy (DMD), leaving the company's PDUFA target action date of August 22, 2026 as the next decision point.
Non-binding vote addresses narrower indication than Capricor proposed
The committee's vote is non-binding. Capricor (NASDAQ: CAPR) said the voting question covered a narrower indication than the one it had proposed and did not include a vote on Deramiocel's overall benefit-risk profile.
Cardiomyopathy in DMD currently has no approved treatment. Capricor said heart muscle deterioration leads to cardiomyopathy and heart failure, the leading cause of death in DMD, which affects approximately 15,000 people in the United States.
Panel feedback on upper limb function described as directionally supportive
In a separate discussion on upper limb function, the committee's feedback was directionally supportive of clinical evidence from the Phase 3 HOPE-3 trial, according to Capricor. That includes results on the trial's primary endpoint, PUL 2.0. No vote was reported on that question.
Capricor targets approval ahead of August 22 PDUFA date
CEO Linda Marbán said the company remains confident in the HOPE-3 data and is focused on working with the FDA toward potential approval before the PDUFA date. She said the committee's discussion of Deramiocel's impact on skeletal muscle was encouraging.
Deramiocel (CAP-1002) is an allogeneic cardiosphere-derived cell therapy. It holds Orphan Drug Designation from both the FDA and the European Medicines Agency, as well as RMAT designation in the U.S. and ATMP designation in Europe. Its Rare Pediatric Disease Designation from the FDA may qualify Capricor for a Priority Review Voucher upon approval.
The company's forward-looking statements also reference ongoing litigation with Nippon Shinyaku Co., Ltd. and NS Pharma, Inc., including Capricor's ability to commercialize Deramiocel independent of its existing distribution agreement. Deramiocel remains investigational and is not approved for commercial use in any indication.