FDA Advisory Committee Votes Against Effectiveness of Capricor’s Duchenne Cell Therapy

Rare Daily Staff

A U.S. Food and Drug Administration advisory committee voted against the effectiveness of Capricor Therapeutics’ lead cell therapy, deramiocel, for treating cardiomyopathy in Duchenne muscular dystrophy, dealing a potential setback weeks before a key regulatory decision.

The FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 9–3 that available evidence does not support deramiocel’s effectiveness for DMD-related cardiomyopathy, the company announced Thursday. The vote is nonbinding, and the FDA is expected to issue a final decision by the therapy’s Aug. 22 PDUFA date.

The panel considered a narrower indication than the company had proposed and did not formally assess the therapy’s overall benefit-risk profile, Capricor noted. Company executives emphasized that the discussion included more favorable feedback on skeletal muscle outcomes, particularly upper limb function measured by the PUL 2.0 scale in the Phase 3 HOPE-3 trial.

The advisory committee’s negative vote introduces uncertainty for deramiocel’s regulatory path, particularly given the FDA’s heightened scrutiny of functional endpoints and clinical meaningfulness in neuromuscular and rare disease settings. However, the agency is not bound by the panel’s recommendation and has, in some cases, diverged from advisory committee votes.

“We remain confident in the strength of the HOPE-3 data,” said Capricor CEO Linda Marbán, adding that patient and clinician testimony during the open public hearing underscored the high unmet need in Duchenne.

Duchenne muscular dystrophy is a rare, progressive genetic disorder that primarily affects boys. While advances in skeletal muscle therapies have emerged, there are currently no approved treatments specifically targeting the cardiomyopathy that ultimately drives mortality in the disease.

Deramiocel (CAP-1002) is an allogeneic cell therapy derived from cardiosphere-derived cells, designed to exert immunomodulatory and anti-fibrotic effects through exosome signaling. The therapy has been studied in more than 250 patients and has received multiple regulatory designations, including RMAT and orphan drug status in the United States.

Capricor said it will continue working with the FDA as it approaches the decision deadline.

Photo: Capricor CEO Linda Marbán