FDA advisory panel rejects Capricor's Duchenne heart-cell therapy deramiocel

An FDA advisory committee voted 9-3 to reject Capricor Therapeutics' experimental cell therapy deramiocel for Duchenne-related cardiomyopathy. The panel said the data did not provide substantial evidence of benefit.

Capricor had reported that deramiocel met the primary endpoints of its Phase 3 trial in patients with heart complications from Duchenne muscular dystrophy. The company previously faced a full FDA rejection of the therapy last July.

The advisory panel's skeptical view reflects concerns about the robustness of the data submitted. The FDA has agreed to review a revised submission and is expected to issue a final decision by August 22.

The outcome highlights the challenges rare-disease developers may face when seeking flexible regulatory pathways and could influence how future rare-disease therapies are evaluated.