FDA panel rejects Capricor's Duchenne drug, citing insufficient efficacy data

The U.S. Food and Drug Administration advisory committee voted 9-to-3 against a Capricor Therapeutics candidate for Duchenne muscular dystrophy. The panel said the clinical data did not provide substantial evidence that the therapy improves the disease, particularly its heart complications.

The decision focuses on the drug's ability to treat Duchenne-related cardiomyopathy, a key endpoint for approval. Without convincing results, the agency cannot recommend the product for market authorization.

Capricor will need to generate additional data or modify its approach before resubmitting. The setback highlights the challenges of developing effective treatments for rare genetic disorders.