FDA doubts efficacy of Capricor’s Duchenne stem-cell therapy after Phase 3 miss

The U.S. Food and Drug Administration said Monday that Capricor Therapeutics’ stem‑cell treatment for Duchenne muscular dystrophy failed to meet the primary goals of its Phase 3 trial, contradicting the company’s earlier statements.

Capricor had announced in December that its drug, deramiocel, achieved both primary and secondary endpoints in a large, randomized study, a claim that now faces regulatory scrutiny.

The trial focused on teenagers and young men who had already lost the ability to walk, a group with limited treatment options. Capricor had previously reported that the therapy preserved upper‑arm function and delayed heart failure in these patients.

The FDA’s concerns raise uncertainty about the drug’s path forward and could affect investor confidence in the company’s rare‑disease pipeline.