FDA advisory panel advises against approving Capricor’s Duchenne cell therapy, deramiocel

The FDA Cellular, Tissue, and Gene Therapies Advisory Committee convened to evaluate deramiocel, a cell‑based treatment for Duchenne muscular dystrophy developed by Capricor Therapeutics.

Capricor highlighted Phase 3 results that showed the therapy preserved upper‑arm function and delayed the onset of heart failure, meeting both primary and secondary endpoints with statistical significance.

Despite the presented data, the advisory committee voted to recommend against approval, citing concerns that outweighed the reported benefits.

The negative recommendation could postpone access for teenagers and young men with Duchenne who have lost the ability to walk and may impact the company’s development timeline and investor outlook.