FDA advisory panel votes 9-3 against approval of Capricor’s Duchenne muscular dystrophy therapy deramiocel

The FDA Cellular, Tissue, and Gene Therapies Advisory Committee focused heavily on the Phase 3 HOPE-3 trial’s secondary endpoint of cardiac function, describing the data as very fragile.

Committee members voted 9-3 to recommend against approving deramiocel, Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy.

Capricor CEO Linda Marbán expressed frustration that the agency reviewed a draft statistical analysis plan that was not part of the original submission. The therapy is under a second FDA review after being rejected a year earlier.

While the FDA is not required to follow advisory recommendations, it often does. Members highlighted the fragility of the results as a key concern.