FDA advisory panel votes 9-3 against Capricor's Duchenne therapy deramiocel

During an FDA Cellular, Tissue, and Gene Therapies Advisory Committee meeting, reviewers focused on the Phase 3 HOPE-3 trial's secondary cardiac endpoint and described the data as very fragile. The panel voted 9-3 against recommending approval of Capricor's cell therapy deramiocel.

Capricor CEO Linda Marbán expressed frustration, saying the agency's briefing document included a draft statistical analysis plan that was never part of the original submission. Her remarks did not sway the committee.

While the FDA is not required to follow advisory committee recommendations, it often does. The negative vote highlights concerns about the robustness of the trial results and adds uncertainty to the therapy's path forward.