Capricor’s deramiocel was voted down by an FDA advisory committee, with members citing fragile evidence around cardiac benefit in Duchenne muscular dystrophy. The panel’s vote was 9-3 against recommending approval after contentious discussion focused on a Phase 3 HOPE-3 analysis of cardiac function. Capricor’s CEO Linda Marbán criticized how reviewers and the committee addressed a draft statistical analysis plan, arguing parts were hard to reconcile with the approval application and the trial’s framing. Despite patient testimony and the therapy’s context of high unmet need, adcom members concluded the available data did not show sufficient effectiveness for the cardiomyopathy indication. With the program already under a second FDA review after a prior rejection, the vote increases the probability of additional evidence requirements or redesign for next submission steps.