Capricor’s deramiocel was voted down by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee in a contentious meeting. Panelists voted 9-3 against recommending approval for the Duchenne muscular dystrophy therapy after discussions centered on whether HOPE-3 trial evidence showed substantial effectiveness for cardiomyopathy endpoints. FDA reviewers and committee experts highlighted what they described as “very fragile” benefit signals, along with disagreements over statistical analysis-plan handling during the review cycle. Capricor’s CEO Linda Marbán challenged the agency’s framing, arguing the committee was pressured to consider draft statistical work not intended for the application. The decision stops short of a final FDA action, but it keeps deramiocel’s path uncertain as the company prepares for its ongoing second review posture.