Capricor’s Deramiocel faced a major hurdle as an FDA Cellular, Tissue, and Gene Therapies Advisory Committee recommended against approval of the company’s resubmitted biologics license application. In a 9-3 vote with no abstentions, panelists concluded the evidence from the phase 3 HOPE-3 trial did not provide substantial evidence of effectiveness for Deramiocel in treating cardiomyopathy in Duchenne muscular dystrophy. The advisory committee vote is expected to influence the FDA’s decision timing, with a Prescription Drug User Fee Act (PDUFA) target date of August 22. The committee’s concerns followed an earlier negative FDA evaluation of the resubmitted BLA, including questions around endpoints, hypersensitivity rates versus placebo, and the absence of an updated statistical analysis plan submitted prior to the resubmission. Capricor described the therapy as an allogeneic cardiosphere-derived cell (CDC) product intended to modulate macrophage behavior, but panel scrutiny focused on whether the submitted clinical record met regulatory effectiveness standards for the specific DMD cardiomyopathy indication.