Capricor’s deramiocel (allogeneic cell therapy) was rejected by an FDA advisory committee after a meeting centered on conflicting interpretations of the HOPE-3 trial’s statistical analysis plan. The CTGT panel voted 9-3 against recommending approval, with committee members describing the available evidence as “very fragile.” Debate focused on whether the FDA and company used comparable statistical analysis approaches and whether trial endpoints could be properly distinguished from analysis artifacts. Capricor argued the trial’s success was based on an analysis plan version finalized prior to unblinding, while FDA reviewers criticized the company’s handling of an earlier draft plan that became obsolete during the trial. The decision arrives after deramiocel had already been rejected once and is now under the FDA’s second review cycle. Panel member comments also reflected the tension between the urgent need in advanced DMD and the committee’s assessment that the evidence presented did not meet the threshold for substantial proof of effectiveness.