FDA panel votes 9-3 against Duchenne heart therapy

A U.S. Food and Drug Administration advisory panel voted on Wednesday, July 29, against recommending approval of an investigational cell therapy for a heart condition associated with Duchenne muscular dystrophy, citing insufficient evidence of its effectiveness. The Cellular, Tissue, and Gene Therapies Advisory Committee voted 9-3 that the therapy, deramiocel, did not provide substantial evidence for treating cardiomyopathy in patients with the rare genetic disease.

The vote follows a critical FDA staff review published on Monday, which concluded that the pivotal Phase III HOPE3 trial did not meet its pre-specified primary and secondary efficacy endpoints. The agency's analysis found no statistically significant difference between deramiocel and placebo in improving upper limb function or preserving a key measure of heart function, left ventricular ejection fraction. The dispute centers on which statistical analysis plan should be used to interpret the trial data. The FDA maintains its review used a plan finalized before the study began, while developer Capricor Therapeutics argues the agency relied on an early draft and that its own final analysis shows the therapy was effective.