FDA Committee Rejects Capricor's Deramiocel for Duchenne Cardiomyopathy
An FDA advisory committee has voted against approving Capricor Therapeutics' cell therapy, deramiocel, for treating cardiomyopathy in patients with Duchenne muscular dystrophy. The committee cited 'fragile' efficacy data and issues with the study's design as reasons for their decision. The vote was 9 to 3 against approval, indicating a challenging path ahead for Capricor before the FDA's final decision on August 22. The HOPE-3 Phase III trial, which tested deramiocel, showed significant slowing of upper-limb function decline but did not achieve statistical significance in cardiac improvement. The FDA reviewers criticized the statistical analysis and handling of missing data, noting that most patients had preserved cardiac function at baseline, limiting conclusions about treating established cardiomyopathy. 
