Capricor’s DMD Therapy Rejected 9-3 by FDA Panel

Capricor Therapeutics’ cell therapy deramiocel for Duchenne muscular dystrophy cardiomyopathy was judged by an FDA advisory committee to have failed to demonstrate efficacy in a 9-3 vote. The company claimed that the therapy met its goals in a phase 3 trial, including preserving cardiac ejection capacity, but the FDA countered that changes to the statistical analysis reduced confidence in the data. The FDA is scheduled to decide whether to approve deramiocel by Aug. 22, and the likelihood of rejection has increased after the panel’s recommendation.