FDA Advisory Committee Votes 9-3 Against Efficacy of Capricor’s Cell Therapy for DMD Cardiomyopathy

The FDA advisory committee voted 9-3 against recognizing the efficacy of Capricor’s cell therapy, deramiocel, submitted as a treatment for cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Amid continued disagreement between the company and the FDA over changes to the statistical analysis plan (SAP), the improvement in left ventricular ejection fraction (LVEF) did not reach statistical significance even in the analysis favored by the company (p=0.09). The FDA is not bound by the committee’s recommendation but plans to make a final decision on whether to approve deramiocel by Aug. 22.