FDA Panel Rejects Duchenne Therapy Amid Broader Evidence Scrutiny

The FDA's Cellular, Tissue, and Gene Therapies Advisory Committee delivered a crushing blow to Capricor Therapeutics on Wednesday, voting 9-3 against recommending approval of Deramiocel, a cell therapy for cardiomyopathy in Duchenne muscular dystrophy. The panel concluded the Phase III HOPE-3 trial did not "provide substantial evidence of effectiveness," setting up an August 22 decision date that now looks bleak.

Vital Signs

• 9-3 vote — FDA advisory committee...