An independent FDA advisory panel is meeting today to decide whether a cell therapy should be recommended for approval to treat the heart disease that has become the leading cause of death in Duchenne muscular dystrophy. Two days before the meeting, the FDA published a briefing document saying the pivotal trial did not show the therapy works.
The Cellular, Tissue, and Gene Therapies Advisory Committee convened at 9:30 a.m. Eastern at the FDA's White Oak campus in Silver Spring, Maryland, and the session is being livestreamed. The product is deramiocel, an investigational cell therapy from Capricor, Inc., submitted under Biologics License Application 125842 for the treatment of cardiomyopathy in male patients with Duchenne muscular dystrophy.
