FDA staff flags concerns about Capricor's Duchenne heart disease therapy
CAPR•FDA reviewers raise effectiveness concerns
U.S. Food and Drug Administration staff reviewers raised concerns about the effectiveness of Capricor Therapeutics' CAPR.O cell therapy in treating a heart condition related to Duchenne muscular dystrophy, dragging the company's shares 67% lower in premarket trading.
The FDA released its briefing documents on Monday, ahead of its external advisers' meeting on Wednesday to discuss the effectiveness and risk-benefit profile of the therapy, deramiocel.
Duchenne muscular dystrophy is a rare inherited disease that causes progressive muscle weakness and primarily affects boys.
There are currently no FDA-approved therapies specifically for Duchenne muscular dystrophy-associated cardiomyopathy, a form of heart disease that commonly develops as the disease progresses.
