July 27 (Reuters) – U.S. Food and Drug Administration staff reviewers raised concerns about the effectiveness of Capricor Therapeutics’ 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.
(Reporting by Sahil Pandey and Christy Santhosh in Bengaluru; Editing by Devika Syamnath and Leroy Leo)
