U.S. Food and Drug Administration staff reviewers have raised concerns about the effectiveness of Capricor Therapeutics’ cell therapy in treating a heart condition related to Duchenne muscular dystrophy, a rare inherited disease that causes progressive muscle weakness and primarily affects boys.
Background
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. 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.
The concerns raised by FDA staff reviewers have dragged the company’s shares 67% lower in premarket trading.
Original reporting: Appleton, WI News Feed (HLL/CB) — read the source article.
