Skip to Main Content

In rather blunt language, a team of Food and Drug Administration reviewers believe that a drug developed by Sarepta Therapeutics is unlikely to benefit children suffering from Duchenne muscular dystrophy. The rare disease causes muscles in boys to stop working and eventually results in death because they can no longer breathe. About 20,000 new cases are diagnosed each year.

Their assessment appears throughout various portions of a 73-page document that was released on Friday in advance of a meeting next week of FDA experts. The panel will review clinical trial data for the drug, which is known as eteplirsen, and may be asked to vote on whether to recommend the agency should approve the treatment. Wall Street reacted by sending Sarepta stock plummeting more than 50 percent in midday trading.


“The news is grim,” wrote Needham analyst Chad Messer in an investor note.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!


Comments are closed.