Sarepta Therapeutics announced that the FDA postponed an advisory committee meeting scheduled for January 22 to discuss the company's filing for the Duchenne muscular dystrophy (DMD) therapy eteplirsen due to an anticipated severe winter snowstorm.
In briefing documents posted ahead of the meeting, FDA staff suggested that Sarepta had not provided enough evidence to support the effectiveness of eteplirsen, with the reviewers questioning the validity of a "small" clinical study used to establish the efficacy of the therapy. The review caused the company's share price to fall as much as 58 percent.
The FDA indicated that the date of the meeting to review eteplirsen will be announced later. Sarepta added that if there is a change to the agency's target review date of February 26 then it will provide an update at that time.
Earlier this month, the FDA declined to approve BioMarin Pharmaceutical's DMD therapy Kyndrisa (drisapersen).
For related analysis, see ViewPoints: "First do no harm" – Sarepta's eteplirsen at least has that going for it, which is nice, and ViewPoints: BioMarin's rough ride at an FDA AdComm may be good news for Sarepta, PTC Therapeutics.
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