FDA to review Sarepta drug

The FDA made clear that its guidance doesn't reflect how it will vote on Sarepta's drug, and said the company should be prepared for an advisory committee meeting to evaluate the application before the agency makes its decision, Sarepta said.

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"They haven't reversed their decision from November," Sarepta Chief Executive Chris Garabedian said in a telephone interview. "They've reinforced the questions and concerns they had of being able to accept an NDA and consider accelerated approval of just the data set they had in-house, but evolved to say, 'If you give us a little more data, it should be fileable.'"

Garabedian is expected to appear on "Street Signs" at 2 p.m.

The last study Sarepta conducted involved just 12 patients, a very small set for the FDA to consider. But Duchenne muscular dystrophy is a rare, fatal condition for which no other treatment options exist. And the company argues its data, most recently reported out to 120 weeks in these 12 patients, show the drug helps stem declines in walking ability.

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FDA to review Sarepta drug