FDA Boosts Sarepta With Prognosis for its Rare Disease Drug

August 13, 2014

For the first time in months, Sarepta Therapeutics investors and parents of children with Duchenne muscular dystrophy received some good news. A top FDA official says the agency is exploring all “potential pathways” to approve new drugs to treat the rare childhood disease, which causes muscles in boys to stop working and eventually results in death because they can no longer breathe.

“We share your sense of urgency to make safe and effective drugs available for patients with Duchenne muscular dystrophy as soon as possible,” Janet Woodcock, who heads the FDA Center for Drug Evaluation and Research, wrote in response to a petition that was started on the ‘We the People’ page of the White House website. The petition urged the FDA to approve the Sarepta drug quickly.

Indeed, Woodcock specifically mentioned the possibility the agency would place the Sarepta drug in its accelerated approval program, in which the agency relies on a surrogate marker to suggest a clinical benefit. Sarepta recently released results of a small study showing improvements in the distance that boys could walk after taking the drug, an indication of improved lung function.

Her note comes after a series of setbacks surrounding Sarepta and its efforts to win FDA approval for its drug, which is called eteplirsen. Notably, the agency last November had told Sarepta not to seek accelerated approval, which sent its shares plunging and greatly disappointed many families who believe this is a breakthrough medication.

Although recent study results and talks with the agency suggested progress was being made was being made toward an FDA decision, Sarepta remained under a cloud with revelations that several employees have left in recent months. Last week, the chief scientific officer was fired after less than a year with the drug maker after raising concerns with board members about Chris Garabedian, the chief executive, and his managerial moves, The Wall Street Journal wrote.

The turnover prompted the board to limit Garabedian’s role in regulatory and medical affairs, as well as his participation in talks with the FDA, according to this story in The Wall Street Journal, which noted that Garabedian – a veteran of Celgene and Gilead Sciences, who is known as a forceful manager – had unusually wide latitude in decision making.

To what extent the note from Woodcock will amount to a significant change in fortunes remains to be seen. But the response is unusual. After Woodcock noted that Sarepta plans to file a new application for approval by the end of this year and initiate several additional clinical trials as well, she attempted to instill confidence in the review process by noting the agency carries out “our analyses expeditiously.”

One analyst says her response is a “very bullish” indication.

Read more here. 

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