http://www.wsj.com/articles/parents-tell-fda-about-benefits-of-mu...

http://www.wsj.com/articles/parents...cy-has-doubts-about-1461622924?mod=rss_Health


FDA will make final decision on May 26. They do not necessarily have to accept Advisory Panel's negative decision of today but will likely do so.

FDA Panel Votes Not to Recommend Approval for Muscular Dystrophy Drug
ByThomas M. Burton
The Wall Street Journal
Updated April 25, 2016 8:48 p.m. ET

COLLEGE PARK, Md.—An advisory panel to the Food and Drug Administration voted not to recommend approval of a drug for the crippling disease called Duchenne muscular dystrophy.
The advisory committee voted 7-3, with three abstentions, on the central question of whether evidence of effectiveness for the drug eteplirsen from Sarepta Therapeutics Inc. SRPT 35.66 % had been proven by a single, small study of just 12 patients.
“I felt this wasn’t a well-controlled study,” said panel chairman G. Caleb Alexander, an associate epidemiology professor at the Johns Hopkins School of Public Health. The panel members who abstained said they were undecided because they were moved by public testimony from parents who believed their children had been helped by the drug.
The decision followed decidedly negative comments from FDA reviewers who concluded that the study fell far short of producing enough evidence.

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