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An advisory panel to the Food and Drug Administration gave an unfavorable review on Tuesday to drisapersen, an experimental drug for Duchenne muscular dystrophy, voting that inconsistencies in clinical trial data suggested the BioMarin Pharmaceutical treatment is unlikely to work.

There was no official up-down vote on whether to recommend approval. But 15 of 17 panel members did indicate that the lack of statistical significance in a late-stage study of 186 patients weakened positive outcomes seen in two smaller and earlier studies. The FDA is expected to make a final decision on the drug byDec. 27.


The vote came after FDA staff cited “life-threatening” side effects and a lack of “substantial evidence” of effectiveness in documents released last week.

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