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Advisory Committee Finds Insufficient Evidence for Duchenne’s Drug’s Efficacy

Oct. 3, 2017

An FDA advisory committee voted 10 to 1 Wednesday that a muscular dystrophy drug candidate does not have enough supporting data to be considered effective.

The Peripheral and Central Nervous System Drugs Advisory Committee voted on the efficacy of PTC Therapeutics’ drug candidate Ataluren.

Aaron Kesselheim of Brigham and Women’s Hospital, who voted with the majority, noted that despite some compelling evidence for the drug’s efficacy, much of it was from post hoc examinations of pre-existing trials.

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