FDA Panel Votes Down Duchenne Muscular Dystrophy Drug Eteplirsen

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FDA Panel Votes Down Duchenne Muscular Dystrophy Drug Eteplirsen
FDA Panel Votes Down Duchenne Muscular Dystrophy Drug Eteplirsen

The FDA's Peripheral and Central Nervous System Drugs Advisory Committee handed down yet another negative vote for the latest drug meant to address Duchenne muscular dystrophy (DMD) in patients with mutations amendable to exon 51 skipping.

The 8-5 vote signifies that panel members feel the drug, Sarepta's etiplirsen, does not meet the minimum standards for accelerated approval based on inadequate efficacy. In November of last year, the same panel also opposed the clinical evidence for Biomarin Pharmaceutical's drisapersen, which is meant to treat the same condition.

Multiple hours of patient and family testimony complicated the vote, which was held off-site in order to accommodate the hundreds of civilians in attendance for the public comment period, however ultimately, panel members felt that the claims presented by families did not match the study findings.

Expressing concern from the start, William Dunn, MD, director of the FDA's Office of Drug Evaluation I for the Division of Neurology Products, made a plea to the panel. “We come to you with sincere concerns, not because we take some perverse delight in keeping new medicines from those who urgently need them ... but because it is our — we the FDA, and you, our advisory committee — collectively, it is our fundamental responsibility to ensure, as required by law, that the treatments we approve are effective.”

The panel found several issues with Sarepta's single clinical trial and exploratory studies, which had no adequate placebo control, including an overestimation of the percent of dystrophin-positive muscle fibers and discrepancies in clinical endpoints, including the 6-minute walk test, which was the primary endpoint.

Ultimately, the committee voted 7-6 against the finding that the studies show that eteplirsen induces production of dystrophin at a level that is reasonably likely to predict clinical benefit, and 7-3 with 3 abstentions against the finding that there is substantial evidence from the historically-controlled study that indicate eteplirsen is effective for the treatment of DMD.

While the FDA is not required to follow the advice of the panel, it often does. That vote is scheduled for May 26, 2016.

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