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FDA Panel Votes Against Muscular Dystrophy Drug Eteplirsen

Approval decision expected by May 26

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has concluded that the investigational medication eteplirsen (Sarepta Therapeutics) is not effective for treating Duchenne muscular dystrophy, a fatal muscle-wasting disease that affects approximately 13,000 children, mostly boys. The panel voted 7 to 3 against the drug, with three abstentions. The FDA must decide by May 26 whether to follow its panel’s recommendation.

Prior to the committee’s public hearing, FDA staffers had released lengthy documentation that took a dim view of the drug.

The main issue was whether eteplirsen can sufficiently produce increased levels of the protein dystrophin. Without this protein, muscle fibers degenerate, preventing voluntary movement. The FDA staff had raised doubts about the clinical relevance of a small, 12-patient study that Sarepta relied on to make its case, as well as the viability of the six-minute walking tests that trial participants underwent.

In the new ruling, the FDA panel decided that eteplirsen failed to demonstrate that it could produce enough dystrophin to provide a clinical benefit.

Laura Gottschalk, a senior fellow at the National Center for Health Research, a nonprofit think tank, told the committee: “Unfortunately, the data do not meet a scientific standard of evidence of effectiveness. These boys and their families deserve better.”

Source: STAT; April 25, 2016.

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Decision supported by data from more than 4,000 patients