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Muscular dystrophy drug looks set for commercial success despite clinical doubts

BMJ 2016; 355 doi: https://doi.org/10.1136/bmj.i5346 (Published 03 October 2016) Cite this as: BMJ 2016;355:i5346
  1. Owen Dyer
  1. Montreal

A newly approved drug to treat Duchenne muscular dystrophy seems likely to be used by most US neurologists, even though its benefit has yet to be proved, treatment costs $300 000 (£230 000; €270 000) a year, and the head of the Food and Drug Administration has said that the main clinical study on which it rests “should probably be retracted by its authors.”

Eteplirsen, made by Sarepta Therapeutics and marketed as Exondys 51, was approved in the United States this month after a long internal battle between FDA scientists who doubted its efficacy and the director of the FDA’s Center for Drug Evaluation and Research, Janet Woodcock. The details of this dispute, which led to formal complaints against Woodcock from several top agency scientists, are laid out in an unusually frank report by the FDA’s commissioner, Robert Califf.1

The drug’s approval has been widely interpreted as a victory for Woodcock’s vision of an FDA that is more responsive to patients’ demands, especially those with …

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