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Comment & Response
April 11, 2017

FDA Approval of Eteplirsen for Muscular Dystrophy

Author Affiliations
  • 1Center for Duchenne Muscular Dystrophy, David Geffen School of Medicine at University of California, Los Angeles
JAMA. 2017;317(14):1480. doi:10.1001/jama.2017.2601

To the Editor Drs Kesselheim and Avorn raised concerns about the approval by the US Food and Drug Administration (FDA) of eteplirsen to treat Duchenne muscular dystrophy (DMD), such as reliance of the pivotal study on a surrogate measure.1 However, the 2012 Food and Drug Administration Safety and Innovation Act allows accelerated approval of drugs to treat rare and serious diseases based on surrogate end points that are “reasonably likely to predict clinical benefit.”2 Thirteen DMD experts spoke in support of accelerated approval for eteplirsen at the Peripheral and Central Nervous System Drugs Advisory Committee meeting.3 In addition, a letter to the FDA from 36 experts concluded that the 12 treated boys in the pivotal study are “clearly performing better than our collective clinical experience and the published literature would predict”4 and “the findings of this trial are sufficiently robust to support the proposed mechanism of action of eteplirsen, to provide a plausible explanation for the relative gain in function observed within the treatment group, and serve to bolster confidence that there is a positive treatment effect.”4