Sarepta's Eteplirsen Approved After Contentious Internal FDA Debate

Sep 20 2016

• By Sue Sutter

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CDER Director Woodcock grants accelerated approval after FDA Commissioner Califf refuses to overturn her finding that the drug's effect on dystrophin is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy.

FDA Commissioner Robert Califf deferred to Center for Drug Evaluation and Research Director Janet Woodcock on the accelerated approval of Sarepta Therapeutics Inc.'s Exondys 51 (eteplirsen) even though her favorable view of the Duchenne muscular dystrophy drug was opposed by the clinical review team, other senior personnel within the drugs center and the agency's acting chief scientist.

The agency granted accelerated approval Sept. 19 of eteplirsen, an antisense oligonucleotide, for treatment of Duchenne muscular dystrophy (DMD) in...

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