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Exondys Approval: FDA Commissioner's Draft Decision Drew Internal Rebuke

 
• By Sue Sutter

Even at the final step, FDA’s review of Sarepta's Duchenne muscular dystrophy drug remained collaborative and contentious, as Commissioner Califf's view that accelerated approval would not set a new precedent drew pushback from ODE I Director Unger, while Acting Chief Scientist Borio said the draft decision downplayed the 'miniscule' amount of dystrophin with eteplirsen.

FDA entrance sign 2016

FDA officials involved in the dispute over whether to approve Sarepta Therapeutics Inc.'s Exondys 51 (eteplirsen) did not hold back their criticisms when FDA Commissioner Robert Califf circulated his draft decision supporting accelerated approval for the Duchenne muscular dystrophy drug.

Office of Drug Evaluation I Director Ellis Unger questioned Califf's conclusion that Center for Drug Evaluation and Research Director Janet Woodcock had considered all relevant scientific evidence in deciding to grant accelerated approval

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