Accelerated Approval After Eteplirsen: A Lowered Bar Or A Unique Event?

What CDER Director Woodcock saw as a flexible and reasonable approach to approval of Sarepta's muscular dystrophy drug, ODE I Director Unger viewed as a threat to the substantial evidence standard.

FDAs internal disagreement over the adequacy of Sarepta Therapeutics Inc.'s efficacy evidence for Exondys 51 (eteplirsen) evolved into a debate over the intent, utility and future of the accelerated approval regulatory pathway.

Office of Drug Evaluation I Director Ellis Unger and others within the agency worried that granting eteplirsen accelerated approval for...

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