The Eteplirsen Approval: Former FDA Officials Weigh In On The Science

A former CDER director, office director, division director, and supervisory reviewer agreed to comment for the record on the merits of the science that formed the basis of FDA’s accelerated approval of the Duchenne Muscular Dystrophy therapy Exondys 51.

FDA’s decision to grant accelerated approval to Sarepta’s DMD drug Exondys 51 (eteplirsen) has generated divergent opinions on what the approval means to the agency standards for approval and to the relationships between the different review levels within the Center for Drug Evaluation and Research.

Further coverage of those issues can be found in the sidebars, but behind them all is the important fundamental assessment...

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