No More Sarepta-Like Development, FDA Officials Say
OND Director Jenkins says Sarepta's Exondys 51 shouldn't be a model, gets an earful from patient advocates.
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Refining A Controversial Endpoint: Sarepta Banking On Dystrophin Levels For Golodirsen Approval In DMD
While glodirsen did show higher levels of dystrophin production than eteplirsen did, increase is still unlikely to satisfy US FDA reviewers who disagreed with Janet Woodcock's decision to grant accelerated approval to eteplirsen.
At inconveniently timed NORD conference, the former CDER deputy center director finds himself unable to speak for either FDA or the Pharmaceutical Research and Manufacturers of America.
PTC Therapeutics argues FDA did not apply the same standard to Translarna in Duchenne muscular dystrophy as previous DMD products, but FDA says accelerated approval was not possible because of negative efficacy data.