FDA’s Unger On Exondys Approval As Precedent: 'We Hope Not'
The review manager most opposed to the eteplirsen approval offered his views of the process that led to the final decision and the 'circus' atmosphere of the advisory committee.
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Rejection of Sarepta’s golodirsen has become latest flashpoint in debate over release of complete response letters. In this case, though, FDA’s reasoning seems fairly obvious, based on the prior eteplirsen review.
Patient advocates asked what they could do after US FDA’s complete response letter for the proposed Duchenne drug, but Sarepta's CEO said the company would get the product back on track to approval itself, a marked contrast to engaged role the community played the last time the firm ran into a roadblock at the agency.
US FDA allowing Sarepta to seek accelerated approval for follow-on DMD agent – following 'precedent' set by eteplirsen. Now the new challenge is to define exactly what that precedent is.