Sarepta Complained To Woodcock About Exondys 51 Review Early And Often
Documents also show that Sarepta officials begged CDER director for approval on multiple occasions prior to the agency's September 2016 decision.
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The EMBARK trial’s availability to more patients likely contributed to the much quicker enrollment than confirmatory trials for Exondys 51, Vyondys 53 and Amondys 45, which also saw enrollment targets and eligibility ages change over the years.
Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.
FDA has twice refused to file PTC Therapeutics' NDA for the Duchenne muscular dystrophy treatment because of 'clearly and convincingly negative' pivotal trials and questionable post hoc analyses; sponsor argues ataluren should be afforded the same consideration and flexibility given to Sarepta's DMD drug eteplirsen, which received a controversial accelerated approval.