What was Sarepta's regulatory track record before Elevidys?
Exondys 51 in 2016 set the template: Janet Woodcock overruled both her review team and a 7-6 advisory vote, two reviewers resigned, and the confirmatory trial deadline was missed — a pattern critics say Elevidys repeated on a larger scale.
Elevidys followed a contested precedent. Sarepta's first DMD drug, Exondys 51, was approved in 2016 on data from 12 boys after the FDA's drug centre director, Janet Woodcock, overruled her review team and an advisory committee that had voted 7-6 against. The FDA's acting chief scientist said this might be the first time a centre director had overruled both a review team and advisory committee on effectiveness, and two review team members resigned. Sarepta then missed the original November 2020 deadline for Exondys 51's confirmatory trial. Elevidys itself faced a long road, including clinical holds in 2018 and 2021, before its 2023 accelerated approval. Critics see a pattern of senior officials giving Sarepta latitude that reviewers opposed; supporters see regulators appropriately responding to an urgent unmet need.