With Sarepta’s Vyondys, US FDA Staff Saw Opportunity To Right Perceived Wrongs With Exondys
ODE I director Ellis Unger chafed at the notion of approving the Duchenne muscular dystrophy drug golodirsen on the same unvalidated surrogate marker, this time in the face of infection and renal toxicity concerns; OND’s Peter Stein granted Sarepta’s appeal while also calling attention to the company’s failure to timely conduct the eteplirsen confirmatory trial.
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The FDA and Sarepta have had a controversial history with the accelerated approvals of the firm’s earlier drugs for Duchenne muscular dystrophy – but the company’s investigational gene therapy, SRP-9001, now under a priority review, could be a genuine breakthrough.
Everyone knows that cancer drugs dominate use of the Accelerated Approval pathway by the US FDA. But it is remarkable just how rare a non-cancer AA is.
Sarepta has experience pursuing accelerated approvals in the US for Duchenne muscular dystrophy therapies, but with a costly gene therapy will payers require data from a controlled Phase III study?