Sarepta Scores Another Remarkable Approval With Vyondys 53 For Duchenne
Executive Summary
Formal dispute resolution was key element in four-month turnaround from rejection by US FDA to approval for golodirsen, the second Sarepta DMD drug to clear the agency after appearing to have minimal prospects.
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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.