Sarepta Scores Another Remarkable Approval With Vyondys 53 For Duchenne
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.
You may also be interested in...
Keeping Track: Viltepso Earns Priority Review Voucher; Manufacturing Trips Fennec; Eagle Drops Ryanodex Exertional Heat Stroke Bid
The latest drug development news and highlights from the Pink Sheet’s US FDA Performance Tracker.
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.
Despite obvious similarities in the contentious reviews of Sarepta’s Vyondys 53 and its predecessor Exondys 51, drug developers should focus on the differences between the two reviews.