39. Vyondys 53

FDA Building 2
(FDA)

Active ingredient: golodirsen
Disease: Duchenne muscular dystrophy
Peak sales estimate: $220 million
Approved: Dec. 12, 2019
Company: Sarepta Therapeutics

The scoop: Like its sister Duchenne drug Exondys 51, Sarepta Therapeutics’ Vyondys 53 had a roller-coaster ride toward final FDA approval. The agency first surprised Sarepta and industry watchers by rejecting the new drug application for Vyondys 53 in August, citing safety concerns. But analysts suspected it was a political move driven by controversy around Exondys 51’s approval based on dystrophin as a surrogate marker. Yet, after Sarepta filed a dispute, the agency quickly overturned its own decision in a go-ahead just four months after the complete response letter. In a small clinical trial, patients on Vyondys 53 saw their dystrophin production rise from 0.1% to 1.02% after 48 weeks of treatment, an improvement the FDA labeled as “reasonably likely to predict clinical benefit.” RBC Capital Markets analysts viewed the decision as confirmation for “FDA’s continuing flexibility in areas of high unmet need like DMD.” They predicted that the drug, approved for the roughly 8% of DMD patients who bear a genetic mutation amenable to exon 53 skipping, could reach $220 million in peak global sales. — Angus Liu

39. Vyondys 53

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