FDA officials: There was “no scientific basis” for Duchenne drug OK as Sarepta complained of “dire financial” condition
Two senior FDA officials mounted a vehement assault on Janet Woodcock’s decision to push through an approval of Sarepta’s Duchenne muscular dystrophy drug Exondys 51. New documents posted by the FDA, including a round of memos on the issue in September, warned FDA Commissioner Robert Califf that he was allowing an approval even though Woodcock had not considered all the analysis they had done to underscore the company’s weak case, adding that there was no scientific basis to conclude that the drug was reasonably likely to benefit patients.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.