Sarepta rockets up after FDA approves its controversial Duchenne drug
After years of fierce debate in a seesaw battle over the fate of Sarepta’s Duchenne muscular dystrophy drug eteplirsen, the FDA has granted the drug conditional approval in the US, making this the first drug OK’d to treat the lethal disease.
The approval comes with the threat of swift retribution, though, if investigators fail to follow through with positive data from a bigger study. The agency’s statement includes a requirement that Sarepta will now have to confirm that the drug actually works as designed, spurring production of dystrophin in a way that can improve motor function in the 13% of patients amenable to exon 51 skipping. And the agency says that it may withdraw its OK without that data.
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.