- The FDA will not complete its review of Sarepta Therapeutics' drug for Duchenne muscular dystrophy (DMD) by the stated target date of May 26, the company said in a statement Wednesday.
- Sarepta said the FDA notified the company that it will be continuing its "review and internal discussions" over eteplirsen but will not reach a decision by Thursday. No new date was given.
- Shares of Sarepta spiked by as much as 25% in pre-market and early trading on hopes the delay might indicate a higher possibility of approval for eteplirsen. But an advisory panel last month voted against approval of the drug, criticizing the trial methodology and small size.
Hundreds of patient advocates and DMD patients had packed that advisory panel meeting and made an impassioned case for eteplirsen's approval. Many of public speeches testified to the benefit the drug seemingly produced in some of the boys who received the drug, although clinical data failed to impress the independent experts who advise the FDA.
In the end, the panel voted 7-3, with three abstentions, that eteplirsen did not demonstrate efficacy in treating DMD. In another vote, members of the committee decided the drug failed to produce sufficient amounts of dystrophin, a key protein for muscle function, to produce a clinical benefit.
While the FDA is not required to follow the advice of the advisory committee, it usually does so.
If the FDA rejects eteplirsen, it would be the third DMD drug to fail regulatory review this year following earlier rejections of BioMarin's drisapersen and PTC Therapeutics' ataluren.
Sarepta said the FDA indicated it would "strive to complete their work in as timely a manner as possible."
A rejection would be devastating for the many boys with Duchenne and could cripple Sarepta. As of March 31, Sarepta had roughly $130 million in cash and equivalents, plus short-term investments. The company posted a net loss of nearly $60 million in the first quarter and it does not generate any revenue.
DMD is a rare genetic disorder primarily affecting young boys which causes progressive muscle loss, and eventually leads to death. Sarepta estimates it affects approximately one in every 3,500 - 5,000 boys worldwide. Eterplirsen is targeted at a smaller subset of roughly 13% of the wider DMD patient population.