|Sarepta CEO Ed Kaye|
The FDA has rescheduled its long-awaited advisory committee review of Sarepta's eteplirsen ($SRPT) for April 25, just 10 days after the third anniversary for the company's claim that the agency was willing to consider an accelerated review of the Duchenne muscular dystrophy drug.
Initially slated for earlier in the year, the agency's timetable was upset by a winter blizzard that locked down Washington, DC. Now regulators say they will hold the daylong meeting, which will draw a large crowd of patient advocates, at College Park Marriott Hotel and Conference Center in Hyattsville, MD.
Sarepta is now at a new low point after going through a years-long roller coaster ride with its drug.
Three years ago, then-CEO Chris Garabedian whipped up investors with a release claiming that the biotech was making real progress in getting the FDA to accept dystrophin production as a surrogate marker that could be used for an early OK. But the data that it was tracking on walking distance among the boys taking the drug were from a tiny study. And an internal review of the drug from the FDA simply dismissed the company's case on efficacy.
Earlier this week the biotech reported that it is laying off 30 staffers and consolidating its operations as it awaits the FDA's decision.
As of now, the drug has been given little chance of an approval when it comes up for a formal decision. Rival drugs from BioMarin ($BMRN) and PTC Therapeutics ($PTCT) have been shown the door without ceremony, and the harsh agency report on eteplirsen made it clear that the FDA had little use for the data offered by Sarepta.
The weather delay, though, did give the agency a chance to push back the PDUFA date, allowing extra time to consider additional data. Patient advocates will come out strong for an approval, regardless of what Sarepta can or cannot prove. And that may be the best card that Sarepta can play right now.
There is no treatment option for DMD in the U.S.--PTC's ataluren remains on the market in Europe, despite two failed studies--making this a particularly controversial decision.
The PDUFA date is May 26.
- here's the notice