A few days ago, shares of Sarepta plunged over pure speculation that new FDA rules governing expanded access to experimental drugs were a prelude to a formal rejection of its troubled Duchenne drug application. On Monday evening, the stock bounced right back after the biotech ($SRPT) announced that regulators have come looking for fresh data on the key biomarker for efficacy, raising renewed hope among its many advocates that an approval may yet occur.
According to Sarepta’s announcement, the FDA wants its researchers to “provide dystrophin data, as measured by western blot, from biopsies already obtained from the ongoing confirmatory study of eteplirsen (PROMOVI), as part of its ongoing evaluation of the eteplirsen New Drug Application (NDA). The Company plans to submit data from thirteen patient biopsy samples, at baseline and Week 48, to the FDA over the coming weeks to facilitate a prompt decision on the NDA by the Agency.”
Eteplirsen is designed to spur the production of dystrophin, mending at least partly the terrible damage done by DMD. If Sarepta can provide some proof that the drug does that--something that regulators have insisted is completely lacking in the NDA--then the agency will have a reason to provide an accelerated approval even in the absence of any evidence that the drug has made a difference in improving patients' ability to function.
That’s the theory.
The reality so far, though, is that the biotech has pinned its hopes up to now on a study involving only 12 patients, a thin dataset that would rarely even be considered for an approval and nothing close to the trial that regulators have repeatedly insisted on in one-on-one conversations with company officials.
The news today comes after back-to-back agency reviews that can only be viewed as a harsh rejection of the company’s upbeat assessment of the data, as well as a negative expert panel vote that recommended against an OK at this point.
In a normal world, that would have been it for Sarepta’s near-term hopes. But there’s nothing normal about the company or the drug.
Hundreds of patient advocates, patients and families turned out to give their raucous support for an approval during the panel review and vote. And backers have continued to protest--with widespread support from prominent elected officials and newspapers--that this is their last and only near-term hope for a drug that they insist has made a dramatic difference for slowly dying boys.
That passionate lobbying effort has caused the FDA to veer from its usual course, with two earlier delays that signaled the agency's inability to reach a decision. And while there’s no guarantee that the FDA will provide an accelerated approval, it clearly won’t trigger a rejection until it can prove that it has exhausted all efforts to find a reason for an accelerated OK.
“We view this decision as speaking to the immense pressure the agency is under,” noted RBC’s Simos Simeonidis, who has consistently found little reason to believe an approval could be forthcoming this year. “FDA appears to leave no stone unturned in its attempt to explore every possible pathway and scenario under which it could possibly approve a drug that under almost every other circumstance we can think of would have been rejected given the severe weaknesses and deficiencies in its dataset.”
In the meantime, there's every reason to believe that the roller coaster ride that Sarepta shareholders have been on for years now is not close to the end.
- here's the release
Debate over Duchenne drug hits fever pitch as D-day approaches
In setback, a majority of FDA experts reject Sarepta’s emotional campaign to gain Duchenne drug OK
Duchenne docs--aka Sarepta investigators, advisers and advocates--back controversial case for eteplirsen