Shares of Sarepta shot up, again, on Monday as investors responded warmly to Oppenheimer’s bullish views on a possible accelerated approval for its Duchenne muscular dystrophy drug eteplirsen. The stock closed up 26% as Oppenheimer analysts weighed the risks and huge upside associated with a fast approval for one of the most controversial drugs that has ever been pitched to regulators.
To be sure, there’s also been plenty of skepticism about Sarepta’s ($SRPT) chances with eteplirsen, which was fueled by the recent advisory committee vote against an accelerated approval, as outside experts sided with agency insiders who slammed Sarepta for ignoring its advice on pushing ahead quickly with a large, controlled study that could determine whether or not the drug had a positive effect on boys afflicted by the lethal disease.
Leerink’s Joseph Schwartz has lowered the chances of success for gaining an accelerated approval now to 15%, well down from a once optimistic 40%.
“Whereas the FDA had proposed a (placebo)-controlled study six times to SRPT, the company failed to fulfill the Agency's demand; the ongoing Ph. III PROMOVI exemplifies this,” noted Schwartz.
Jefferies as well as RBC’s Simos Simeonidis have adopted a skeptical tone as well.
“In one of the most emotional and highly charged Advisory Committee meetings in memory, with panelists speaking directly to the patient community at times (once in tears), the panel voted 3-7 against eteplirsen's evidence of efficacy (with 3 abstaining),” noted Simeonidis. “Even though the final decision rests with FDA, and despite assurances that no decision has been made, we continue to see slim chances for approval.”
Leading the way on the most optimistic interpretation of senior FDA official Janet Woodcock’s involvement in last week’s review, TheStreet’s Adam Feuerstein sparked the first big post-AdCom rally for Sarepta several days ago, laying out a case that despite the fact that a majority of the expert panel voted against an approval, Woodcock’s remarks indicated that the agency could be responsive to the intense viral public campaign to approve the drug now. And the bull case has been supported by editorials from mainstream pubs like the Wall Street Journal and the Washington Post demanding that the FDA make the drug available to all patients who could benefit from the exon 51-skipping therapeutic.
Patients and families have left no doubt that they believe in this drug and the slim data set that was available from a tiny 12-patient study that’s been the basis of Sarepta’s quest for an accelerated approval. And with the only other experimental drugs for this condition--from BioMarin ($BMRN) and PTC Therapeutics ($PTCT)--already barred for now by the FDA due to the poor data they gathered, this is the only chance these patients will have access to a drug anytime in the next few years.
Dying children and their parents make for a powerful group of advocates, as Chimerix ($CMRX) learned firsthand when it initially denied a compassionate use request for its antiviral from the parents of a young boy. And even if there is no solid data to provide proof that the drug helps patients, as the FDA has repeatedly insisted, the question now is whether the FDA bows to the public campaign and gives a green light on a case that would be instantly rejected for virtually any other drug.
That's a realistic possibility, and fortunes will be won and lost based on who's right. The PDUFA deadline is May 26.