Patient advocates descend on FDA panel as Sarepta squares off against regulators

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Hundreds of Duchenne muscular dystrophy activists descended on an FDA panel review of Sarepta's eteplirsen Monday morning, looking to exert the maximum amount of pressure possible to spur an endorsement from the experts.

The initial presentation by the company today, including a lengthy defense by interim CEO Edward Kaye of the company’s data from a small study of 12 patients, underscored the deep-seated disconnect between drug investigators and regulators. Eteplirsen, Kaye says, is the first drug to spur a significant increase in dystrophin. Data from the rival drug drisapersen prove that the company’s use of data drawn from historical results is accurate, he added, and there was a clear and dramatic difference in loss of ambulation in the drug arm and the control group used.

The FDA’s review disagrees openly with all of these interpretations.

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Sarepta, though, has some major league political supporters. Just days ago a group of 24 U.S. Senators and members of the House signed letters supporting an accelerated approval. Former Republican presidential candidate Rick Santorum tweeted over the weekend that he plans to be there to offer his support. 

A rejection here would likely bar the door to any new drug approval for Duchenne’s for several years. BioMarin is now considering punting drisapersen after an FDA rejection--its longterm survival may rest on a European approval — while PTC Therapeutics may be forced to jerk its drug off the market after a Phase III failure and FDA refuse-to-file note. And that stark reality will heighten the drama today.

Friday afternoon, Sarepta’s ($SRPT) path to a formal marketing decision from the FDA on its Duchenne muscular dystrophy drug eteplirsen took yet another bizarre twist.

The FDA posted a set of discussion points and questions for Monday’s advisory panel review of the drug. It was immediately apparent to a varied group of analysts that the language reflected the agency’s harsh view of the sparse and questionable data used to ask for an accelerated approval based on the results of a trial that enrolled only 12 boys.

Question: “Has the Applicant provided substantial evidence from adequate and well controlled studies that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit?”

Based on the FDA’s stubborn opinion issued in two analyses, the agency would never call Sarepta’s study “adequate and well controlled.” And it clearly discounted any evidence of dystrophin production.

But asking the panel any question opens the possibility that the intense public pressure being applied on the FDA by patient advocates could influence the outside experts to vote for an early OK. And the biotech’s shares--battered yesterday by an internal FDA review that adopted a clearly negative tone--soared more than 35%.

“We view the fact that panel members will have to vote on Monday as an incremental positive for SRPT,” wrote RBC’s insightful Simos Simeonidis, “simply because of the understandable pressure committee members will be under, in the presence of the DMD community and families.” After all, if the FDA had intended a negative outlook Monday, why would they put the advisers on the spot?

But that didn’t prevent analysts from taking a skeptical view of Sarepta’s chances.

“Despite that view, when we read through the actual questions and the language of the accompanying discussion (see below), we see a document with the exact same tone and tenor as the January and April briefing documents,” Simeonidis observed. “The questions and the discussion ask the panelists to focus on the trial conduct, the evidence for dystrophin and clinical efficacy. And we expect the answer to all of these to be No.”

“Prepare for a wild ride,” noted Baird’s Brian Skorney. “After years of ambiguity surrounding this review, we head into Monday expecting nothing more than to be surprised.”

After the roller coaster ride that has taken investors and biotech observers though plenty of dramatic plot twists in recent years--including high-level exits, disputes over dealings with the FDA and more--Sarepta can still surprise when it's least expected.

- here are the questions