With Sarepta’s fate uncertain, BioMarin CEO Bienaimé weighs the future of Duchenne drug

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NEW YORK--Come Monday, Sarepta ($SRPT) will get a very clear idea of the odds it faces on finally achieving its years-long quest to gain an FDA approval of its Duchenne muscular dystrophy drug eteplirsen. And you can expect that BioMarin CEO Jean-Jacques Bienaimé will be paying close attention to all of the opinions voiced by the FDA’s outside experts on DMD as the agency panel meets to discuss the drug's fate.

Back in early January, Bienaimé was disappointed by the FDA’s decision to reject its rival drug, drisapersen, with regulators left unimpressed by the efficacy data that was submitted for an approval. In an interview with me Wednesday afternoon, the CEO made it clear that the company ($BMRN) may face some tough choices on their drug, which cost $680 million in cash. And if the cards don’t start turning in its favor, time may be running out on at least one of the three drugs that's been making an uphill run at a fast marketing OK.

“The European decision is the next step,” says Bienaimé. If they can keep drisapersen on track at the EMA, an opinion should arrive in two or three months. “If they reject it,” he noted, “we have to decide if we continue it.”

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Given the fact that the company is probably looking at mounting a late-stage study to get the drug back up in front of the U.S. regulators--though they’re still waiting on a final debrief with the FDA following the January rejection--BioMarin can decide if it should devote the resources necessary for that or move on to the early-stage programs the company has in place for DMD.

It’s not simply a question of resources, he adds, but the “practicality” of doing another study under the circumstances. 

Bienaimé was in Manhattan on Wednesday to give analysts an update on the company’s pipeline, including some encouraging extended data on its mid-stage drug vosorotide for dwarfism (achondroplasia), now moving to Phase III, as well as a promising early-stage gene therapy for hemophilia A (BMN 270). In his perspective, the company has a set of late-stage drugs--including the rare disease drug pegvaliase and a therapy for a form of Batten disease--and a sizable pipeline overall that will command a significant amount of R&D resources. 

If the regulatory hurdles at the agencies on both sides of the Atlantic are too steep on DMD, the company will have plenty of other work on its plate.

A rejection in Europe is by no means a sure thing just because the FDA rejected the drug. PTC Therapeutics ($PTCT) won a conditional OK in Europe two years ago for its DMD drug, even though it had failed a mid-stage study and was on its way to failing a late-stage trial. Those back-to-back failures prompted the FDA to refuse to even file the biotech’s application for review, satisfied that investigators never provided a convincing case on efficacy. But just days ago the U.K.’s drug watchdog NICE reversed itself and recommended the PTC drug, though European regulators say that they will revisit their decision at some point.

And so it goes. The FDA also provided a harsh review for eteplirsen a few months ago, though analysts are waiting now to see whether new documents out tomorrow could change in light of additional data. In the meantime, patients are rallying in the hundreds to mount a show of support in the looming panel review.

But Bienaimé says he doubts that kind of lobbying campaign will make much difference.

“I don’t think they’ll be swayed by that,” he noted with a shake of his head. “The FDA wants to see data.”

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