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The Readout Damian Garde

The panel of experts between Biogen and billions

Biogen’s decision to seek approval for a once-terminated treatment for Alzheimer’s disease is going to put a lot of pressure on the FDA. But before the agency gets to make its final decision, Biogen will have to win over a roundtable of experts in the Maryland suburbs.

That roundtable is called an advisory committee, and it’s tasked with voting on whether the FDA should approve a given product. The votes are non-binding, but when there’s controversy involved — and there’s plenty when it comes to Biogen’s drug — those panel decisions can be key indicators of where the FDA is headed.

STAT talked to experts with experience at the FDA’s neurology panel, the one that governs Alzheimer’s drugs, and they had some ominous warnings for Biogen. The company’s data merits a rejection on its face, according to one neurologist. And the very fact the FDA is convening a panel suggests the agency is looking for a reason to say no, according to another.

Read more.

Amarin drama, ahead of the drama

In just nine short days, an FDA advisory committee will meet to assess the clinical evidence in support of Vascepa, the closely watched fish-oil-derived heart drug from Amarin. But the pre-show drama is underway.

As STAT's Matthew Herper notes, a brief suggestion by Jefferies analyst Michael Yee — offered on a podcast — that Cleveland Clinic cardiologist Steven Nissen was in "correspondence with the FDA" over Vascepa sent certain members of bio-Twitter into a tailspin. Nissen, of course, is famous for having been outspoken about the cardiovascular risks of Merck’s Vioxx — and he's previously made no secret of his skepticism on Vascepa.

But Nissen told STAT said there's no there there. "There’s been no conversation that anybody could ever interpret under any circumstances as representing a discussion about Vascepa, end of story, period,” he said.

Read more.

About that lack of Alzheimer’s drugs

Just as the cupboard of experimental therapies for Alzheimer’s from major drug companies looks pretty bare (notwithstanding Biogen’s aforementioned and controversial revival), there was a surprise over the weekend. Chinese regulators conditionally approved Oligomannate, a drug derived from seaweed and made by Shanghai Green Valley Pharmaceuticals.

The company said a Phase 3 trial demonstrated the drug slowed cognitive decline compared to placebo, and that it has plans to run another, global Phase 3 trial so it can file for approval in other countries. But the company hasn’t published the full data yet, and some outside experts expressed some caution. One key question: Just how much of an effect did the drug have on cognitive function?

In addition to being the first Alzheimer’s drug approved since 2003, Oligomannate stands out because it was designed to affect the neuroinflammation that some scientists think contributes to Alzheimer’s through modulating the gut microbiome. An amyloid clearing drug this is not.

Read more.

How’s business at Sarepta?

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The past few months of conversation around Sarepta Therapeutics have, understandably, focused on its surprising FDA rejection and the question of whether its gene therapy will ever become a product. It’s easy to forget that Sarepta already has an approved drug, and that drug might be reaching its revenue peak.

On Thursday, we’ll find out just how well Exondys 51, approved to treat Duchenne muscular dystrophy, is doing on the market. The treatment got off to an impressive launch but has since approached what looks like a commercial zenith.

That shouldn’t be surprising: DMD affects about one in 5,000 boys, and Sarepta’s drug is approved only for the roughly 13% of them with a certain genetic mutation. Its revenue potential was always limited.

But the bull case for Sarepta was that the company would use the short-term Exondys 51 windfall to build a pipeline of therapies that would make for a bright future. Now that the picture has grown cloudy, analysts might have a different take on Sarepta’s revenue numbers.

More reads

  • Aveo's cancer drug runs into a skeptical FDA. (BioPharma Dive)
  • After an unprecedented death, FDA offers few hints for the future of its strategy on fecal transplants. (STAT Plus)
  • Obscure model puts a price on good health — and drives down drug costs. (Wall Street Journal)
  • She was destined to get early Alzheimer’s, but didn’t. Did a rare mutation protect her? (STAT)

Thanks for reading! Until tomorrow,

Tuesday, November 5, 2019

STAT

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