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Illustration: Aïda Amer/Axios

The FDA will soon decide the fate of Biogen's experimental Alzheimer's drug. But there is one glaring issue — there is no conclusive evidence the drug effectively treats the crippling neurological disease.

Why it matters: This will be one of the FDA's most important decisions in years. The outcome will show whether the federal agency sides with the overwhelming scientific consensus that the drug isn't proven to work, or with an industry and a patient population desperate for anything to be approved.

The background: Two years ago, Biogen halted two clinical trials of its infused drug, aducanumab.

  • But the company looked again at the clinical trial data and argued one of the failed trials suggested some benefit for Alzheimer's patients on a higher dose of the drug. The other trial still failed.
  • The FDA generally wants "two adequate and well-controlled trials" for drug approval, although it does on rare occasions consider one well-controlled trial with "confirmatory evidence."
  • Biogen applied for FDA approval last summer.

Where things stand: Independent physicians, statisticians, drug researchers and others in the field have urged the FDA to reject the drug, warning the study supporting aducanumab is small, flawed and possibly a false positive. Side effects like brain swelling have also raised concerns.

  • The most notable dissent came during the FDA's advisory committee meeting last November, when none of the 11 members voted in favor of the drug's approval.
  • "What's the point of an advisory committee if you approve this? The FDA just doesn't consider your opinion at all," said Brian Skorney, a pharmaceutical stock analyst at Baird who added it would be a "huge misstep for the agency" to approve the drug.
  • The FDA's statistical reviewer of aducanumab said there was "no compelling substantial evidence of treatment effect or disease slowing and that another study is needed."
  • David Knopman, a Mayo Clinic neurologist who was a site investigator for the aducanumab trials, previously said the drug's evidence was "terribly weak." He told Axios he believes Biogen needs to run another clinical trial, and the company could have been close to finishing if it had initiated a trial two years ago.

The other side: Biogen did not make executives available for an interview and would not answer questions on the record. The company's market value will plummet if aducanumab doesn't get approval.

  • Many physicians and scientists who support aducanumab's approval have financial ties to Biogen and Eisai, which also developed the drug.
  • Alzheimer's patients who tried the drug, and their families, are looking for any kind of treatment that stops the disease's progression and broadly want the FDA to approve the drug despite the dearth of evidence.
  • "There's a dire and drastic need to offer relief," said Joanne Pike, a top executive at the Alzheimer's Association. She said the organization "respectfully disagrees" with the FDA's expert committee. When asked if Biogen should have started another trial two years ago or if Biogen should start another trial if the drug isn't approved, she said, "I'm not going to comment on that."

The bottom line: "The FDA's credibility would take an enormous hit if this product is approved," Caleb Alexander, a physician and drug researcher at Johns Hopkins who was on the FDA's advisory committee for this drug, told Axios. "It's important they get it right. There's no reason they should follow the science any less here."

Go deeper

FDA needs more time on Juul e-cigarette ban decision

The Food and Drug Administration said Thursday it will delay its decision on top-seller Juul, but thousands of other electronic cigarettes will be ordered off the U.S. market.

Why it matters: The stalled decision prolongs the agency's determination on whether there is enough data to show if adult cigarette smokers switching to a less harmful option outweighs the detrimental costs of young people getting hooked on nicotine by vaping.

Sep 10, 2021 - Health

FDA "will follow the science" to approve COVID vaccine for kids under 12

A nurse gives a boy a dose of the Pfizer vaccine at a clinic. Photo: Paul Hennessy/SOPA Images/LightRocket via Getty Images

The FDA said on Friday that it is "working around the clock" to support the process of making the coronavirus vaccine available for children under the age of 12.

Why it matters: The number of children getting hospitalized due to the virus is increasing, with some infectious disease doctors worrying that the Delta variant could be causing more serious illness in kids.

Updated Sep 10, 2021 - Axios Events

Watch: A conversation on what's working to get Americans vaccinated

On Friday, September 10, Axios health care reporter Caitlin Owens and health care editor Tina Reed hosted a virtual conversation on what strategies are working to get Americans vaccinated, featuring former FDA Commissioner Dr. Scott Gottlieb and Walgreens Chief Medical Officer Dr. Kevin Ban.

Dr. Scott Gottlieb spoke about reducing vaccine hesitancy, an increase of COVID-19 cases in conjunction with the return to school, and how to ensure the effective distribution of booster shots when the time comes.

  • On why reducing hesitancy is more difficult in the later stages of vaccine rollout: “We’re at that point right now where every vaccine is harder to administer, because you’re probably trying to entice a consumer who is more reluctant to take the vaccine or finds it less accessible for a variety of reasons.”
  • On the recent rise in cases in younger populations returning to school: “Studies have looked at what are the most effective interventions—keeping kids in defined social pods within the school so you’re not having the entire grade commingling...and also implementing routine testing of asymptomatic children, ideally twice a week.”

Dr. Kevin Ban explained Walgreens’ decision to mandate vaccination for some employees, what has been working to increase vaccination, and the potential hurdles of administering booster shots.

  • On Walgreens’ thought process behind implementing an employee vaccine mandate: “Collectively as a group, we have followed the science since the beginning of this epidemic. And along with the effectiveness of these vaccines, we’ve noted the safety. And so collectively, as a leadership team, we thought that the right thing to do here was to, in fact, mandate vaccines.”
  • On the effectiveness of local outreach in vaccination efforts: “Meeting people where they are and listening to them and trying to understand what is it that’s preventing you, and then addressing that, is powerful. It’s hard to do across the country, but you can do it locally.”

In a final Why It Matters segment, Axios' Caitlin Owens and Tina Reed echoed similar curiosities about the impending rollout of booster shots, and raised the question of how the process may mirror or differ from that of the initial vaccine distribution.

Tune in next month for October's Vitals Check-Up.