(Bloomberg) – Biogen Inc. Alzheimer’s Treatment Trial by Biogen Inc. There was no backing from an advisory board of the US Food and Drug Administration on Friday, placing the drug at a crossroads when the agency considered approval.
Outside experts voted 8-1, with 2 undecided, that data from a single clinical trial with positive results was not enough to show that Biogen’s drug worked. They also voted 10 to 0, with an undecided 1, that the positive study should not be considered the primary evidence that the drug works based on conflicting evidence from another trial.
The votes contradict a report prepared by FDA critics prior to the meeting advocating the effectiveness of the drug, called aducanumab, despite disagreements within the agency.
The test they were asked to evaluate is not over yet, and although the data looks positive, they cannot know for sure.
Joel Perlmutter, a board member and professor of neurology at the University of Washington School of Medicine in St. Petersburg “There is a huge risk of approving something that doesn̵7;t work,” says Louis, Missouri.
The approval of Biogen therapy will be a pivotal milestone in the long-standing battle against Alzheimer’s, a dementia that affects an estimated 5.8 million Americans. The Alzheimer Association said in a report last year, there are other drugs that temporarily improve some symptoms, but none of them slow or stop the lethal destruction of cells. cerebral nerves.
Aducanumab has also become a particularly important drug for Biogen, which has seen its market value fluctuate significantly over the past two years as they paused their research on the drug, beginning. re-study and then submit it to regulators for approval earlier this year.
Biogen shares were halted during a regular Friday trading day. On Wednesday, shares rose 44 percent following positive reviews from FDA staff, though some of those gains were returned Thursday, when Biogen fell 7.5 percent.
Biogen’s representative did not immediately respond to a request for comment.
The recommendations of the advisory committee are not binding. The FDA often calls on expert councils, including researchers, healthcare professionals, and patient representatives, when reviewing whether to approve a new drug. The FDA appears to have positively reviewed the drug, Wall Street analysts said.
“This will be a test against the FDA about what happens when the FDA is at one extreme and the panel seems to be on the other, with science and evidence,” said Mizuho analyst Salim Syed. Without them is the core of the discussion. an email to Bloomberg News.
The road is complicated
Aducanumab targets the amyloid plaque that builds up in the brains of Alzheimer’s patients, although scientists don’t know what role it plays in the disease. Brain scans showed that the drug removed the plaque, but whether or not that would help is unclear.
FDA did not follow the typical process for advisory boards this time, starting with a joint report with Biogen on aducanumab clinical trial data prior to the meeting. Usually, the FDA posts its own report outlining the employee’s review of the data.
The agency declined to answer questions about why it incorporated the report and whether Biogen could review it before it is made public. It also gives panel members the option to vote undecided when often asked for a yes or no answer.
This therapy has taken a turbulent path for the FDA to consider. Biogen halted his study of the drug in March 2019 for signs of inactivity, then revived in October of that year after saying that reviewing the data in one of the two trials showed. it was successful.
Biogen, based in Cambridge, Massachusetts, presented data from two aducanumab trials at a conference in December. One trial showed that the drug could slow disease progression, while the trial The other has no effect. Researchers question the positive results because not all participants completed the trials.
Kyle Krudys, a clinical analyst at the FDA, told the advisers during a recorded presentation that the test results that did not show that the drug’s effects on patients would not reduce the cumulative test results. pole. “I concluded that the applicant provided substantial evidence of effectiveness to support approval,” Krudys said.
Meanwhile, Tristan Massie, a statistical reviewer at FDA, determined that a third clinical trial after completion was needed to determine if the aducanumab was working.
“Excluding data from a large trial for not enough good reason is unscientific, statistically inconsistent, and misleading,” Massie said in a presentation prepared for advisers.
‘Not worthy of attention’
Billy Dunn, Director of the Office of Neuroscience of the FDA’s New Drug Office, summed up the agency’s view on the drug in a positive way at the beginning of the meeting, without regard to concerns of Massie. He focused on ranking Alzheimer’s as the sixth leading cause of death in Americans, according to the Center for Disease Control and Prevention.
“We are very sensitive to the need for new drugs for Alzheimer’s,” Dunn said.
G. Caleb Alexander, a board member and epidemiologist at the Johns Hopkins Bloomberg School of Public Health, calls the FDA documents “notably inconsistent” and states the statistical review of Massie “well done”.
“It seems the TV audio and video are out of sync,” Alexander said.
If the aducanumab is deleted, sales are expected to total around $ 4.7 billion by 2025, according to Bloomberg data. Biogen developed therapy with Japanese drugmaker Eisai Co.
Eli Lilly & Co. The Indianapolis-based development had some experimental Alzheimer’s drugs, and the company’s stock fell 4.4 percent following the panel’s vote. Like Biogen’s medicine, Lilly’s donanemab aims to clear the amyloid plaque from the brain.
(Updated with additional voting at advisory meeting in second paragraph)
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