A panel of expert advisers on Friday unanimously agreed that a late-stage trial of Eisai and Biogen's Alzheimer's drug Leqembi verified the benefit of the treatment for those at an early stage of the disease, clearing the way for traditional U.S. approval.
All six advisers on the panel voted in favor of Leqembi for treatment of the mind-wasting illness.
Regular approval by Food and Drug Administration is expected to expand Medicare payment for the treatment. It would also make Leqembi the first in its class of drugs that work by removing sticky amyloid plaques from the brain to achieve the regulatory milestone.
Leqembi won accelerated approval by the FDA in January based on that ability to remove amyloid from the brain.
"I believe the benefit versus risk are beneficial, acceptable and in line with this class of therapeutics, especially considering the burden of the disease and the progressive nature of the disease," said panel member Dr. Tanya Simuni, professor of neurology at Northwestern University Feinberg School of Medicine.
The accelerated approval restricted Medicare payment of the drug to those in a clinical trial, but no such trials are underway for Leqembi, resulting in negligible sales so far. Most U.S. Alzheimer's patients are Medicare-eligible.
FDA staff reviewers on Wednesday said Eisai's late-stage trial showing Leqembi slowed cognitive decline by 27% in early Alzheimer's patients appeared to confirm the drug's benefit and raised no new safety issues.
"Overall, it demonstrated clearly that this is an effective treatment in the population as it was defined," said Dr. Robert Alexander, committee chair and an Alzheimer's expert at the Banner Alzheimer's Institute. He added that he thought the study "clearly demonstrated a clinical benefit," calling the results "robust."
Wall Street analysts widely expect the FDA to grant traditional approval for the drug. Analysts have forecast Leqembi sales topping $1 billion in 2026 and reaching $5.7 billion by 2030.
The FDA, which usually follows the recommendations of its expert advisers, is expected to make its approval decision by July 6.
The federal agency that runs Medicare has said it will pay for the treatment if doctors participate in a health agency database, known as a registry, but has yet to release details of its plan.
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