FDA panel weighs US approval for Alzheimer's drug from Eisai and Biogen

The agency is expected to make its decision on traditional approval by July 6. 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.

A panel of expert advisers are discussing whether to recommend traditional U.S. regulatory approval for Eisai and Biogen's new Alzheimer's drug Leqembi later on Friday, a move expected to expand Medicare payment for the treatment. Leqembi won accelerated approval by the Food and Drug Administration in January based on its ability to remove toxic amyloid plaques from the brain. The agency is expected to make its decision on traditional approval by July 6.

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. Dr. Teresa Buracchio, acting director of the FDA's office of neuroscience, said the agency is aware of the public debate about whether the study results were clinically meaningful.

She told the panel the agency believes the late-stage trial data makes "a compelling case" that the drug provides a clinically meaningful benefit, which the FDA defines as improvement in how a patient feels, functions or survives. However, she stressed that the agency has not yet made up its mind about whether to approve the treatment and its view could change based on the panel's discussions.

Jefferies analyst Michael Yee in a note said the advisory meeting "is progressing smoothly and remains on track for a likely positive vote." Several Wall Street analysts read the commentary in the FDA briefing documents as a sign that traditional approval was likely. UBS analyst Colin Bristow had said he expects Friday's panel meeting "to essentially be a formality" that will lead to approval and broaden access to the 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. Regular approval would make Leqembi the first in its class of drugs that work by removing sticky amyloid plaques from the brain to achieve the regulatory milestone.

Analysts have forecast Leqembi sales topping $1 billion in 2026 and reaching $5.7 billion by 2030. 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.

Patient advocacy groups expressed opposition to that plan, saying making Medicare coverage conditional to participation in a registry may reduce access to the drug. The expert panel has been asked to vote on whether the late-stage clinical trial of Leqembi verifies its benefit as a treatment for Alzheimer's, a fatal, mind-robbing disease that affects 6 million Americans, according to the Alzheimer's Association.

The agency has also asked the experts to discuss the risks and benefits of the treatment, with a focus on people who carry two copies of Alzheimer's risk gene called APOE4, which can increase side effects such as brain swelling. FDA also wants input on the drug's use in patients taking blood thinners, and in those with a condition called cerebral amyloid angiopathy, in which the protein amyloid - which Leqembi targets - builds up in artery walls and can cause bleeding in the brain.

Analysts, however, do not expect the issue to prevent approval.