As Alzheimer's disease partners Eisai and Biogen lay the groundwork for a wider launch of Leqembi, their efforts center on winning a full FDA approval. Wednesday, that vision came into better focus as the FDA released a document showing how the agency views the drug.In advance of a highly anticipated FDA advisory committee set for Friday, the agency released a briefing document that appears to support a full FDA approval for Leqembi.
On efficacy, the agency said the drug's treatment effect in its pivotal trial, called Study 301, is "supported by the consistently favorable results for the primary and secondary endpoints across the prespecified subgroups of interest."
In the study, investigators tested Leqembi against placebo in patients with mild cognitive impairment due to Alzheimer's or mild Alzheimer's dementia. The study measured the drug's efficacy on an endpoint called the Clinical Dementia Rating-Sum of Boxes at 18 months of treatment.
On that primary endpoint and three secondary clinical endpoints, Leqembi met the mark in the trial, the FDA said.
As for safety, the agency flagged risks of amyloid-related imaging abnormalities, cerebral hemorrhages and infusion-related reactions. But the reviewers concluded that the "risks can be described in the prescribing information and do not appear to preclude traditional approval of lecanemab."
Summarizing the FDA document, Mizuho analyst Salim Syed wrote to clients that the FDA review is "super clean" and that it tells a "simpler story" than the review of Eisai and Biogen's first Alzheimer's drug, Aduhelm.
The Leqembi document is 35 pages versus the 130-page document for Aduhelm, and the FDA is only asking its external experts to vote on one question this time around, compared to four for Aduhelm, Syed pointed out.
Specifically, the FDA wants its committee experts to vote on whether the results from Study 301 "verify the clinical benefit of lecanemab for the treatment" of Alzheimer's disease.
That question will be answered by the end of the day Friday, when the Peripheral and Central Nervous System Drugs Advisory Committee Meeting will meet to review the data on Leqembi.
Ahead of the meeting, one member of the committee, David Weisman, M.D., dropped out of the discussion, apparently because of his financial ties to the companies.
Leqembi won an accelerated FDA approval early this year and is due for a decision on a full approval by July 6. A full approval is essential for a wider rollout of the drug, because the Centers for Medicare & Medicaid Services (CMS) has placed restrictions on its use under the FDA's accelerated pathway. Right now, the drug can only be given in a clinical trial setting to patients on Medicare.
Last week, CMS proposed widening access to the new class of Alzheimer's drugs with full approvals under a registry approach to collect real-world data. But quickly afterward, trade group PhRMA voiced concerns that the arrangement still raises barriers to access.

Reference: https://investors.biogen.com/news-releases/news-release-details/fda-accepts-eisais-filing-supplemental-biologics-license