Outside advisers to the Food and Drug Administration (FDA) on Friday unanimously recommended the agency grant full approval to a new kind of Alzheimer’s drug that modestly slows cognitive decline in early-stage patients.
The panel voted 6-0 that Leqembi shows benefits in treating people with early-stage Alzheimer’s and mild cognitive impairment. The positive vote starts a ticking clock for the FDA to decide whether to follow the panel’s recommendation. The agency needs to make a decision by July 6, and usually follows the panel’s advice.
Teresa Buracchio, acting head of the FDA’s neuroscience division, said the small panel was due to members recusing themselves due to conflicts of interest.
The drug is not a cure and doesn’t stop people with Alzheimer’s from getting worse. But data from a late-stage trial of nearly 1,800 participants showed patients who took Leqembi experienced a 27 percent slower rate of cognitive decline compared to those on a placebo.
The drug was given accelerated approval in January.
Leqembi, manufactured by Eisai and Biogen, is a new type of treatment that targets sticky plaques of protein in the brain called amyloid, which disrupts communication between brain cells, eventually killing them.
The only other drug in the anti-amyloid class is Aduhelm, which was approved by the FDA over strong objections from its advisory panel over lack of evidence that the drug actually works. The approval eventually became the subject of a congressional investigation, which found the process was “rife with irregularities.”
Critics have said there was no real clinically meaningful impact of Leqembi, and the differences between people on the placebo and people taking the drug were minimal.
The drug also comes with serious safety concerns. During the study, 13 percent of patients who received Leqembi had brain swelling and 14 percent had brain bleeding.
“There are adverse effects,” said panel chair Robert Alexander, chief scientific officer at Banner Health’s Alzheimer’s Institute in Phoenix, Arizona. “But they’re monitorable and I think the benefit is clear.”
Supporters said the differences early in the disease can amount to several additional months, and any delay in the progression of the disease matters.
The FDA doesn’t take price into consideration, but the cost is looming, and the agency’s decision is crucial for Medicare coverage. Medicare will currently only cover Leqembi for patients enrolled in a clinical trial, and there are no clinical trials underway.
The twice-a-month infusion costs $26,500 a year, and the Centers for Medicare and Medicaid Services has said it won’t pay for any anti-amyloid drugs until they are fully approved. Even then, the agency will require patients and providers to participate in a registry to track real-world effectiveness.
Alzheimer’s advocates and congressional Democrats have slammed the registry requirement as an unnecessary restriction. Registries are sometimes used for medical devices, but rarely for drugs.
According to Sen. Bernie Sanders (I-Vt.), the price tag is a major problem regardless of Medicare coverage.
In a letter sent this week, Sanders said the price tag is “unconscionable” and urged the Department of Health and Human Services to use its authority to break Leqembi’s patent monopoly if Eisai, which developed the drug, and Biogen, which is manufacturing it, refuse to lower the price.