FDA on Friday issued accelerated approval to lecanemab, an experimental Alzheimer's drug developed by Eisai and Biogen, for certain Alzheimer's patients. But many experts say that until the drug is covered by Medicare, its high price tag will prevent many patients from accessing it.

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Background

Similar to Aduhelm, lecanemab reduces abnormal clumps of beta amyloid in patients' brains, proteins that are typically a sign of Alzheimer's disease.

In a study published in the New England Journal of Medicine last year, patients who received lecanemab saw 27% slower cognitive decline, which translated to a 0.45 score improvement on the Clinical Dementia Rating sum of boxes, compared to those who received a placebo.

However, the drug was also associated with "serious adverse events," which occurred in 14% of lecanemab patients compared to 11% of placebo patients. Nearly 7% of patients taking lecanemab dropped out of the trial because of the drug's negative side effects, nearly twice the number of patients who dropped out in the placebo group.

More than a quarter of patients taking lecanemab experienced infusion-related reactions, including fever and flu-like symptoms, typically after the first dose of the drug. A small percentage of those in the placebo group experienced the same.

FDA approves lecanemab

On Friday, FDA issued accelerated approval to lecanemab, which will be sold under the brand name Leqembi. However, the drug label will include narrower and more cautionary language than the label given to Aduhelm.

Leqembi's label says the drug is only for use in patients with early and mild Alzheimer's disease and instructs doctors not to treat patients without doing tests to confirm they have a buildup of amyloid proteins.

This follows a congressional report issued last week that found FDA's approval of Aduhelm was "rife with irregularities" that overlooked internal concerns about the drug's safety and efficacy.

An FDA official involved in lecanemab's review said "there were many important lessons learned" since the agency approved Aduhelm.

"In the case of Leqembi, more detail and clarity on the most appropriate patient population for use of the drug, and greater explanation around safety" for brain swelling, brain bleeding, and the use of blood thinners with the drug are "now included in the label," the official said.

Reaction

Some experts praised FDA's approval, saying that any progress in fighting Alzheimer's disease is good news.

Sharon Cohen, a neurologist and director of the Toronto Memory Program, said patients taking lecanemab may be able to recognize loved ones, stay active, or perform everyday tasks longer.

"We need to keep in mind that we are dealing with a fatal disease," she said. "It's not fatal instantaneously … but slowly, millions of neurons are dying."

Joy Snider, a professor of neurology at Washington University School of Medicine in St. Louis, said lecanemab is "not a cure. It doesn't stop the disease completely. It doesn't make people get better." However, the drug "does slow down progression in very mild disease," she said.

That could be meaningful to patients and their families, Snider said. "Maybe you could keep driving for an extra six months or a year. Maybe you could keep doing your checkbook for an extra six months to a year."

However, many experts also expressed concern over the drug's high price tag, which Eisai officials on Friday said will be $26,500 per year for biweekly dosing.

Matthew Schrag, a neurology researcher at Vanderbilt University, questioned whether the drug's benefits were worth its price and potential side effects.

"Most patients won't notice the difference," he said. "This is really quite a small effect and probably below the threshold of what we'd call clinically significant. … Is this slight, measurable benefit worth the hefty price tag and the side effects patients may experience? I have pretty serious doubts."

As it stands, Medicare will not cover lecanemab unless CMS determines the drug has clearer evidence of helping patients. Eisai has said it intends to establish a patient assistance program to provide lecanemab at no cost to "eligible uninsured and underinsured patients, including Medicare beneficiaries, who meet financial need and other program criteria."

The Alzheimer's Association has "filed a formal request asking CMS to provide full and unrestricted coverage for FDA-approved Alzheimer's treatments," according to Maria Carrillo, the organization's chief science officer.

"It's clear there won't be coverage on day one and that's never been true of any FDA approved drug before," said Robert Egge, chief public policy officer of the Alzheimer's Association. "It's going to be very difficult if not impossible for Medicare beneficiaries to get coverage."

In order for patients to qualify for lecanemab, they have to undergo tests showing they're in the early stages of dementia and their brains contain amyloid protein deposits, which will require multiple visits to specialists and will ultimately result in a very long queue, according to Jakub Hlávka, a health policy expert at the University of Southern California.

"Roughly speaking we can expect that to take about five years before all the currently eligible patients may get cleared through that queue," he said.

If Medicare and other payers ultimately decide to cover lecanemab, it could cost tens of billions of dollars each year, Hlávka added. Given the high price tag, officials may consider a different approach to caring for people with dementia.

"One of the potential solutions would be to see if we can pool all of the dementia patients who are covered under different plans into a single risk pool and then provide coordinated access and care to those patients," Hlávka said.

That would allow the government to balance lecanemab's cost against potential savings if dementia patients are able to avoid expensive nursing home care, Hlávka said. And it would make it easier for the government to negotiate lecanemab's price. (Alltucker/Hassanein, USA Today, 1/8; Belluck, New York Times, 1/6; Hamilton, "Shots," NPR, 1/6; Gonzalez, Axios, 1/6; Perrone, Associated Press, 1/6; Walker, Wall Street Journal, 1/7)