FDA approves Alzheimer’s drug that slows the disease modestly

American health authorities approved on Friday a followed closely Alzheimer’s drug that slows the brain-robbing disease modestly, albeit with potential safety risks that patients and their doctors must weigh carefully.

The drug, Leqembi, is the first to be convincingly shown to slow the decline in memory and thinking that defines Alzheimer’s by targeting the disease’s underlying biology. The Food and Drug Administration approved it for patients with Alzheimer’s, especially those with mild or early disease.

Leqembi, from Japan’s Eisai and its US partner Biogen, is a rare success in a field accustomed to failed experimental treatments for the incurable condition. The delay in cognitive decline caused by the drug is likely to be only several months, but some experts say it can still improve people’s lives in a meaningful way.

“This drug is not a cure. It doesn’t stop people from getting worse, but it measurably slows the progression of the disease,” said Dr. Joy Snider, a neurologist at Washington University in St. Louis. “That could mean someone could have an extra six months to a year of being able to drive.”

Snider stressed that the drug, pronounced “leh-KEM-bee,” comes with drawbacks, including the need for twice-monthly infusions and possible side effects such as brain swelling.

The FDA approval came via its accelerated pathway, which allows drugs to be launched based on early results, before they are confirmed to benefit patients. The agency’s use of this shortcut approach has come under increasing scrutiny from government watchdogs and congressional investigators.

Last week, a congressional report found that FDA approval of a similar Alzheimer’s drug called Aduhelm – also from Biogen and Eisai – was “riddled with irregularities”, including a series of meetings with drug company employees that went undocumented.

Scrutiny of the new drug, known chemically as lecanemab, is likely to mean most patients won’t start receiving it for months, as insurers decide whether and how to cover it.

The drug will cost about $26,500 for a typical year’s treatment. Eisai said the price reflects the drug’s benefit in terms of improved quality of life, reduced burdens for caregivers and other factors. The company pegged the value at more than $37,000 per year, but said it priced it lower to reduce costs for patients and insurers. An independent group assessing the drug’s value recently said the drug would need to be priced below $20,600 per year to be cost-effective.

About 6 million people in the United States and many more worldwide have Alzheimer’s, which gradually attacks areas of the brain needed for memory, reasoning, communication and daily tasks.

The FDA’s approval was based on a study of over 800 people with early signs of Alzheimer’s who were still able to live independently or with minimal assistance.

Since then, Eisai has published the results of a larger 1,800-patient study that the FDA will review to confirm the drug’s benefit, paving the way for full approval later this year.

The larger study tracked patients’ performance on an 18-point scale that measures memory, judgment and other cognitive abilities. The doctors compile the assessment from interviews with the patient and a close contact. After 18 months, patients receiving Leqembi declined more slowly – a difference of less than half a point on the scale – than patients receiving a dummy infusion. The delay was just over five months.

There is little agreement as to whether this difference can lead to real benefits for patients, such as greater independence.

“Most patients won’t notice the difference,” said Dr. Matthew Schrag, a neurology researcher at Vanderbilt University. “This is really quite a small effect and probably below the threshold of what we would call clinically significant.”

Schrag and some other researchers believe a meaningful improvement would require at least a difference of one full point on the 18-point scale.

Leqembi works by removing a sticky brain protein called amyloid that is a hallmark of Alzheimer’s. But it is not clear exactly what causes the disease. A number of other amyloid-targeting drugs have failed, and many researchers now believe combination therapies will be necessary.

Aduhelm, the similar drug, was marred by controversy about its effectiveness.

The FDA approved the drug in 2021 against the advice of the agency’s own outside experts. Doctors hesitated to prescribe the drug, and insurance companies limited coverage.

The FDA did not consult the same expert panel before approving Leqembi.

While there is “less drama” surrounding the new drug, Schrag said many of the same concerns apply.

“Is this small, measurable benefit worth the high price tag and side effects patients may experience?” he asked. – I have quite serious doubts.

About 13% of patients in Eisai’s study had swelling in the brain and 17% had small brain bleeds, side effects seen with previous amyloid-targeted drugs. In most cases, these problems did not cause symptoms, which can include dizziness and vision problems.

Also, several Leqembi users died while taking the drug, including two who were on blood-thinning medication. Eisai has said the deaths cannot be attributed to the drug. The FDA label warns doctors to use caution if they prescribe Leqembi to patients on blood thinners.

Insurers are likely to only cover the drug for people like those in the company’s study — patients with mild symptoms and confirmation of amyloid buildup. It usually requires expensive brain scans. A separate type of scan will be needed to periodically monitor for swelling and bleeding in the brain.

A key issue in the drug’s rollout will be the coverage decision by Medicare, the federal health plan that covers 60 million seniors and other Americans. The agency severely limited coverage of Aduhelm, essentially wiping out the US market and causing Biogen to abandon marketing plans for the drug.

Eisai executives said they have already spent months discussing the drug’s data with Medicare officials. Coverage is not expected until after the FDA confirms the drug’s benefit, likely later this year.

“Once we have a Medicare decision, we can really launch the drug nationwide,” said Eisai’s U.S. CEO, Ivan Cheung.

Betsy Groves, 73, of Cambridge, Massachusetts, was diagnosed with Alzheimer’s in 2021. A former lecturer at Harvard’s School of Education, she noticed she had trouble remembering some students’ names and answering questions.

Her initial diagnosis, based on a cognitive examination, was later confirmed by a positive test for amyloid.

Groves says she is “more than willing” to try Leqembi, despite the potential side effects and the need for infusions.

“For me, the minute the drug comes on the market — and I get the doctor’s approval — I’m going to take it,” Groves said.

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