The United States approves an Alzheimer’s drug that modestly slows the disease

Washington – US health officials on Friday approved a closely watched Alzheimer’s drug that modestly slows the brain-stealing disease, albeit with potential safety risks that patients and their doctors will have to carefully evaluate.

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

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

“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 Snyder, a neurologist at Washington University in St. Louis. “It could mean someone could get an extra six months to a year to be able to drive.”

Snyder stressed that the drug, which is called “leh-KEM-bee,” comes with downsides, including the need for injections twice a month and potential side effects like brain swelling.

The FDA approval came through its accelerated path, which allows drugs to be released 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 oversight agencies and congressional investigators.

Last week, a congressional report found that the Food and Drug Administration’s approval of a similar Alzheimer’s drug called Aduhelm — also from Biogen and Eisai — was “riddled with irregularities,” including a number of meetings with drug company employees that were not documented.

Screening of the new drug, known chemically as lecanemab, will likely mean most patients won’t start receiving it for several months, as insurance companies decide if and how to cover it.

The drug will cost about $26,500 for a year of treatment. Esay said the price reflects the drug’s benefit in terms of improving quality of life, reducing burdens on caregivers, and other factors. The company has valued it at more than $37,000 annually, but said it has lowered its prices to reduce costs to patients and insurers. An independent group evaluating property value recently said the property must be priced at less than $20,600 per year to be cost-effective.

About 6 million people in the United States and many more worldwide suffer from Alzheimer’s disease, which progressively attacks areas of the brain needed for memory, thinking, communication, and everyday tasks.

The FDA approval was based on one mid-stage study of 800 people with early signs of Alzheimer’s disease who were still able to live independently or with minimal assistance.

Since then, Esay has published the results of a larger study of 1,800 patients that the Food and Drug Administration will review to confirm the drug’s benefit, paving the way for full approval later this year.

The larger study tracked patients’ outcomes on an 18-point scale that measures memory, judgment and other cognitive abilities. Clinicians compile the rating from patient interviews and close contact. After 18 months, patients receiving Leqembi declined more slowly — a difference of less than half a point on the scale — than patients given the placebo. The delay amounted to just over five months.

There is little consensus on whether this difference translates into real benefits for patients, such as increased independence.

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

Schrag and some other researchers believe that serious improvement requires at least a difference of one full point on an 18-point scale.

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

Aduhelm, the similar drug, has been marred by controversy over its effectiveness.

The US Food and Drug Administration approved this drug in 2021 against advice from the agency’s outside experts. Doctors hesitated in prescribing the drug and underwriting the insurance companies.

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 the side effects patients may experience?” Asked. “I have serious doubts.”

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

Several Leqembi users have also died while taking the drug, including two who were taking blood thinners. Esay said the deaths could not be attributed to the drug. The FDA label warns doctors to use caution if they prescribe Leqembi to patients on blood thinners.

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

The main question in bringing up the drug will be the coverage decision by Medicare, the federal health plan that covers 60 million seniors and other Americans. The agency severely restricted coverage of Aduhelm, essentially eliminating its market in the United States and prompting Biogen to abandon plans to market the drug.

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

“Once we get the Medicare decision, we can really launch the drug across the country,” said Eisai US CEO Evan Cheung.

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

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

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

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