FDA approves Leqembi, Alzheimer’s drug from Eisai and Biogen

WASHINGTON. On Friday, U.S. health officials approved a closely watched Alzheimer’s drug that marginally slows the brain-consuming disease, albeit with potential safety risks that patients and their doctors will have to weigh carefully.

Lekambi is the first drug that has been convincingly shown to slow the deterioration of memory and thinking that is characteristic of Alzheimer’s disease by acting on the underlying biology of the disease. The Food and Drug Administration has approved it for Alzheimer’s patients, especially those with mild or early disease.

Lekambi, from Japan’s Eisai and its US partner Biogen, has achieved rare success in a field accustomed to failed experimental treatments for an incurable disease. The delay in cognitive decline caused by the drug is likely only a few months, but some experts say it can still make a big difference in people’s lives.

“This drug is not a cure. It doesn’t stop people from getting worse, but it does noticeably slow the progression of the disease,” said Dr. Joy Snyder, a neuroscientist at Washington University in St. Louis. “That could mean someone could have anywhere from six months to a year to be able to drive.”

Snyder stressed that the drug, pronounced “leh-KEM-bee”, has drawbacks, including the need for a twice-monthly infusion and possible side effects such as brain swelling.

FDA approval came through an accelerated path that allows drugs to be launched based on early results before they are proven to be of benefit to patients. The agency’s use of this simplistic approach has come under increasing scrutiny from government monitors and congressional investigators.

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Last week, a congressional report showed that the FDA’s approval of a similar Alzheimer’s drug called Aduhelm — also by Biogen and Eisai — was “rife with irregularities,” including a number of meetings with pharmaceutical company employees that have gone undocumented.

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

The drug will cost about $26,500 for a typical year of treatment. Eisai said the price reflects the benefit of the drug in terms of improving quality of life, reducing the burden on caregivers and other factors. The company has valued it at over $37,000 a year, but said it set the price lower to cut costs for patients and insurers. An independent drug pricing group recently said that a drug must cost less than $20,600 a year to be profitable.

Approximately 6 million people in the US and many more around the world suffer from Alzheimer’s disease, which progressively affects areas of the brain needed for memory, reasoning, communication, and daily tasks.

The FDA approval was based on one interim study of 800 people with early signs of Alzheimer’s who were still able to live on their own or with minimal assistance.

Eisai has since published the results of a larger study involving 1,800 patients, which 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. Doctors make a rating from a conversation with the patient and close contact. After 18 months, patients treated with Lekambi had a slower decline—less than a half point difference on the scale—compared with patients receiving a sham infusion. The delay was just over five months.

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

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

Schrug and some other researchers believe that meaningful improvement would require at least one full point difference on an 18-point scale.

Lekambi clears the brain of a sticky protein called amyloid, which is one of the hallmarks of Alzheimer’s disease. But it is not clear what exactly causes the disease. A number of other amyloid-targeting drugs have failed, and many researchers now believe that combination treatments will be needed.

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Aduhelm, a similar drug, has been overshadowed by controversy over 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 insurers limited coverage.

The FDA did not consult the same panel before approving Lekambi.

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

“Is this small, measurable benefit worth the high cost and side effects that patients may experience?” he asked. I have some pretty serious doubts.

About 13% of patients in Eisai’s study had cerebral edema, and 17% had minor cerebral hemorrhages, side effects seen with earlier amyloid-targeting drugs. In most cases, these problems did not cause symptoms, which may include dizziness and vision problems.

In addition, several Leqembi users have died while taking the drug, including two who were taking blood-thinning drugs. Eisai said that the death could not be related to drugs. The FDA label warns physicians to use caution if they prescribe Lekambi to patients taking anticoagulants.

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

A key issue in the introduction of the drug will be the decision on coverage by Medicare, the federal health insurance plan that covers 60 million seniors and other Americans. The agency drastically curtailed the reach of Aduhelm, effectively wiping out its US market and forcing Biogen to scrap the drug’s marketing plans.

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

“Once we make a decision on the Medicare program, we can really launch the drug across the country,” said Eisai US CEO Ivan Chung.

Betsy Groves, 73, from Cambridge, Massachusetts, was diagnosed with Alzheimer’s disease in 2021. The former teacher at Harvard Normal School noted that she had difficulty remembering the names of some students and answering questions.

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

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

“For me, the minute this drug hits the market – and I get my doctor’s approval – I’ll take it,” Groves said.

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