February 8, 2023

WASHINGTON (AP) — U.S. health officials on Friday approved an Alzheimer’s drug that has been shown to moderately slow the early stages of the brain-robbing disease, albeit with potential safety risks that doctors and patients will have to weigh. carefully.

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

Leqembi, from Japan’s Eisai and its US partner Biogen, is an exceptional success in a field accustomed to failed experimental treatments for the incurable disease. The delay in cognitive decline caused by the drug probably amounts to only several months, but some experts say it could still significantly improve people’s lives.

“This medicine is not a cure. It doesn’t prevent people from getting worse, but it slows the progression of the disease considerably,” 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 to be able to drive.”

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

The approval came through the FDA’s fast track, which allows the launch of drugs based on early results, before they are confirmed to benefit patients. The agency’s use of that shortcut has come under increasing scrutiny. from government watchdogs and congressional investigators.

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

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

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The drug will cost $26,500 for a typical year of treatment. Eisai said the price reflects the drug’s benefit in terms of improved quality of life, reduced burdens on caregivers and other factors. The company estimated its total value at $37,000 per year, but said it priced the drug lower to reduce costs for patients and insurers. An independent group that assesses the value of drugs recently said the drug would have to be priced below $20,600 to be profitable.

Some 6 million people in the US and many more around the world have Alzheimer’s, which gradually attacks areas of the brain needed for memory, reasoning, communication and daily tasks.

The FDA approval was based on a mid-stage study of 850 people with early symptoms of Alzheimer’s who also tested positive for a type of brain plaque that is a hallmark of the disease.

Since then, Eisai has published the results of a larger study of 1,800 patients. which the FDA is expected to review soon to confirm the drug’s benefit, with a decision on full approval set for later this year.

The largest study tracked patient outcomes on an 18-point scale that measures memory, judgment, and other cognitive abilities. Doctors compile the rating from patient interviews and close contact. After 18 months, the patients who received Leqembi declined more slowly (a difference of less than half a point on the scale) than the patients who received a dummy infusion. The delay amounted to just over five months.

There is little consensus on whether that difference translates into 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.”

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

Leqembi works by removing a sticky brain protein called amyloid that is a key indicator of Alzheimer’s disease. But it’s not clear exactly what causes the disease. A number of other drugs targeted against amyloid have failed, and many researchers now believe that combination treatments will be needed.

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

The FDA approved that drug in 2021 against the advice of the agency’s own outside experts.. Doctors were hesitant to prescribe the drug and insurers restricted coverage.

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

Rep. Rosa DeLauro of Connecticut, a frequent critic of the FDA, said in a statement Friday that she was “deeply concerned that the FDA decided to waive” a panel meeting on the drug.

Schrag said many of the same concerns apply to both drugs.

“Is this small, measurable benefit and the side effects that patients may experience worth paying the high price for?” she asked. “I have pretty serious doubts.”

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

Additionally, several Leqembi users have died while taking the drug, including two who were taking blood thinners. Eisai has said the deaths cannot be attributed to the drug. The FDA label warns doctors to be careful if they prescribe Leqembi to patients taking blood thinners.

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Insurers will likely only cover the drug for people like those in the company’s study: patients with mild dementia and confirmed amyloid buildup. That usually requires expensive brain scans or a spinal fluid test. Doctors will need to perform a different type of scan to periodically check for swelling and bleeding in the brain.

A key issue in the drug’s launch will be insurance coverage by Medicare, the federal health plan that covers 60 million seniors and other Americans. The agency severely restricted coverage of Aduhelm, essentially killing off its US market and prompting Biogen to abandon marketing plans for 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, which is expected later this year.

“Once we have a decision on Medicare, we can really roll out the drug nationwide,” said Ivan Cheung, US CEO of Eisai.

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

His initial diagnosis, based on a cognitive exam, was later confirmed by a positive amyloid test.

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

“For me, the moment the drug comes on the market, and I get my doctor’s approval, I’ll take it,” Groves said.

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AP Medical Writer Lauran Neergaard contributed to this report

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