WASHINGTON — U.S. health officials on Friday approved a closely watched Alzheimer’s drug that modestly slows the brain-robbing disease, despite its potential risks safety concerns that patients and their physicians will have to carefully consider.
Leqembi is the first drug to be convincingly shown to slow the memory and thinking processes that define Alzheimer’s disease by targeting the underlying biology of the disease. The Food and Drug Administration has approved it for patients with Alzheimer’s disease, especially those with mild or early-stage disease.
Leqembi, from Japan’s Eisai and US partner Biogen, is a rare success in the field of familiarity with failed experimental treatments for incurable diseases. The delay in drug-induced cognitive decline can last only a few months, but some experts say it can still significantly improve people’s lives.
“This medicine is not a cure-all. Joy Snider, a neuroscientist at Washington University in St. Louis, said it doesn’t stop people from getting worse, but it slows the progression of the disease significantly. “That means someone might have an extra six months to a year to be able to drive.”
Snider stresses that the drug, pronounced “leh-KEM-bee,” comes with downsides, including the need for a twice-monthly infusion and possible side effects like brain swelling.
FDA approval comes through an accelerated pathway, allowing drugs to launch based on initial results, before they are confirmed to benefit patients. The agency’s use of that shutdown method has come under increasing scrutiny from government watchdogs and congressional investigators.
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Last week, a congressional report found that the FDA’s approval of a similar Alzheimer’s drug called Aduhelm – also from Biogen and Eisai – was “fraught with anomalies”, including several meetings with undocumented pharmaceutical company employees.
The scrutiny of the new drug, chemically known as lecanemab, is likely to mean that most patients won’t start receiving it for months, as insurance companies decide whether to cover it. or not and how.
The drug will cost about $26,500 for a typical year of treatment. Eisai said drug prices reflect the benefits of drugs in improving quality of life, reducing burden on caregivers and other factors. The company has pegged its value at more than $37,000 per year, but said it lowers the price to reduce costs for patients and insurers. An independent drug value assessment group recently said the drug would have to cost less than $20,600 per year to be cost-effective.
About 6 million people in the United States and many others worldwide have Alzheimer’s, a disease that gradually attacks brain regions essential for memory, reasoning, communication, and everyday tasks.
The FDA approval was based on a mid-term study in 800 people with early signs of Alzheimer’s, who were still able to live independently or with minimal help.
Since then, Eisai has published the results of a larger study of 1,800 patients that the FDA will review to confirm the drug’s benefits, paving the way for full approval later this year.
The larger study tracked patient outcomes on an 18-point scale that measures memory, judgment, and other cognitive abilities. Physicians synthesize assessments from interviews with patients and a close contact. After 18 months, patients receiving Leqembi declined more slowly – less than half a point on the scale – compared with patients receiving 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 a difference,” said Dr. Matthew Schrag, a neurologist at Vanderbilt University. “This is actually a fairly small effect and is probably below the threshold of what we would call clinically significant.”
Schrag and several other researchers believe that a meaningful improvement would require at least one difference on an 18-point scale.
Leqembi works by removing a sticky protein in the brain called amyloid, a hallmark of Alzheimer’s disease. But it is not clear exactly what causes the disease. A host of other amyloid-targeted drugs have failed, and many researchers now think that combined treatments will be needed.
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Aduhelm, the same drug, was marred by controversy over its effectiveness.
The FDA approved that drug in 2021 on the advice of experts outside of the agency. Doctors were hesitant to prescribe drugs, and insurance companies limited coverage.
The FDA did not consult with the same panel of experts before approving Leqembi.
Although there is “less drama” around the new drug, Schrag said many of the same concerns apply.
“Is this small, measurable benefit worth the hefty price tag and possible side effects that patients may experience?” he asks. “I have pretty serious doubts.”
About 13% of the patients in Eisai’s study had brain swelling and 17% had minor bleeding in the brain, a side effect seen with previous amyloid-targeting drugs. In most cases, those problems cause no symptoms, which can include dizziness and vision problems.
In addition, several people taking Leqembi have died while taking the drug, including two who were taking blood thinners. Eisai has said that the deaths cannot be attributed to drugs. The FDA label warns doctors to exercise caution if they prescribe Leqembi to patients taking blood thinners.
Insurers are likely to only cover the drug for people like those in the company’s study – patients with mild symptoms and confirmed amyloid buildup. That often requires expensive brain scans. A separate type of scan is required to periodically monitor brain swelling and bleeding.
A key question in drug rollout will be coverage decisions by Medicare, the federal health program that covers 60 million seniors and other Americans. The agency severely restricted its coverage of Aduhelm, essentially wiping out its US market and causing Biogen to abandon marketing plans for the drug.
Eisai executives said they spent months discussing their drug data with Medicare officials. Coverage is not expected until after the FDA confirms the benefit of the drug, possibly later this year.
“Once we have a decision on Medicare, we can really roll it out nationally,” said Eisai US CEO Ivan Cheung.
Betsy Groves, 73, of Cambridge, Massachusetts, was diagnosed with Alzheimer’s disease in 2021. Once a lecturer at the Harvard School of Education, she found she had trouble remembering some students’ names and answering questions. .
Her initial diagnosis, based on cognitive testing, was later confirmed by a positive amyloid test.
Groves said she was “very willing” to try Leqembi, despite the potential side effects and the need for fluids.
“For me, as soon as that drug hits the market — and I get my doctor’s approval — I take it,” says Groves.
