HO CHI MINH CITY –
U.S. health officials on Friday approved a closely watched Alzheimer’s drug that has been shown to modestly slow the early stages of the brain-robbing disease, despite its risks potential safety risks that physicians and patients 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 specifically for patients with mild or early dementia.
Leqembi, from Japan’s Eisai and US partner Biogen, is a rare success in a field already accustomed to failed experimental treatments for the incurable disease. 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.
Joy Snider, a neuroscientist at Washington University in St. Louis, said: “This drug is not a cure-all. It doesn’t stop people from getting worse, but it slows the progression of the disease significantly.” “That could mean someone could have an extra six months to a year to be able to drive.”
Snider stresses that the drug, pronounced “leh-KEM-bee,” comes with drawbacks, including the need for a twice-monthly infusion and possible side effects such as brain swelling and bleeding. blood.
The approval comes through the FDA’s accelerated pathway, which allows drugs to go to market based on initial results, before they are confirmed to be beneficial for patients. The agency that used that shortcut came under increasing scrutiny from government watchdogs and congressional investigators.
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 irregularities”, including a number of 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 the drug. this or not and for whom.
The drug will cost $26,500 for a typical year of treatment. Eisai says the price reflects the drug’s benefits in improving quality of life, reducing burden on caregivers, and other factors. The company pegged its overall value at $37,000 per year, but said it priced the drug lower 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 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 is based on a mid-term study in 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 that the FDA is expected to review soon to confirm the drug’s benefits, giving it 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 well below the threshold that 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 key marker 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.
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.
Rep. Rosa DeLauro of Connecticut – a frequent critic of the FDA – said in a statement Friday that she is “deeply concerned that the FDA has chosen to waive” a panel meeting on the drug.
Schrag said many of the same concerns apply to both drugs.
“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 dementia and confirmed amyloid buildup. That usually requires expensive brain scans or spinal fluid tests. Doctors will need to perform another type of scan to periodically check for swelling and bleeding in the brain.
A key question in drug rollout will be coverage of 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, which is expected later this year.
“Once we have a decision on Medicare, then we can really roll out the drug nationally,” said Ivan Cheung, CEO of Eisai in the US.
Betsy Groves, 73, was diagnosed with Alzheimer’s in 2021. Once a lecturer at Harvard’s graduate school, 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, of Cambridge, Massachusetts, 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 comes on the market – and I get my doctor’s approval – I’ll take it,” Groves said.
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AP Health Writer Lauran Neergaard contributed to this report
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The Associated Press Health and Science Division receives support from the Howard Hughes Medical Institute Science and Media Education Group. AP is solely responsible for all content.
