US health officials on Friday approved a closely watched Alzheimer’s drug that modestly slows the brain-robbing disease, albeit with possible safety risks that patients and their doctors 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 US Food and Drug Administration (FDA) has approved it for Alzheimer’s patients, specifically those with mild or early-stage disease.
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 drug is not a cure. It doesn’t stop people from getting worse, but it does slow 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 has drawbacks, including the need for twice-monthly infusions and possible side effects such as brain swelling.
The FDA’s approval came through its fast-track, which allows drugs to be launched 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 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 series of meetings with pharmaceutical company staff. that were left 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 how to cover it.
The drug will cost about US$26,500 (CAD$35,613) 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 pegged its value at more than US$37,000 (C$49,725) per year, but said it priced it lower to reduce costs for patients and insurers. An independent group that assesses the value of drugs recently said that the price of the drug would need to be less than US$20,600 (CAD$27,684) per year to be profitable.
About six 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 in 800 people with early signs of Alzheimer’s who were still able to live independently or with minimal assistance.
Eisai has since published the results of a larger study of 1,800 patients that the FDA will review to confirm the drug’s benefit, paving the way for full approval later this year.
Today we are approving a new drug for the treatment of Alzheimer’s disease, the second in a new category of drugs approved for Alzheimer’s disease that focus on the fundamental pathophysiology of the disease. https://t.co/ZXJCUuTYPk pic.twitter.com/TV36k6CO6K
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.”
Schrag and some other researchers believe that significant improvement would require at least a full point difference on the 18-point scale.
‘I have pretty serious doubts’
Leqembi works by removing a sticky protein from the brain called amyloid, which is a hallmark of Alzheimer’s. 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 hesitated to prescribe the drug and insurers restricted coverage.
The FDA did not consult the same panel of experts 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 and the side effects that patients may experience?” she asked. “I have pretty serious doubts.”
About 13 percent of the patients in Eisai’s study had brain swelling and 17 percent 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.
Insurers will likely only cover the drug for people like those in the company’s study: patients with mild symptoms and confirmed amyloid buildup. That usually requires expensive brain scans. A separate type of scan will be needed to regularly monitor brain swelling and bleeding.
question about medicare
A key issue in the drug’s launch 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 killing off its US market and prompting Biogen to drop 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, likely later this year.
“Once we have a decision on Medicare, we can really launch the drug nationwide,” said Ivan Cheung, Eisai’s US CEO.
Betsy Groves, 73, of Cambridge, Massachusetts, was diagnosed with Alzheimer’s in 2021. A former professor at the Harvard 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 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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