The health authorities of the United States approved this Friday 6, a new drug for the treatment of Alzheimer’s. While the drug has shown results that modestly delay disease, the drug also has potential patient safety risks, to the point where patients and their doctors require careful evaluation of its use due to its side effects.
The drug, called Leqembi, is the first to convincingly demonstrate the ability to delay the decline in memory and thinking, the defining symptoms of Alzheimer’s disease. The regulatory agency in the United States (FDA, its acronym in English, the equivalent of Anvisa in Brazil) has approved the medicine specifically for those with mild or early-stage disease.
Leqembi, from Japan’s Eisai and US partner Biogen, is a rare success in a field accustomed to failed experimental treatments for a disease considered incurable. Although the drug only manages to delay cognitive decline for a few months, some experts say the drug can still significantly improve people’s lives.
“This drug is not a cure. It doesn’t stop people from getting worse, but it slows the progression of the disease considerably,” said Joy Snider, a neurologist at Washington University in St. Louis. Louis. “This could mean that someone might have another six months or a year to be able to drive, for example,” she says. Snider pointed out, however, that the drug has drawbacks, including the need for twice-monthly infusions and possible side effects such as brain swelling.
In late November, the data released offered the first detailed look at the drug’s effects. The preview of the results, also in September, caused the shares of both companies to rise at the time.
At the time, specialists were even enthusiastic about Leqembi’s results, but they were also reticent about the drug’s effects. “The benefit is real, and so are the risks,” said Jason Karlawish, then co-director of the Penn Memory Center at the University of Pennsylvania. He is not involved in the research.
Quick approval
The FDA approval came through a bureaucratic shortcut that allows drugs to be released based on initial results, before confirming patient benefits. The agency’s use of this approach has come under increasing criticism 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 “riddled with irregularities,” including a series of meetings with drug company officials who had not been documented. Scrutiny of the new drug, known chemically as lecanemab, is likely to mean most patients won’t start taking it for months while insurers decide whether and how to cover it.
The drug will cost about US$26,500 (R$138,590) for a typical year of treatment. Eisai said the price reflects the drug’s benefits in terms of improving quality of life, reducing the burden on healthcare workers and other factors. The company set its value at more than $37,000 (R$193,500) a year but said it priced it lower to cut costs for patients and insurers. An independent group recently said that lecanemab’s price would need to be less than US$20,600 (R$107,730) a year to be profitable.
About 6 million people in the United States, and many more around the world, have Alzheimer’s, a disease that gradually attacks areas of the brain needed for memory, thinking, communication and daily activities.
Warnings
The FDA approval was based on an intermediate-stage study of 800 people who showed early signs of Alzheimer’s and were still able to live independently or with minimal assistance. Eisai has since released results from 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 compile the assessment from patient interviews and close contacts. After 18 months, participants who received Leqembi had slower disease progression — a difference of less than half a point on the scale — than patients who received a sham infusion. The “delay” was just over five months.
There is little consensus that this difference translates into real benefits for patients, such as greater independence. “Most patients won’t notice the difference,” said Matthew Schrag, a neurology researcher at Vanderbilt University. “This is actually a very small effect and probably below the threshold of what we would call clinically meaningful.” Schrag and some other researchers believe that significant improvement would require at least a complete difference on the scale.
Leqembi works by clearing a sticky brain protein called amyloid, which is a hallmark of Alzheimer’s disease, the cause of which is not yet clear. Several other amyloid-targeting drugs have failed, and many researchers now believe combination treatments will be needed.
Aduhelm, the similar drug, has been marred by controversy over its effectiveness. The FDA approved this drug in 2021 against the advice of the agency’s outside experts. Doctors have hesitated to prescribe the drug, and insurers have beefed up coverage. The FDA did not consult the same panel of experts before approving Leqembi.
While there’s “less drama” surrounding the new drug, Schrag said many of the same concerns apply. “Is this small, measurable benefit worth the high price of side effects patients can experience?” she wonders. “I have serious doubts.”
Aduhelm, the similar drug, has been marred by controversy over its effectiveness. The FDA approved this drug in 2021 against the advice of the agency’s outside experts. Doctors have hesitated to prescribe the drug, and insurers have beefed up coverage. The FDA did not consult the same panel of experts before approving Leqembi.
While there’s “less drama” surrounding the new drug, Schrag said many of the same concerns apply. “Is this small, measurable benefit worth the high price of side effects patients can experience?” she wonders. “I have serious doubts.”
Side effects
About 13% of patients in the Eisai study had brain swelling and 17% had minor bleeding in the brain, side effects seen with previous amyloid-targeting drugs. In most cases, these problems have caused no symptoms, which can include dizziness and vision problems. Additionally, several Leqembi users have died while taking the drug, including two who were taking blood-thinning medications.
Eisai said the deaths could not be attributed to drugs. The FDA label warns doctors to be careful if prescribing Leqembi to patients taking blood thinners. Insurers are likely to cover the drug only for people like those in the company’s study — patients with mild symptoms and confirmed amyloid buildup. This typically requires expensive brain scans. A separate type of scan will be needed to periodically monitor swelling and bleeding from the brain.
A key issue in the drug’s rollout will be the coverage decision for Medicare, the federal health plan that covers 60 million older and other Americans. The agency severely limited coverage of Aduhelm, essentially eliminating its US market and prompting Biogen to abandon marketing plans for the drug.
Eisai executives said they spent months discussing their drug data with Medicare officials. Coverage isn’t expected until the FDA confirms the drug’s benefits, likely later this year. “Once we make a decision about Medicare, we can really roll it out across the country,” said Ivan Cheung, CEO of Eisai US.
Betsy Groves, 73, of Cambridge, Massachusetts, was diagnosed with Alzheimer’s in 2021. A former professor at Harvard’s school of education, she noted that she was having difficulty remembering some students’ names and answering questions. Her initial diagnosis, based on a cognitive scan, was later confirmed by a positive test for amyloid.
She says she is “more than willing” to try Leqembi, despite possible side effects and the need for infusions. “For me, the moment the drug hits the market—and I get my doctor’s approval—I’ll take it,” she says.
(WITH INTERNATIONAL AGENCIES)
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