U.S. health officials on Friday approved a closely watched Alzheimer’s drug that only slightly slows the disease in the brain, but with potential safety risks that patients and their doctors will need to watch carefully.
Lekembe is the first drug that has been convincingly shown to slow the decline in memory and thinking that characterizes 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.
Likembe, from the Japanese company Eisai and its American partner Biogen, has achieved rare successes in a field that has become accustomed to the failure of experimental treatments for difficult cases. The delay in cognitive decline from the drug may only be months, but Dr. Joy Snyder and other experts say it can still significantly improve people’s lives.
“This drug is not a cure. It doesn’t prevent people from cognitive decline, but it does significantly slow disease progression,” said Snyder, a neurologist at Washington University in St. Louis.
Snyder pointed out that the drug has drawbacks, including the need for twice-monthly injections and potential side effects such as brain swelling.
Approval from the US Food and Drug Administration came through its fast track, which allows drugs to be released based on early results, before they are confirmed for patients. The agency’s use of this simplified approach has come under increasing scrutiny from government watchdog agencies and congressional investigators.
And last week, a congressional report found that the Food and Drug Administration’s approval of a similar Alzheimer’s drug called Aduhelm (also made by Biogen and Essay) was “flawed.”
The review of the new drug, known chemically as lecanemab, likely means most patients won’t start receiving it for several months as insurers decide if and how to cover it.
About 6 million people in the United States and many others around the world suffer from Alzheimer’s disease, which gradually attacks areas of the brain needed for memory, thinking, communication and daily tasks.
The FDA approval was based on a mid-term study of 800 people with early signs of Alzheimer’s disease who were still able to live independently or with minimal assistance.
Since then, Esay has released the results of a larger study involving 1,800 patients that the Food and Drug Administration will review to confirm the drug’s benefits, paving the way for full approval later this year.
The larger study tracked patients’ results on an 18-point scale that measures memory, judgment and other cognitive abilities. Clinicians compiled the assessment from patient interviews and close contacts.
After 18 months, the number of patients receiving Lyquimbi gradually decreased – by a difference of less than half a point on the scale – compared to patients who received a dummy injection. The delay amounted to just over five months.
There is little consensus on whether this difference translates into real benefits for patients, such as greater independence.
“Most patients won’t notice a difference,” said Vanderbilt University neuroscientist Dr. Matthew Schrag. “It’s really a very small effect and probably below the threshold of what we call clinically important. .”
Schrag and a few other researchers believe that significant improvement would require at least a full point difference on the 18-point scale.
The drug works by breaking down a sticky brain protein called amyloid, which is one of the hallmarks of Alzheimer’s disease. But it is not known exactly what causes the disease. A series of other amyloid-targeting drugs have failed, and many researchers now believe that combination therapies are needed.
Adohelm, a similar drug, has been marred by controversy over its effectiveness.
The US Food and Drug Administration approved the drug, in 2021 against independent expert advice from the agency, doctors have been reluctant to prescribe the drug and insurance companies have restricted coverage.
The FDA did not consult the same group of experts before approving Lekembe.
While there’s “less drama” around the new property, Schrag said many of the same concerns apply.
“Is this small measurable benefit worth the high price and the side effects patients may experience?” he asked. “I have very serious doubts.
About 13% of patients in Esay’s study had brain swelling and 17% had small bleeding in the brain, side effects seen with previous amyloid-targeting drugs. In most cases, these problems did not cause symptoms, which can include dizziness and vision problems.
Several Lekmbe users have also died while taking the drug, including two who were taking blood thinners. Esay said the deaths could not be attributed to drugs. The Food and Drug Administration label warns physicians to exercise caution when prescribing Lycombe to patients taking blood thinners.
Insurance companies will likely only cover the drug for people like those in the company’s study – patients with mild symptoms and confirmed amyloid buildup. This usually requires expensive brain scans. A separate type of scan will be needed to periodically monitor swelling and bleeding in the brain.
The main issue in discussing the drug will be the coverage decision by Medicare, the federal health plan that covers 60 million seniors and other Americans.
Esay executives said they have already spent months discussing their drug data with Medicare officials. Coverage is not expected until the US Food and Drug Administration confirms the drug’s benefits, likely later this year.
“Once we have a decision on Medicare, we can really launch the drug nationwide,” said Esaie’s U.S. CEO Evan Cheung.
Betsy Groves, 73, from Cambridge, Massachusetts, was diagnosed with Alzheimer’s disease in 2021. A former lecturer at Harvard School of Education, she noticed she had trouble remembering the names of some students and answer questions.
His initial diagnosis, based on a cognitive scan, was later confirmed by a positive test for amyloid.
Groves says she’s “more than willing” to try the new drug, despite the potential side effects and the need for injections.
“For me, as soon as this drug is on the market, and I have my doctor’s approval, I will take it,” she said.
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