US health officials on Friday approved a closely monitored Alzheimer’s drug that only slightly slows the disease in the brain, albeit with potential safety risks that patients and their doctors will have to monitor carefully.
Lekembe is the first drug shown convincingly 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 Alzheimer’s patients, specifically those with mild or early-stage disease.
Likembe, of the Japanese company Eisai and its US partner Biogen, has achieved rare success in a field that has become accustomed to failed experimental treatments for difficult cases. The delay in cognitive decline as a result of the drug may only be several months, but Dr. Joy Snyder and some other experts say it can still meaningfully improve people’s lives.
“This drug is not a cure. It doesn’t prevent people from cognitive decline, but it does slow the progression of the disease significantly,” said Snyder, a neurologist at Washington University in St. Louis. ability to drive.”
Snyder stressed that the drug comes with downsides, including the need for injections twice a month and potential side effects such as brain swelling.
The US Food and Drug Administration’s approval came through its accelerated course, which allows drugs to be released based on early results, before they are confirmed to benefit patients. The agency’s use of this shortcut approach has come under increasing scrutiny from government oversight 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 “riddled with irregularities.”
Scrutiny of the new drug, known chemically as lecanemab, likely means most patients will not 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 more people around the world suffer from Alzheimer’s disease, which progressively attacks areas of the brain needed for memory, thinking, communication and everyday tasks.
The FDA approval was based on one mid-stage 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 published the results of a larger study of 1,800 patients that the Food and Drug Administration will review to confirm the drug’s benefit, paving the way for full approval later this year.
The larger study tracked patients’ outcomes on an 18-point scale that measures memory, judgment and other cognitive abilities. The clinicians compiled the rating from patient interviews and close contact.
After 18 months, the number of patients receiving Lyquimbi gradually decreased — by a difference of less than half a point on the scale — than 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 will not notice a difference,” said Dr. Matthew Schrag, a neuroscience researcher at Vanderbilt University. “This is really a very small effect and probably below the threshold of what we call clinically significant.”
Schrag and some other researchers believe that a meaningful improvement would require at least a difference of one whole point on the 18-point scale.
The drug works by removing a sticky brain protein called amyloid, which is one of the hallmarks of Alzheimer’s disease. But it is not clear exactly what causes the disease. A series of other amyloid-targeting drugs have failed and many researchers now believe combination therapies are needed.
Adohelm, a similar drug, has been marred by controversy over its effectiveness.
The US Food and Drug Administration approved this drug, in 2021, against the advice of experts independent of the agency, doctors have been reluctant to prescribe the drug and insurance companies have restricted coverage.
The FDA did not consult the same panel of experts before approving Lekembe.
While there is “less drama” surrounding the new property, Schrag said many of the same concerns apply.
“Is this small, measurable benefit worth the high price and side effects that patients may experience?” he asked. “I have very serious doubts.”
About 13 percent of the patients in Esay’s study had brain swelling and 17 percent had a small bleed 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 the drug. The Food and Drug Administration label warns doctors to use caution if they prescribe Lycombe to patients who are 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 monitor brain swelling and bleeding periodically.
The main question in bringing up 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 after the US Food and Drug Administration confirms the drug’s benefit, likely later this year.
“Once we have a Medicare decision, we can really launch the drug across the country,” said Esaie’s US CEO, Evan Cheung.
Betsy Groves, 73, of Cambridge, Massachusetts, was diagnosed with Alzheimer’s disease in 2021. She is a former lecturer at Harvard University’s School of Education, and noticed that she had trouble remembering some students’ names and answering questions.
Her initial diagnosis, based on a cognitive scan, was later confirmed by a positive test for amyloid.
Groves says she is “more than willing” to try the new drug, despite the potential side effects and need for injections.
“For me, as soon as this drug is on the market, and I get my doctor’s approval, I will take it,” she said.