WASHINGTON (AP) – US health officials Friday approved a closely watched Alzheimer’s drug that appears to slow the disease that robs the brain, though with potential safety risks patients and their doctors will have to weigh carefully.
The drug, Lecanbi, is the first to be convincingly shown to slow the memory and thinking decline that defines Alzheimer’s by targeting the underlying biology of the disease. The Food and Drug Administration has approved it for Alzheimer’s patients, especially those with mild or early-stage disease.
Lekembi, from Japan’s Eisai and its US partner Biogen, is a rare breakthrough in a field accustomed to failed experimental treatments for the incurable condition. The drug is likely to delay cognitive decline by only several months, but Dr. Joy Snyder and some other experts say it could still meaningfully 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 significantly,” said Snyder, a neurologist at Washington University in St. Louis. “This could mean that someone could have an additional six months to a year to be able to drive.”
Snyder stressed that the drug, called “LEH-KEM-B,” comes with downsides, including the need for twice-a-month infusions and potential side effects such as brain swelling.
The FDA’s approval came through its accelerated route, which allows drugs to be launched based on preliminary results before they are confirmed to benefit patients. The use of that shortcut approach by the agency has come to the fore. increasing scrutiny From government watchdogs and congressional investigators.
last week, a congress report Found to have FDA approval for similar Alzheimer’s drug adhelm- — also from Biogen and Eisai — was “rife with irregularities,” including multiple meetings with drug company employees that went undocumented.
The investigation into the new drug, known chemically as lecanumab, will mean most patients won’t start receiving it for months, as insurers decide whether to cover it.
Some 6 million people in the US and many more worldwide have Alzheimer’s, which slowly attacks areas of the brain needed for memory, reasoning, communication and daily functions.
The FDA’s approval was based on a mid-stage study in 800 people with early symptoms of Alzheimer’s who were still able to live independently or with minimal assistance.
Since then, Eisai has published a large result 1,800 Patient Study That the FDA will conduct a 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. Doctors compile ratings from interviews and close contact with the patient. After 18 months, there was a more gradual decline in patients receiving Lekambi compared to patients receiving a dummy infusion – a difference of less than half on the scale. The amount of delay was just over five months.
There is little agreement on whether this difference translates into actual benefits for patients, such as greater independence.
Vanderbilt University neurology researcher Dr. “Most patients won’t notice a difference,” said Matthew Schrag. “It’s actually quite a small effect and probably below the threshold of what we call clinically significant.”
Schrag and some other researchers believe that a difference of at least one full point on an 18-point scale would be needed for meaningful improvement.
Lekembi works by clearing up a sticky brain protein called amyloid which is a hallmark of Alzheimer’s. But it’s not exactly clear what causes the disease. A range of other amyloid-targeting drugs have failed and many researchers now think that combination treatments will be needed.
EduHelm, a similar drug, was dogged by controversy over its effectiveness.
The FDA did not consult with the same expert panel before approving Leqembi.
Despite “less drama” surrounding the new drug, Schrag said many of the same concerns apply.
“Is this modest, measurable benefit worth the hefty price tag and side effects patients may experience?” He asked. “I have very serious doubts.”
About 13% of patients in Eisai’s study had brain swelling and 17% had small hemorrhages in the brain, side effects seen with earlier amyloid-targeting drugs. In most cases there are no symptoms of the problems, which can include dizziness and vision problems.
In addition, several Leqembi users died while taking the drug, including two who were on blood-thinning drugs. Christian has said that medicine cannot be held responsible for the death. The FDA label warns doctors to use caution if they prescribe Lekembi to patients taking blood thinners.
Insurers are likely to cover the drug only for patients with mild symptoms and those confirmed to have amyloid buildup, such as those included in the company’s study. This usually requires expensive brain scans. A different type of scan will be needed to periodically monitor brain swelling and bleeding.
A key question in the drug’s rollout will be coverage decisions by Medicare, the federal health plan that covers 60 million seniors and other Americans. Agency severely restricted coverage Aduhelm’s, essentially wiping out its US market and propelled Biogen drop marketing plans for medicine.
Eisai officials said they’ve already spent months discussing their drug’s data with Medicare officials. Coverage is not expected until the FDA confirms the drug’s benefit, possibly later this year.
“Once we have a decision from Medicare, we can actually launch the drug across the country,” said Evan Cheung, Christian’s US CEO.
Betsy Groves, 73, of Cambridge, Mass., was diagnosed with Alzheimer’s in 2021. A former lecturer at the Harvard School of Education, he noticed that he was having trouble remembering some students’ names and answering questions.
Her initial diagnosis, based on a cognitive examination, was later confirmed by a positive test for amyloid.
Groves says he’s “more than willing” to try Lekembi, despite the potential side effects and need for infusions.
“For me, the moment the drug comes on the market — and I get my doctor’s approval — I’m going to take it,” Groves said.
AP Medical Writer Lauren Neergaard contributed to this report
The Associated Press Department of Health and Science is supported by the Howard Hughes Medical Institute’s Science and Educational Media Group. AP is solely responsible for all content.
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