Alzheimer’s drug Leqembi backed by FDA advisers for full approval
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Independent advisers to the Food and Drug Administration unanimously agreed Friday that a new Alzheimer’s disease drug, Leqembi, benefits patients, all but ensuring the medication will be the first of its class to get full approval for the memory-robbing illness. Wp Get the full experience. Choose your plan ArrowRight The 6-0 vote confirming the clinical benefit of the medication came at the end of an all-day meeting during which the agency’s outside experts reviewed data from a late-stage trial that showed Leqembi slowed clinical and functional decline by 27 percent compared with a placebo at 18 months. The data indicated that the drug doesn’t just treat symptoms of Alzheimer’s — like other available therapies do — but also changes the course of the disease. The data was initially disclosed last fall.
The FDA, which gave the drug a bullish review in documents released Wednesday, is expected to grant Leqembi regular approval by early July. During the meeting Friday, Teresa Buracchio, acting director of the agency’s Office of Neuroscience, acknowledged an ongoing debate about whether Leqembi produces a large enough benefit for patients.
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But Buracchio made clear that the FDA believes the benefit is “clinically meaningful” because it extends a patient’s time in the earlier, less severe phase of the illness. Leqembi does not reverse damage caused by Alzheimer’s, but it slows progression of the disease by about five to six months, the data indicates.
Even as the drug moves forward, controversies about its safety and cost appear unlikely to abate. And a controversial plan by Medicare to impose restrictions on coverage is fueling fierce disagreements among politicians, medical experts and advocates for people with Alzheimer’s.
Tensions were on display during the public hearing part of Friday’s meeting of the Peripheral and Central Nervous System Drugs Advisory Committee.
Patricia Bencivenga, special projects coordinator for PharmedOut, a project at Georgetown University that criticizes pharmaceutical industry marketing practices, said Leqembi should not get full approval. “It doesn’t work,” she said, adding that the drug poses significant safety problems and could make dementia worse.
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But several other speakers said a slowdown in disease progression would give desperate patients valuable time to take part in the lives of their families and communities.
“We just want the chance for a little more time to be the people we are today, tomorrow,” said Doreen Monks, 70, who was forced to retire as a nurse practitioner in 2016 after being diagnosed with early-onset Alzheimer’s.
About 6.5 million Americans have Alzheimer’s, which is the fifth-leading cause of death for people 65 and older. Leqembi and similar therapies being developed reduce amyloid plaques in the brain, a characteristic of the disease. The medications are monoclonal antibodies — man-made proteins that can bind to specific targets in the body.
Leqembi was approved by the FDA on an accelerated basis in January based on preliminary data showing its amyloid-lowering effect. That approval required a confirmatory study verifying that the drug benefited patients by slowing the disease.
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The medication, which is made by Eisai, a Japanese pharmaceutical company, and Biogen, based in Cambridge, Mass., is administered intravenously twice a month. The price is $26,500 a year. The drug is designed for people with mild cognitive impairment or early dementia caused by Alzheimer’s.
Traditional approval from the FDA would pave the way for a broad launch of Leqembi and some form of coverage by Medicare, the federal health program for seniors and people who are disabled. The Centers for Medicare and Medicaid Services, which runs Medicare, said recently it would cover Leqembi — and other Alzheimer’s drugs that receive traditional approval — for patients who are willing to take part in data registries designed to track the real-world effects of the treatments.
Leqembi, which is also called lecanemab, has the potential to cause serious side effects, including brain swelling and bleeding; three deaths from brain hemorrhages occurred in the extended part of the trial. The FDA said the patients had a condition called cerebral amyloid angiopathy, which is the buildup of amyloid in small arteries and capillaries in the brain. The condition can be difficult to detect by imaging tests.
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But in a discussion about whether the FDA should strengthen the label for the drug, committee members said they were reluctant to urge the agency to exclude patients who might have the condition. Their opinions were more divided about whether the drug should be withheld from patients on anticoagulants, which, according to some indications, raises the risk of complications.
Some committee members said that people on blood thinners should not take the drug, but others said not enough data exists to warrant blocking the treatment for such a large swath of older people.
Under the current label for the drug, the agency warns about the potential complications and suggests increased monitoring.
Anti-amyloid drugs, which racked up failure after failure in trials before signs of potential effectiveness emerged in recent years, have roiled the Alzheimer’s field for years.
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Another antibody drug, Aduhelm, received accelerated approval from the FDA in 2021 but failed in the market amid intense skepticism about contradictory data and sharp coverage curbs imposed by Medicare. Another drug in the same group, donanemab by Eli Lilly, has produced promising results and could receive regular FDA approval later this year or early next year.
Eisai has estimated that the number of people with early Alzheimer’s who would be eligible for Leqembi would total about 100,000 three years after the drug receives traditional approval. That estimate, which some analysts say is conservative, reflects the significant time required to adopt new screening and diagnostic technologies to confirm the accumulation of amyloid beta in the brain, the company has said.
In 2022, Medicare said it would not cover any Alzheimer’s antibody drug approved by accelerated approval unless a patient is enrolled in a CMS-approved clinical trial. It has billed its recent decision to provide coverage for certain patients taking fully approved drugs — provided they participate in an ongoing registry — as a way to gather much needed information about the treatments.
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Some experts have endorsed Medicare’s stance, arguing there are outstanding questions about safety issues and efficacy that need to be addressed. But advocates such as the Alzheimer’s Association and others have denounced that plan, saying requiring registries for coverage will delay access to the treatment and discriminate against people who cannot access academic medical centers, which are best positioned to take part in data collection efforts.
The emergence of Leqembi also is fueling debate on Capitol Hill. Sen. Bernie Sanders (I-Vt.), chairman of the Senate health committee, recently urged the Biden administration to take steps to lower the cost of the drug.
“We cannot allow pharmaceutical companies to bankrupt Medicare and our federal government in the process,” Sanders said.
But Rep. Anna G. Eshoo, the ranking Democrat on the House subcommittee on health, and Rep. Nanette Barragán — both California Democrats — expressed concerns in the other direction: that the Medicare coverage plan might make it difficult for everyone who needs Leqembi to get it.
“There needs to be clarity and transparency about the standards for coverage for FDA-approved treatments for deadly diseases with unmet medical needs,” the lawmakers wrote to CMS Administrator Chiquita Brooks-LaSure. “Please do not allow CMS’s demand for additional evidence generation be a barrier to patient care.”
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Source: The Washington Post
