US regulator expands access to Alzheimer's drug lecanemab

The U.S. Food and Drug Administration (FDA) has granted full approval to the drug lecanemab (known commercially as Leqembi) for people in the U.S. with confirmed amyloid positivity and a diagnosis of mild cognitive impairment (MCI) or early-stage Alzheimer's disease.

The ruling comes after the FDA's advisory panel voted unanimously in early June that the drug shows "clinical benefit" for the treatment of the disease.

With U.K. regulators also scrutinizing data on the drug, the positive U.S. ruling should serve as a "wake-up call" to this country's policymakers, said Alzheimer's Research UK's Chief Executive, Hilary Evans. A U.K. decision is expected later this year or early 2024.

Lecanemab, made by pharmaceutical company Eisai, is an antibody-based treatment designed to remove amyloid, one of the hallmark proteins that builds up in the brains of people with Alzheimer's. The drug is given as an infusion into a person's arm every two weeks.

The FDA's recommendation is based on data from Eisai's CLARITY AD Phase 3 clinical trial, which involved 1,800 patients and was published in November 2022.

The FDA review concluded that the trial showed the drug's 27% slowing of cognitive decline verified "clinical benefit" to patients.

"Today's action is the first verification that a drug targeting the underlying disease process of Alzheimer's disease has shown clinical benefit in this devastating disease," said Teresa Buracchio, acting Director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research. "This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer's disease."

Lecanemab had previously been available under the FDA's more limited "accelerated" approval program in January. This had only enabled US residents with appropriate medical insurance, or those who could afford to pay for it directly, to access the drug. Today's ruling is expected to greatly expand access to lecanemab, as it enables the Centers for Medicare and Medicaid Services (CMS) to provide wider insurance coverage under the US government's Medicare plan, which covers older people with limited income and resources.

In a statement last month, the CMS said full FDA approval would allow it to cover lecanemab, provided that patients agreed to enroll in a national registry that tracks the drug's safety and effectiveness in the real world. But some have argued this may make it harder for some to access the treatment.

The FDA's prescribing information for lecanemab includes a warning for clinicians to monitor for side-effects known as amyloid-related imaging abnormalities (ARIA) as well as infusion related reactions.

From a U.K. perspective, the FDA ruling is "welcome news" according to Alzheimer's Research UK's Chief Executive, Hilary Evans, who called for urgent action on this side of the Atlantic. "This welcome news from the US signals a step towards a world where Alzheimer's disease is treatable, but should also serve as a wake-up call."

"Today's ruling involved careful review of data from a rigorous study of 1,800 patients. A similar process is currently underway in the U.K., led by the Medicines and Healthcare products Regulatory Agency (MHRA). We've written to the MHRA urging them to complete this review as a matter of urgency, without compromising on quality."

Evans called for prompt action to stop people with Alzheimer's in the U.K. being left in limbo, and highlighted wider benefits of a quick decision. "We believe that a rapid regulatory decision on lecanemab will further strengthen the U.K.'s international reputation as a leader in dementia, attract even greater commercial investment in U.K. clinical trials, and ultimately benefit the one in two of us who will either develop dementia in the future, care for someone who has it, or both."

She also called on healthcare leaders to ensure the NHS is ready for new treatments. "Today's news underlines the urgency of getting our own NHS ready for new medicines like lecanemab."

This, Evans said, must include an "overhaul" of the way people with Alzheimer's disease are diagnosed.

"People won't be able to access these drugs without a confirmed diagnosis of Alzheimer's, but this is primarily done using a PET scan, and the U.K. has one of the lowest numbers of scanners per capita in the developed world," she said. "We're concerned that without urgent action, this would significantly restrict access and create inequities in care should lecanemab become available in the U.K."

Looking further ahead, Evans pointed to new diagnostics are on the horizon—particularly blood tests for Alzheimer's that are being developed. "We're supporting research to hasten their use in clinical practice, so lecanemab's potential arrival should act as a warning to accelerate work on this front, and help ensure people who could benefit from these new medicines are identified quickly."

Progress for people with Alzheimer's disease is long overdue, said Evans, and although drugs like lecanemab are a good first step, more effective therapies will be needed to make real inroads against diseases like Alzheimer's.

"As a first-generation treatment, lecanemab's effects are modest, and it comes with side effects that can be serious. Science can, and must, do better—both in terms of delivering drugs that are even more effective, with fewer side effects, and also treatments for people with later stage Alzheimer's and those with other forms of dementia," she said.

"At Alzheimer's Research UK, we will work tirelessly to help bring about a world free from the heartbreak of dementia, and for a cure for the diseases that cause it."

Provided by Alzheimer's Research UK