FDA advisers vote that lecanemab shows benefit as an Alzheimer’s treatment
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An advisory panel for the US Food and Drug Administration voted unanimously Friday that the Alzheimer’s drug lecanemab shows “clinical benefit” for the treatment of the disease, paving the way for the medication to be considered for full FDA approval.
A decision from the FDA is expected by July 6.
Lecanemab, a monoclonal antibody sold under the brand name Leqembi, is one of the first dementia drugs that appears to slow the progression of cognitive decline. The drug is not a cure but works by binding to amyloid beta, a hallmark of Alzheimer’s disease.
In January, the FDA granted accelerated approval of Leqembi for people with mild cognitive impairment or mild dementia, even though there were some safety concerns due to the treatment’s association with certain serious adverse events, including brain swelling and bleeding.
The accelerated approval program allows for earlier approval of medications that treat serious conditions and “fill an unmet medical need” while researchers continue studying the drug to confirm, verify and describe its clinical benefit. If those additional studies show a benefit, the FDA can grant traditional full approval for the drug. But if the confirmatory studies do not show benefits, the drug could be taken off the market.
If lecanemab receives traditional FDA approval, the federal Centers for Medicare and Medicaid Services has said it will provide broader coverage – meaning Medicare patients could get greater access to the treatment. However, the coverage will come with some qualifications.
“If the FDA grants traditional approval CMS is prepared to ensure anyone with Medicare Part B who meets the criteria is covered,” CMS Administrator Chiquita Brooks-LaSure said in a statement this month.
Medicare will cover the approved drugs when a physician and clinical team participate in the collection of evidence about how these drugs work in the real world, also known as a registry, CMS said. Providers will be able to submit the evidence through a CMS-facilitated portal.
Patient groups and the pharmaceutical industry, however, have voiced concerns about the use of a registry, saying it will create a barrier to treatment.
“If Leqembi is given approval for wide-scale coverage, it could mean that those on Medicare would have the option of pursuing treatment, which would mean a significant spike in Medicare costs and a potentially arduous path for patients who would hope to qualify for treatment,” Michael Abrams, managing partner of the global health care consultancy firm Numerof & Associates, said in an email Friday. He was not involved in the FDA advisory committee’s vote but has been watching discussions around lecanemab closely.
The committee’s support for lecanemab serves as an “important milestone” on its pursuit for possible full FDA approval, experts say.
“The support vote for Lecanemab is an important milestone for every patient living with Alzheimer’s disease, every family with a loved one who is affected by Alzheimer’s disease, and indeed every person at-risk for developing Alzheimer’s disease in the future,” Dr. James Galvin, chief of cognitive neurology and director of the Comprehensive Center for Brain Health at UHealth - The University of Miami Health System, said in an email Friday. He was not involved in the FDA committee’s meeting.
“It has been nearly two decades since the last Alzheimer’s treatment received full FDA approval, and never before has a disease-modifying medication received full FDA approval,” he wrote. “This vote today almost assuredly will be followed by two momentous decisions – full FDA approval and agreement by CMS to cover treatment in some form. This is a critical issue to assure that all patients from all social and economic backgrounds have access to medication.”
What the science says about lecanemab
On Friday, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee met to discuss the results from a confirmatory Phase 3 study on lecanemab as drugmaker Eisai seeks full approval. At the end, members voted 6-0 in favor of the drug’s clinical benefit.
“We’ll consider whether any of the new data impacts our current understanding of the safety of lecanemab and the benefit-risk assessment,” Dr. Teresa Buracchio, acting director of the FDA’s Office of Neuroscience, said in Friday’s meeting.
In the study, 897 participants were given a placebo and 898 were given lecanemab, administered biweekly as an intravenous infusion. The study found that, 18 months later, lecanemab slowed disease progression by at least 26% in certain measurements, according to data that Eisai presented to the FDA advisory panel Friday.
“I thought the evidence for clinical benefit was very clear, very robust,” Dr. Merit Cudkowicz, chief of neurology at Massachusetts General Hospital and a member of the FDA advisory committee, said in the meeting. Many of the other members agreed.
The data also showed that 26% of participants given lecanemab had infusion-related reactions, compared with 7% of those given a placebo. Among lecanemab recipients, 17% had brain bleeding, compared with 9% in the placebo group, and 13% had brain swelling, compared with 2% given a placebo.
“These tended to occur early in treatment, supporting monitoring during the first six months of treatment,” Dr. Michael Irizarry, senior vice president and deputy chief clinical officer of Alzheimer’s disease and brain health at Eisai, told the FDA committee Friday.
The potential for side effects may affect the drug’s coverage, Abrams said. “Despite the fact that Leqembi slowed cognitive decline in early Alzheimer’s patients by 27% in the trial, the treatment also carries serious risks of brain swelling and bleeding,” he said. “That could be reason for CMS to impose prerequisites on its use, limiting the number of patients who would qualify (at least initially) and mitigating the potential $5B cost to the program.”
In a previous study, about 6.9% of the trial participants given lecanemab dropped out due to adverse events, compared with 2.9% of those given a placebo. Overall, there were serious adverse events in 14% of the lecanemab group and 11.3% of the placebo group.
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The most common adverse events in the lecanemab group were reactions to the intravenous infusions and abnormalities on their MRIs, such as brain swelling and bleeding, also known as amyloid-related imaging abnormalities, or ARIA, which can become life-threatening. The drug’s prescribing information carries a warning about ARIA, the FDA says.
Some people who get ARIA may not have symptoms, but it can occasionally lead to hospitalization or lasting impairment. And the frequency of ARIA appeared to be higher in people who had a gene called APOE4, which can raise the risk of Alzheimer’s disease or other dementias. ARIA “were numerically less common” among APOE4 noncarriers.
The FDA advisory committee on Friday discussed concerns about lecanemab’s overall risks versus benefits for APOE4 carriers. “The ARIA rate was pretty striking,” said committee member Dr. Michael Gold in Friday’s meeting.
More than 6.5 million people in the United States live with Alzheimer’s disease, according to the Alzheimer’s Association, and that number is expected to grow to 13.8 million by 2060.
CNN’s Tami Luhby contributed to this report.
Source: CNN
