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An advisory panel to the U.S. Food and Drug Administration unanimously voted Friday that the Alzheimer’s drug lecanemab has “clinical benefit” for treating the disease, paving the way for full FDA approval of the drug.
A decision from the FDA is expected on July 6th.
Lecanemab, a monoclonal antibody sold under the brand name Leqembi, is one of the first dementia drugs that appear 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, although there were some safety concerns because the treatment was associated with certain serious adverse events, such as brain swelling and bleeding.
The accelerated approvals program allows for earlier approval of drugs that treat serious diseases and “meet an unmet medical need” while researchers continue to study the drug to confirm, verify and characterize its clinical utility. If these additional studies show a benefit, the FDA can grant conventional full approval for the drug. However, if the confirming studies show no benefit, the drug could be withdrawn from the market.
According to the federal Centers for Medicare and Medicaid Services, if lecanemab gets the traditional FDA approval, it will offer broader coverage — meaning Medicare patients could have greater access to the treatment. However, the insurance coverage will come with some limitations.
“If the FDA grants traditional approval, CMS stands ready to ensure that everyone with Medicare Part B who meets the criteria is covered,” CMS Administrator Chiquita Brooks-LaSure said in a statement this month.
Medicare covers the cost of approved drugs when a doctor and clinical team participate in the collection of evidence about how those drugs work in the real world, also known as a registry, CMS said. Providers can submit the evidence via a CMS-supported portal.
However, patient groups and the pharmaceutical industry have expressed concerns about using a registry, stating that it would pose a barrier to treatment.
“If Leqembi is approved for comprehensive coverage, it could mean those receiving Medicare would have the opportunity to seek treatment, which would mean a significant increase in Medicare costs and a potentially onerous path for patients to who hope to qualify for treatment,” Michael Abrams, a managing partner at global health consulting firm Numerof & Associates, said in an email on Friday. He was not involved in the FDA Advisory Committee vote, but has closely monitored the lecanemab discussions.
The committee’s endorsement of lecanemab is an “important milestone” on the road to potential full FDA approval, experts say.
“The vote of support for lecanemab is an important milestone for every patient living with Alzheimer’s disease, for every family with a loved one who has been affected by Alzheimer’s disease, and indeed for every person at risk of having 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’s health care system — said in an email on Friday. He was not present at the FDA committee meeting.
“It has been nearly two decades since the last Alzheimer’s treatment received full FDA approval, and never before has a disease-modifying drug received full FDA approval,” he wrote. “Two significant decisions will almost certainly follow today’s vote – full FDA approval and CMS’ approval to undertake the treatment in some form. This is crucial to ensure that all patients from all social and economic backgrounds have access to medicines.”
What the science says about lecanemab
On Friday, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee met to discuss the results of a confirmatory Phase 3 study of lecanemab as drugmaker Eisai seeks full approval. In the end, the members voted 6-0 for the clinical benefit of the drug.
“We will consider whether the new data impacts our current understanding of the safety of lecanemab and the benefit-risk assessment,” said Dr. Teresa Buracchio, acting director of the FDA’s Office of Neuroscience, at Friday’s meeting.
In the study, 897 participants received a placebo and 898 received lecanemab given as an intravenous infusion every two weeks. The study found that 18 months later, lecanemab slowed disease progression by at least 26% by certain measures, according to data Eisai presented to the FDA’s advisory panel on Friday.
“I found the evidence of clinical utility to be very clear and very robust,” said Dr. Merit Cudkowicz, Chief of Neurology at Massachusetts General Hospital and a member of the FDA Advisory Committee, at the meeting. Many of the other members agreed.
The data also showed that 26% of the participants given lecanemab had infusion-related reactions, compared with 7% of the participants given a placebo. Among the lecanemab recipients, 17% had a brain haemorrhage, compared with 9% in the placebo group, and 13% had brain swelling, compared with 2% who received placebo.
“These tended to occur early in treatment and supported monitoring during the first six months of treatment,” said Dr. Michael Irizarry, senior vice president and deputy chief clinical officer for Alzheimer’s disease and brain health at Eisai, told the FDA committee on Friday.
The potential for side effects could affect the drug’s coverage, Abrams said. “Despite the fact that Leqembi slowed cognitive decline by 27% in early-stage Alzheimer’s patients in the study, the treatment also carries serious risks of brain swelling and bleeding,” he said. “That could be a reason for CMS to place eligibility requirements, limit the number of patients who would qualify (at least initially), and lower the potential $5 billion cost of the program.”
In a previous study, about 6.9% of study participants given lecanemab discontinued due to adverse events, compared with 2.9% of those given placebo. Overall, serious adverse events occurred in 14% of the lecanemab group and 11.3% of the placebo group.
The most common adverse events in the lecanemab group were reactions to the intravenous infusions and abnormalities in their MRI scans, such as B. Brain swelling and bleeding, also known as amyloid-related imaging abnormalities or ARIA, which can become life-threatening. The drug’s prescribing information includes a warning about ARIA, according to the FDA.
Some people who get ARIA may not have any symptoms, but it can occasionally lead to hospitalization or permanent disability. And the frequency of ARIA appeared to be higher in people who had a gene called APOE4, which can increase the risk of Alzheimer’s or other dementias. ARIA “were numerically less common” among APOE4 non-carriers.
The FDA Advisory Committee on Friday discussed concerns about lecanemab’s overall risks versus benefits for APOE4 carriers. “The ARIA rate was quite noticeable,” said committee member Dr. Michael Gold in Friday’s session.
According to the Alzheimer’s Association, more than 6.5 million people are living with Alzheimer’s disease in the United States, and that number is expected to rise to 13.8 million by 2060.
CNN’s Tami Luhby contributed to this report.