2023-06-10 12:27:35
A panel of expert advisors from the US Food and Drug Administration (FDA) unanimously agreed last Friday to approve the drug lecanemab for the treatment of those patients who are in an early stage of Alzheimer’s disease. The six panel advisers voted in favor of this drug, which delays cognitive decline caused by dementia by 27%, they stressed after a long session.
The decision represents an enormous boost for the final approval of this drug, which is the first in decades that has shown some effect, albeit modest, against the progression of a neurological disease that affects 50 million people worldwide.
Regular approval from the Food and Drug Administration (a decision expected by July 6) is likely to pay for the treatment by Medicare (the federal health insurance program for people age 65 and older). That approval would also make lecanemab the first disease-modifying drug to achieve the regulatory milestone. Current treatments only address the symptoms, but do not change the course of the disease, which affects 6 million Americans, according to the American Alzheimer’s Association.
The drug being developed by the pharmaceutical companies Biogen and Eisai presented, in November 2022, the results of the treatment and obtained accelerated approval from the FDA in January based on its ability to eliminate sticky amyloid plaques from the brain, not without controversy. The panel on Friday considered Eisai’s trial designed to show that the drug benefited patients.
serious side effects
That study, published in November, showed that the drug slowed cognitive decline by 27% in patients with early Alzheimer’s, but it was also associated with some serious side effects for some patients, including brain swelling and bleeding or microbleeds.
“I think the benefit versus risk is beneficial, acceptable, and commensurate with this class of therapy, especially given the burden of disease and the progressive nature of the disease,” said panel member Tanya Simuni, MD, Professor of Neurology at Northwestern University.
“Overall, it clearly demonstrated that this is an effective treatment in the population as defined,” said Dr. Robert Alexander, chairman of the committee and an Alzheimer’s expert at the Banner Institute. He added that he thought the study “clearly demonstrated clinical benefit,” and he called the results “robust.”
The FDA has also asked the expert panel to evaluate the drug’s side effects in certain patient populations. These included patients taking drugs to prevent blood clots, those who have a genetic variant called APOE4 that increases the risk of Alzheimer’s, and those with a rare condition called cerebral amyloid angiopathy (CAA), in which the amyloid protein targeted by the drug accumulates in the walls of the arteries of the brain and can cause bleeding.
Panel members concluded that, overall, the risks for those patients with two copies of the APOE4 gene were balanced by the benefits of the drug. The committee has recommended genetic testing of patients before they take the drug to determine if they have this mutation.
Some FDA advisers on the panel raised concerns about giving lecanemab to patients taking blood thinners. Others argued that patients should be given the choice as long as they are aware of the risks. The panel also noted that it would not recommend excluding patients with CAA, which can be difficult to diagnose, from taking the drug. He suggested limiting the use of lecanemab in patients with the most severe cases of the condition, called cerebral amyloid angiopathy-related inflammation, because of the increased risk of brain bleeding.
“We agree that lecanemab provides a clinical benefit and that this benefit outweighs the risks”
Joanne Pike
Executive Director of the American Alzheimer’s Association
The federal agency that administers Medicare has said it will pay for treatment if doctors participate in a health agency database, but has not yet released details of its plan. In the United States, forecasts are already being made if the drug is approved, and sales of lecanemab are estimated to exceed $1 billion by 2026 and reach $5.7 billion by 2030. The executive director of the Association of Alzheimer’s, Joanne Pike, urged in a statement to approve and expand access for patients “without barriers”.
“We fully agree with the FDA Advisory Committee that lecanemab provides clinical benefit and that this benefit outweighs the risks,” Pike noted.
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