The Food and Drug Administration on Friday approved an Alzheimer’s drug shown in clinical trials to slow cognitive decline in patients in the early stages of the disease.
Experts say the approval gives patients new hope after repeated failures to find effective treatments, although there have been reports of side effects from the new drug.
The drug lecanemab, which will be marketed under the name Legembi, is a monoclonal antibody infusion given every two weeks.
The FDA has approved Legembi for use in people with mild cognitive impairment or early Alzheimer’s disease.
The drug was approved on an accelerated basis, which allows early approval for new drugs that “fill an unmet medical need.” At the same time, companies are required to conduct additional clinical trials to confirm the benefits of their drugs or risk being pulled from the market.
The agency said its decision was based on mid-stage trials of 856 patients with Alzheimer’s disease.
In late September, Eisai and Biogen, which developed the drug, said a phase 3 clinical trial of 1,795 patients found that Legembi slowed cognitive decline by 27% in people taking it after 18 months. The FDA said Friday that it expects to review the phase 3 data “soon.”
The FDA did not seek the advice of its advisory committee, which it did before controversially approving another Alzheimer’s drug made by Biogen called Aduhelm in 2021.
Legembi is not a cure; it aims to slow the progression of the disease by removing clumps of beta-amyloid – which scientists have long believed to be one of the main causes of the disease – from the brain.
Experts say the benefit is small, but anything that can give patients extra time with family and other loved ones is worth adding.
Eisai’s U.S. chairman and CEO Ivan Cheung told NBC News that a two-week supply of the drug is expected to cost about $25,000 per patient per year. Cheung said the Centers for Medicare & Medicaid Services currently limits coverage for drugs that target amyloid in the brain, so uptake of new drugs may be limited at first.
Safety concerns have been raised about the drug. At least three deaths may have been linked to the drug after patients suffered brain swelling or brain haemorrhage.
According to the company’s phase 3 trial data, about 12.6% of patients taking the drug experienced brain swelling, compared to only 1.7% of those in the placebo group. About 17% of patients taking the drug had a brain bleed, compared with 9% in the placebo group.
Cheung said the company is still “confident” the drug’s benefits outweigh its risks.
“The disease is devastating,” she said, “and comes with a lot of emotional turmoil for all the families involved.”
According to the Centers for Disease Control and Prevention, Alzheimer’s is the seventh leading cause of death in the United States. Most FDA-approved medications for Alzheimer’s are intended to help with symptoms, not to slow the progression of the disease.
Friday announcement It follows a scathing congressional report released last week detailing the approval of a different Alzheimer’s drug called Aduhelm. The drug, developed by Biogen, was approved in 2021, even though an FDA advisory committee concluded it was unlikely to work. In a report last week, investigators said the agency did not follow its own standards and that its approval of Aduhelm was “rife with irregularities.”
In response to the report, Public Citizen, an advocacy group, sent a letter urging the FDA to delay its decision on Legembi’s approval while the companies complete additional clinical trials needed for full approval.
The drug has its advocates.
Donna Wilcock, assistant dean of biomedicine at the University of Kentucky, said Legembi should be approved. The data supporting Legembi’s use are “robust,” he said, adding that the trial results were “the best I’ve seen for an Alzheimer’s drug in my 25 years.”
The Alzheimer’s Association, Eisai and Biogen said the FDA should approve Legembi for early-stage Alzheimer’s based on phase 3 clinical trial results.
The organization is urging the Centers for Medicare & Medicaid Services to reconsider its decision on the new Alzheimer’s treatment. Last year, in response to weak data supporting Aduhelm’s approval, CMS said it would not cover Alzheimer’s drugs that target amyloid unless patients are in a clinical trial.
With the drug becoming more widely available, more patients could have “more months to get to know their spouse, children and grandchildren,” said Maria Carrillo, senior research fellow at the Alzheimer’s Association.
In a statement released after the FDA’s approval on Friday, CMS suggested it may reconsider its position on coverage of some Alzheimer’s treatments.
“CMS is reviewing the available data and may revise the current coverage based on this review,” the agency said.
CORRECTION: (January 6, 4:08 pm ET): An earlier version of this article misstated the stage of clinical trial data submitted to the FDA. The agency said its decision to approve Legembi was based on phase 2 clinical trials, not phase 3.
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