Jan. 6, 2023 – The FDA has approved a new drug for the treatment of early-stage Alzheimer’s disease even though clinical trial results showed modest cognitive benefits.
The drug, lecanemab, brand name Leqembi, “is the latest therapy to target and affect the underlying disease process of Alzheimer's, instead of only treating the symptoms of the disease," Billy Dunn, MD, director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research, said in a press release.
Like its controversial cousin aducanumab lecanemab was approved under the FDA's accelerated approval pathway, which can be used to fast-track a drug that provides a meaningful therapeutic advantage over existing treatments for a serious or life-threatening illness.
Modest Benefit, Adverse Events
The drug’s label will note that “treatment with Leqembi should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was studied in clinical trials."
The FDA approved the treatment based on clinical trials that found modest cognitive benefit in patients with early Alzheimer’s disease -- but at a cost of increased risk for bleeding in the brain.
After 18 months of treatment in the clinical trial, lecanemab slowed cognitive decline by 27% compared with placebo.
While the results are "welcome news," the difference might not be clinically meaningful, a recent editorial in the journal TheLancet cautioned.
Also, a newly published case report in the New England Journal of Medicine describes a patient with Alzheimer’s who died after taking lecanemab.
Alzheimer's Association Reaction
Still, in anticipation of accelerated approval of lecanemab and the anti-amyloid drug donanemab, which the FDA has also fast-tracked, the Alzheimer's Association filed a formal request last month with the Centers for Medicare & Medicaid Services to provide full and unrestricted coverage for FDA-approved Alzheimer’s treatments.
"Each day matters when it comes to slowing the progression of this disease," Joanne Pike, a doctor of public health and president and CEO for the Alzheimer's Association, said in a news release at the time.
"The current CMS policy to severely limit access to these treatments eliminates people's options, is resulting in continued irreversible disease progression, and contributes to greater health inequities. That's not acceptable," Pike added.
After news of today's approval was released, Pike said in a new release that the association “welcomes and celebrates this action by the FDA. We now have a second approved treatment that changes the course of Alzheimer’s disease in a meaningful way for people in the early stages of the disease."
Maria C. Carrillo, PhD, chief science officer at the Alzheimer's Association called today's approval "a milestone achievement."
"The progress we’ve seen in not only this class of treatments, but also in the diversification of treatment types and targets over the past few years, is exciting and provides real hope to those impacted by this devastating disease," Carrillo said.
Critical Issues
Alvaro Pascual-Leone, MD, PhD, professor of neurology at Harvard Medical School said FDA approval of lecanemab and its adoption in the clinic represents a "very exciting development and prospect; but arguably some critical issues need to be considered."
The health care system, he says, "is not currently prepared to cope with the challenges and demands of lecanemab," as well as future drugs.
"First, we need better workflows to identify suitable patients who can most benefit from this treatment," he says.