FDA Grants Full Approval to Game-Changing Drug for Alzheimer Disease

The US Food and Drug Administration (FDA) converted Leqembi (lecanemab-irmb), the novel drug for Alzheimer disease, to traditional approval following its determination that a clinical trial verified the benefit of the therapy. Leqembi is the first therapy proven to slow the course of Alzheimer disease and works by reducing amyloid plaques that form in the brain, a hallmark feature of the disease.

Leqembi was first approved in January under the Accelerated Approval pathway, which “allows the FDA to approve drugs for serious conditions where there is an unmet medical need, based on clinical data demonstrating the drug’s effect on a surrogate end point.” In this case, confirmatory data were demonstrated in Study 301 (Clarity AD), a phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group study comprising 1795 patients with Alzheimer disease. The population of interest had a mild cognitive impairment or mild dementia stage of disease and confirmed presence of amyloid beta pathology. Subsequently, patients were randomized in a 1:1 ratio to receive Leqembi at a dose of 10 mg/kg once every 2 weeks or placebo.

According to the findings, Leqembi exhibited a significantly and clinically meaningful reduction of decline from baseline to 18 months on the primary outcome, which was defined as Clinical Dementia Rating Scale Sum of Boxes score compared with placebo. Researchers also observed significant changes between treatment groups with respect to all secondary end points, including the Alzheimer Disease Assessment Scale-Cognitive Subscale 14 score and the Alzheimer Disease Cooperative Study-Activities of Daily Living Scale for Mild Cognitive Impairment score.

“Today’s action is the first verification that a drug targeting the underlying disease process of Alzheimer disease has shown clinical benefit in this devastating disease,” said Teresa Buracchio, acting director of the Office of Neuroscience in the FDA Center for Drug Evaluation and Research in a press release. “This confirmatory study verified that [Leqembi] is a safe and effective treatment for patients with Alzheimer disease.”