The United States Food and Drug Administration (FDA) has granted Leqembi full approval to treat Alzheimer’s disease after a confirmatory trial verified clinical benefit. .“Today’s action is the first verification that a drug targetting the underlying disease process of Alzheimer’s disease has shown clinical benefit in this devastating disease,” said FDA Center for Drug Evaluation and Research Office of Neuroscience Acting Director Teresa Buracchio in a Thursday press release. .“This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer’s disease.”.The release said Leqembi is the first amyloid beta-directed antibody to be converted from an accelerated approval to a traditional one for the treatment of Alzheimer’s disease. It works by reducing amyloid plaques which form in the brain. .Leqembi was approved in January under the Accelerated Approval pathway. This pathway allows the FDA to approve drugs for serious conditions where there is an unmet medical need, based on clinical data demonstrating their effect on an endpoint. .As a requirement of Accelerated Approval, the FDA required the applicant to conduct a clinical trial to verify the anticipated benefit of Leqembi. Its efficacy was evaluated through the results of Study 301 — a Phase 3 randomized, controlled clinical trial. .The release said Study 301 was a multi-centre, randomized, double-blind, placebo-controlled, parallel group study which enrolled 1,795 Alzheimer's patients. It said treatment was initiated in patients with mild cognitive impairment or dementia stage of disease and confirmed presence of amyloid beta pathology. .Patients were randomized in a 1:1 ratio to receive placebo or Leqembi at a dose of 10 milligrams/kilograms once every two weeks. It demonstrated a significant, meaningful reduction of decline from baseline to 18 months on the primary endpoint compared to the placebo..The FDA convened the Peripheral and Central Nervous System Drugs Advisory Committee on June 9 to discuss whether Study 301 provided evidence of clinical benefit of Leqembi to treat Alzheimer’s. All committee members voted yes that the results of the study verified its clinical benefit. .The release went on to say the most common side effects of Leqembi were headache, infusion-related reactions, and amyloid-related imaging abnormalities (ARIA). ARIA most often presents as temporary swelling in areas of the brain seen on imaging studies which resolve over time and might be accompanied by small spots of bleeding in or on the surface. .Intracerebral hemorrhages can occur in patients treated with this class of medications and can be fatal. A boxed warning is included in the prescribing information to alert patients and caregivers to the potential risks associated with ARIA. .The release acknowledged Leqembi should be initiated in patients with mild cognitive impairment or mild dementia stage of Alzheimer’s disease. The labeling states there are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease. .This approval was granted to Eisai. .The Alzheimer Society of Ontario (ASO) welcomed the FDA’s decision to grant full approval to Leqembi. .“Helplessness is steadily giving way to hope,” said ASO CEO Cathy Barrick. .“While we have always been able to lower our risk of developing Alzheimer’s disease through lifestyle choices, the emergence of safe, effective treatments gives healthcare providers and families additional options to live well with Alzheimer’s disease — and for the first time ever, to slow progression of the disease itself.”.Barrick said Ontario had years of advance notice treatment options for it were coming. She said there is “no excuse not to be ready: ready with infusion capacity, diagnostic infrastructure, primary care education.”.The ASO said on June 8 it is encouraged by Health Canada’s acceptance of three biomarker tests developed by Roche Diagnostics to identify the disease. .READ MORE: Alzheimer’s group praises Health Canada approval of test for disease’s detection.“With over 275,000 Ontarians living with dementia today — and three times that number within the next 30 years — Ontario needs new and innovative options for detecting and diagnosing Alzheimer’s disease,” said Barrick. .These tests are an additional, cheaper option for healthcare providers and families to gain information and make advanced care decisions with more confidence.
