Leqembi demonstrated a statistically significant and clinically meaningful reduction of decline from baseline to 18 months on the primary endpoint, the Clinical Dementia Rating Scale Sum of Boxes score, compared to placebo. Patients were randomized in a 1:1 ratio to receive placebo or Leqembi at a dose of 10 milligrams (mg)/kilograms (kg), once every two weeks.
Treatment was initiated in patients with mild cognitive impairment or mild dementia stage of disease and confirmed presence of amyloid beta pathology. Study 301 was a multicenter, randomized, double-blind, placebo-controlled, parallel-group study that enrolled 1,795 patients with Alzheimer’s disease. While the specific causes of Alzheimer’s are not fully known, it is characterized by changes in the brain-including the formation of amyloid beta plaques and neurofibrillary, or tau, tangles-that result in loss of neurons and their connections. The disease slowly destroys memory and thinking skills and eventually, the ability to carry out simple tasks. “This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer’s disease.”Īlzheimer’s disease is an irreversible, progressive brain disorder affecting more than 6.5 million Americans. “Today’s action is the first verification that a drug targeting the underlying disease process of Alzheimer’s disease has shown clinical benefit in this devastating disease,” said Teresa Buracchio, acting director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. Efficacy of Leqembi was evaluated using the results of Study 301 (CLARITY AD), a Phase 3 randomized, controlled clinical trial.
As a postmarketing requirement of the accelerated approval, the FDA required the applicant to conduct a clinical trial, often referred to as a confirmatory study, to verify the anticipated clinical benefit of Leqembi. This pathway 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 endpoint-in the case of Leqembi, reducing amyloid plaques in the brain-that is reasonably likely to predict a clinical benefit to patients. Leqembi was approved in January under the Accelerated Approval pathway. The drug works by reducing amyloid plaques that form in the brain, a defining pathophysiological feature of the disease. Leqembi is the first amyloid beta-directed antibody to be converted from an accelerated approval to a traditional approval for the treatment of Alzheimer’s disease. Food and Drug Administration converted Leqembi (lecanemab-irmb), indicated to treat adult patients with Alzheimer’s Disease, to traditional approval following a determination that a confirmatory trial verified clinical benefit.
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