This Thursday, the U.S. Food and Drug Administration. ?FDA, for its acronym in English? converted Leqembi's drug “lecanemab-irmb”, as a new drug to treat Alzheimer's, to traditional approval after determining that a confirmatory trial verified clinical benefit.
Leqembi is the first amyloid beta-targeted antibody to convert from accelerated to traditional approval for the treatment of Alzheimer's disease.
The drug works by reducing amyloid plaques that form in the brain, a defining pathophysiological feature of the disease.
Leqembi was approved in January under the Accelerated Approval route. 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 an alternate endpoint—in the case of Leqembi, plaque reduction of amyloid in the brain? which is reasonably likely to predict a clinical benefit for patients.
As a post-marketing requirement for 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.
"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 at the Center for Evaluation and FDA Drug Investigation. "This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer's disease."
Alzheimer's disease is an irreversible and progressive brain disorder that affects more than 6.5 million Americans. It slowly destroys memory and thinking skills, and eventually the ability to perform simple tasks.
While the specific causes of Alzheimer's disease aren't fully understood, it is characterized by changes in the brain, including the formation of amyloid beta plaques and neurofibrillary tangles, or tau, that result in the loss of neurons and their connections, he noted. the FDA in a statement.
The randomized, double-blind, placebo-controlled study 301 enrolled 1,795 patients with Alzheimer's disease. Treatment was initiated in patients with mild cognitive impairment or mild disease-stage dementia and confirmed presence of amyloid beta pathology.
Patients were randomized in a one-to-one ratio to receive either placebo or Leqembi at a dose of 10 milligrams once every two weeks.
Thus, Leqembi demonstrated a statistically and clinically significant reduction in decline from baseline to 18 months in the primary endpoint, the Clinical Dementia Rating Scale Sum of Boxes score, compared with placebo.
On June 9, the FDA convened the Central and Peripheral Nervous System Drugs Advisory Committee to discuss whether Study 301 provided evidence of clinical benefit for Leqembi for the treatment of Alzheimer's disease. All committee members voted in the affirmative that the study results verified the clinical benefit of the drug for its indicated use.
The most common side effects of Leqembi were headache, infusion-related reactions, and imaging abnormalities related to 'ARIA' amyloid, a side effect known to occur with the class of antibodies that target amyloid.
The FDA said that Leqembi should be started in patients with mild cognitive impairment or mild dementia in the Alzheimer's disease stage, the population in which the treatment was studied in clinical trials.
However, the label states that there are no safety or efficacy data on starting treatment earlier or later in the disease than those studied.
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