The US Food and Drug Administration converted Lekambi (lekanemab-irmb), a drug indicated for the treatment of adult patients with Alzheimer’s disease, to a conventional approval regimen after determining that a confirmatory study supported clinical benefit. Lekambi – the first anti-amyloid beta antibody moving from accelerated approval to traditional approval for the treatment of Alzheimer’s disease. The drug works by reducing the amyloid plaques that form in the brain, which is the defining pathophysiological sign of the disease.
Lekambi was approved in January as part of the fast-track approval process. This pathway allows the FDA to approve drugs for the treatment of serious conditions where there is an unmet medical need, based on clinical evidence demonstrating the effect of the drug on an alternative endpoint (in the case of Lekambi, reduction of amyloid plaques in the brain) that is reasonably plausible. predict clinical benefit for patients. As a post-marketing requirement for accelerated approval, the FDA required the applicant to conduct a clinical study, often referred to as a confirmatory study, to confirm the expected clinical benefit of Lekambi. The efficacy of Lekambi was evaluated using the results of Study 301 (CLARITY AD), a phase 3 randomized controlled clinical trial.
“Today’s action is the first confirmation that a drug that targets the underlying disease process of Alzheimer’s has demonstrated clinical benefit in this devastating disease,” said Teresa Buracchio, acting director of the FDA’s Office of Neurology Evaluation and Evaluation Center. “This confirmatory study has confirmed that this is a safe and effective treatment for Alzheimer’s patients.”
Alzheimer’s disease is an irreversible progressive brain disease that affects more than 6.5 million Americans. The disease slowly destroys memory and thinking abilities, and eventually the ability to perform simple tasks. Although the specific causes of Alzheimer’s disease are not fully understood, it is characterized by changes in the brain, including the formation of beta-amyloid plaques and neurofibrillary tangles, or tau, which leads to the loss of neurons and their connections.
Study 301 was a multicenter, randomized, double-blind, placebo-controlled, parallel-group study that included 1,795 patients with Alzheimer’s disease. Treatment was initiated in patients with mild cognitive impairment or mild dementia at the disease stage and confirmed presence of beta-amyloid pathology. Patients were randomized in a 1:1 ratio to receive either placebo or Lekambi at a dose of 10 mg/kg once every two weeks.
Lekambi demonstrated a statistically significant and clinically significant reduction in reduction compared to placebo compared to placebo compared to placebo compared to placebo. Statistically significant differences between treatment groups were also demonstrated across all secondary endpoints, including cognitive subscale 14 of the Alzheimer’s Disease Rating Scale.