The US Food and Drug Administration (FDA) has granted standard approval to Leqembi (lecanemab-irmb), a drug developed for the treatment of Alzheimer’s disease. However, concerns have been raised regarding the safety and cost of the medication.
Leqembi carries a list price of $26,500 per year in the United States.
This decision follows a confirmatory trial that demonstrated clinical benefit, making Leqembi the first amyloid beta-directed antibody to transition from accelerated approval to traditional approval for the treatment of Alzheimer’s disease.
According to the FDA, the drug works by reducing amyloid plaques in the brain, which are a defining feature of the disease. Leqembi initially received accelerated approval in January, which allows the FDA to approve drugs addressing serious conditions with unmet medical needs.
The efficacy of Leqembi was evaluated in a Phase 3 randomized clinical trial called Study 301 (CLARITY AD). The trial provided evidence of the drug’s ability to reduce amyloid plaques and indicated potential clinical benefit.
Teresa Buracchio, the acting director of the FDA’s Office of Neuroscience, stated that this action marks the first confirmation that a drug targeting the underlying disease process of Alzheimer’s has shown clinical benefit. The official added that the confirmatory study verified the drug’s safety and effectiveness for Alzheimer’s patients.
Alzheimer’s disease is a progressive brain disorder that affects millions of Americans, causing memory loss, impaired thinking skills, and difficulties with daily tasks. Although the precise causes of the disease remain uncertain, it is characterized by changes in the brain, including the formation of amyloid beta plaques and neurofibrillary tangles, which lead to the loss of neurons and their connections.
Study 301 enrolled 1,795 patients with Alzheimer’s disease, focusing on those in the mild cognitive impairment or mild dementia stage with confirmed amyloid beta pathology. Participants were randomly assigned to receive either placebo or Leqembi at a dose of 10 mg/kg every two weeks.
Leqembi demonstrated a statistically significant and clinically meaningful reduction in decline compared to placebo on the primary endpoint of the Clinical Dementia Rating Scale Sum of Boxes score over an 18-month period, according to the FDA.
On June 9, the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee reviewed the evidence from Study 301 and voted positively, affirming the clinical benefit of Leqembi for treating Alzheimer’s disease.
The FDA announced that the drug’s label will include a “boxed warning” highlighting safety concerns. The most common side effects of Leqembi include headache, infusion-related reactions, and amyloid-related imaging abnormalities (ARIA), which can result in small spots of bleeding in or on the brain’s surface. While ARIA is often asymptomatic, it can cause symptoms such as headache, confusion, dizziness, vision changes, and nausea.
Medicare, the health program for older Americans, previously did not cover the cost of Leqembi beyond clinical trials following its accelerated approval. However, Medicare has now stated that the treatment will be covered once it receives complete FDA approval, as long as healthcare providers participate in registries that gather information about the real-world effectiveness of Leqembi.
The approval of Leqembi was granted to Eisai Inc, according to the FDA’s announcement.
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