The US Food and Drug Administration (FDA) has granted full approval to Leqembi, a drug meant to delay cognitive decline in patients with Alzheimer's disease.
Leqembi (lecanemab) is the first drug that slows Alzheimer's progression to be converted from accelerated to full regulatory approval. It is administered twice monthly through intravenous infusion.
The drug from Japanese manufacturer Eisai, and the U.S.-based drugmaker Biogen, works by reducing amyloid plaques that form in the brain, a defining pathophysiological feature of the disease.
"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," says Teresa Buracchio, acting director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research.
The approval was changed after evaluating the efficacy of Leqembi using the results of Study 301 (CLARITY AD), a Phase 3 randomized, controlled clinical trial.
The study enrolled 1,795 Alzheimer's patients with mild cognitive impairment or mild dementia stage of disease and confirmed the presence of amyloid beta pathology. The participants received either a placebo or Leqembi at a dose of 10mg/kg once every two weeks. The drug delayed cognitive decline by 5.3 months after 18 months of treatment.
The most common side effects of Leqembi were headache, infusion-related reactions, and amyloid-related imaging abnormalities (ARIA), that is temporary swelling in areas of the brain seen on imaging studies that resolve over time. On rare occasions, ARIA can cause symptoms such as headache, confusion, and dizziness and lead to intracerebral hemorrhages (bleeding into the brain tissue).
Therefore, the FDA issued its strongest warning label for the drug and recommended undergoing genetic testing, as people with two copies of a well-known Alzheimer's risk gene may be more at risk of ARIA.
"While we continue efforts to discover new targets and test new treatments, people living with this fatal disease deserve the opportunity to discuss and make the choice with their doctor if an FDA-approved treatment is right for them," says Joanne Pike, DrPH, Alzheimer's Association president, and CEO.
The drug will cost $28,000 a year. However, the Institute for Clinical and Economic Review analysis suggests that the appropriate cost for the drug would be between $8,900 to $21,500 annually.
The Centers for Medicare & Medicaid Services had already announced that Medicare would cover 80% of Leqembi's price once patients meet their Part B deductible.
Over 6 million Americans have Alzheimer's disease, most of whom are 65 and older. It's a progressive disease that results in patients losing the ability to carry on a conversation and respond to their environment.
- The US Food and Drug Administration. FDA Converts Novel Alzheimer’s Disease Treatment to Traditional Approval.
- Alzheimer’s Association. Alzheimer’s Association Welcomes U.S. FDA Traditional Approval of Leqembi: Full Details.
- The Institute for Clinical and Economic Review. ICER Publishes Final Evidence Report on Lecanemab for Alzheimer’s Disease.
- The Centers for Medicare & Medicaid Services. Statement: Broader Medicare Coverage of Leqembi Available Following FDA Traditional Approval.
- Alzheimer’s Association. What is Alzheimer’s Disease?