FDA Accelerates Approval for New Alzheimer Treatment
The FDA has granted accelerated approval for lecanemab-irmb (Leqembi) for the treatment of Alzheimer disease.1
An irreversible disease that affects more than 6.5 million individuals in the United States, Alzheimer disease slowly destroys memory and brain function. The newly approved medication is an important advancement as it targets the fundamental pathophysiology of the disease.
“Alzheimer’s disease immeasurably incapacitates the lives of those who suffer from it and has devastating effects on their loved ones,” said Billy Dunn, MD, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, in a press release. “This treatment option is the latest therapy to target and affect the underlying disease process of Alzheimer’s, instead of only treating the symptoms of the disease.”
The approval follows the results of a Phase 3 randomized, controlled clinical trial, along with the FDA’s use of the Accelerated Approval pathway—an FDA regulation that allows drugs for serious conditions to be approved based on a surrogate endpoint.2
Lecanemab’s efficacy was evaluated in a double-blind, placebo-controlled, parallel-group, dose-finding study, which included 856 patients with Alzheimer disease. The researchers initiated treatment in patients with either mild cognitive impairment or mild dementia stages of the disease and a presence of amyloid beta pathology—a naturally occurring protein that forms plaque and disrupts cell function. Individuals who received a 10 mg dose of treatment every 2 weeks showed a statistically significant reduction in brain amyloid plaque from baseline to week 79 when compared with those who received placebo.
Prescribing lecanemab comes with a warning for amyloid-related imaging abnormalities (ARIA) that does not usually show any symptoms. Still, ARIA may be serious and life-threatening. ARIA is rare and commonly presents as temporary swelling in the brain that usually resolves over time. Further, flu-like symptoms may occur from treatment including nausea, vomiting, and changes in blood pressure.
- FDA grants accelerated approval for Alzheimer’s disease treatment. News release. US Food and Drug Administration; January 6, 2023. Accessed January 10, 2023. https://www.fda.gov/news-events/press-announcements/fda-grants-accelerated-approval-alzheimers-disease-treatment
- Accelerated Approval. US Food and Drug Administration. Published on January 4, 2018. Accessed January 10, 2023. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/accelerated-approval