Alzheimer’s disease: FDA approves lecanemab amid cost and safety concernsBMJ 2023; 380 doi: https://doi.org/10.1136/bmj.p73 (Published 11 January 2023) Cite this as: BMJ 2023;380:p73
- Elisabeth Mahase
- The BMJ
The US Food and Drug Administration (FDA) has approved lecanemab (Leqembi) for treatment of Alzheimer’s disease despite questions over whether the drug will make a difference to patients.
The agency approved the drug through its accelerated approval pathway, stating that the decision was based on phase 2 trial data that showed a reduction in amyloid β (Aβ) plaques in patients at an early stage. As such, the FDA has said that lecanemab should be initiated only in patients with mild cognitive impairment or in those in the mild dementia stage of disease.
Lecanemab, which is priced at $26 500 (£21 840; €24 640) per patient per year, binds to and eliminates Aβ aggregates that are thought to contribute to neurodegenerative processes in Alzheimer’s disease. While the phase 2 trial of 856 patients did not meet its 12 month primary endpoint (clinical change on the Alzheimer’s Disease Composite Score), researchers reported a “reduction in brain amyloid accompanied by a consistent reduction of clinical decline across several clinical and biomarker endpoints” at 18 months.1
Additionally, the phase 3 results, published in the New England Journal of Medicine in December, showed that lecanemab administered by intravenous infusion every two weeks reduced markers of amyloid and resulted in “moderately less” decline on measures of cognition and function after 18 months when compared with placebo.2
Although not officially reviewed by decision makers, the FDA said that the phase 3 results seemed to support its decision to grant accelerated approval, which allows drugs onto the market before efficacy has been proved. …