FNIH Comments on FDA Approval of First Disease-Modifying Treatment for Alzheimer’s

North Bethesda, MD, July 18, 2023—The U.S. Food and Drug Administration’s (FDA) recent traditional approval of Leqembi (lecanemab) marks a significant milestone as the first treatment shown to slow the rate of disease progression in the early and mild dementia stages of Alzheimer’s disease.

“This milestone reflects decades of Alzheimer’s research and is exciting news for patients, their caregivers, and clinicians,” said Julie Gerberding, M.D., M.P.H., CEO of the Foundation for the National Institutes of Health (FNIH). “We are proud that work at the FNIH helped to build a strong foundation for the development and validation of biomarkers, which are essential for drug approvals like Leqembi.”

Biomarkers, measurable indicators of normal or abnormal biological processes, help identify people who might be at risk of getting a disease or those who already have it but don’t show symptoms yet. Biomarkers are also important for determining whether a drug is working as intended in a clinical trial. Leqembi was shown to reduce amyloid plaques in the brain, which are biomarkers of Alzheimer’s disease, and to slow declines in memory and thinking by about 27% after 18 months of treatment.

The FNIH has been leading major public-private partnerships to tackle this complex biomedical problem by accelerating our understanding of Alzheimer’s disease and helping to expedite the development of effective treatments. The Alzheimer’s Disease Neuroimaging Initiative (ADNI), a landmark collaborative effort, was established in 2004 by the National Institutes of Health’s (NIH) National Institute on Aging (NIA) and managed by the FNIH for over two decades, with the primary objective of collecting, analyzing, and sharing comprehensive data on Alzheimer’s disease. Its impact on brain research has been monumental, resulting in the discovery and validation of multiple neuroimaging and biofluid biomarkers used in current Alzheimer’s trials and clinical care.

The FNIH continues its focus on Alzheimer’s disease through the Accelerating Medicines Partnership® (AMP®) for Alzheimer’s Disease and the Biomarkers Consortium, which is currently at an inflection point in developing a scalable blood biomarker for Alzheimer’s and other neurodegenerative diseases. This treasure trove of data has served as a vital resource for researchers worldwide, providing invaluable insights into the early detection, progression, and treatment of Alzheimer’s disease.

“While Leqembi’s approval marks decades of scientific research and discoveries, there is more work to do. We won the battle, but not the war. We need to build on this progress to ensure better, accessible biomarkers and, ultimately, more effective, precise treatments for the millions of people and families living with the burden of Alzheimer’s disease,” said Alessio Travaglia, Ph.D., Director of Translational Science, Neuroscience, FNIH.

The FNIH extends our heartfelt gratitude to the dedicated researchers, caregivers, and patients whose unwavering commitment and participation have been instrumental in achieving this significant milestone toward advancing our understanding and treatment of Alzheimer’s disease.