ADDF Statement On FDA’s Traditional Approval of Lecanemab (Leqembi)

The FDA’s traditional approval of Leqembi marks the first-ever approval of a disease-modifying therapy for Alzheimer’s, signifying the dawn of a new era in Alzheimer’s treatment

With clarity on the modest clinical benefit of anti-amyloids, the time is now for the Alzheimer's field to broaden its focus to develop new therapies centered around the biology of aging

July 6, 2023—Today’s FDA approval of the monoclonal antibody lecanemab (Leqembi) for the treatment of patients with mild cognitive impairment or early stage Alzheimer’s disease and confirmed amyloid plaques in the brain is a significant milestone. This marks the first time a disease-modifying treatment for Alzheimer’s has received traditional approval, and the first time that any Alzheimer's treatment has been converted from accelerated to full traditional approval by the FDA.

"This is encouraging news, and more importantly, the approval of Leqembi will serve as a catalyst for driving further developments and investments in the Alzheimer’s pipeline,” says Howard Fillit, MD, Co-Founder and Chief Science Officer of the Alzheimer’s Drug Discovery Foundation (ADDF). “We finally have clarity around amyloid’s modest effect on cognitive decline. Now, it is more important than ever to double down and widen our focus to developing the next generation of drugs based on the biology of aging that can lead to a combination therapy and precision medicine approach.”

The FDA also provided a warning on the drug’s label for patients and caregivers on the potential risks associated with amyloid-related imaging abnormalities (ARIA).

“Biomarkers are essential in drug development, identifying the optimal patient population for new therapies, allowing us to match the right drugs to the right patients at the right time,” notes Dr. Fillit. “As with other diseases of chronic aging, a precision prevention approach that tailors lifestyle interventions and therapeutics based on a patient’s individual biomarker profile will enable us to not only slow disease progression, but eventually to stop Alzheimer’s in its tracks.”

This milestone further underscores the importance of novel biomarkers and diagnostic tools, which will not only help determine which patients should receive the anti-amyloids, but will lay the groundwork to develop the next class of drugs. Biomarkers such as the Amyvid® PET scan—which received early seed funding from the ADDF—are already being used to enroll patients and demonstrate target engagement in clinical trials for amyloid-targeting drugs like Leqembi. When combined with an early and accurate diagnosis, these biomarkers allow clinicians to better understand each patient’s unique pathologies and optimize treatment accordingly.

Combination therapy and precision medicine remain the most promising path for Alzheimer’s treatment. Anti-amyloid drugs are an important starting point, but they only address one of several pathways associated with the disease. The current Alzheimer’s pipeline is robust and diverse, with over 75% of drugs exploring novel targets—including drugs that target misfolded proteins, inflammation, metabolic disturbances, vascular problems, and other pathways that contribute to the onset of Alzheimer’s. Some ADDF-funded companies that are showing promise in research on novel pathways include Therini Bio for its research around fibrin-driven inflammation and NeuroTherapia for its research around neuroinflammation.

“While we celebrate the FDA approval of Leqembi, we acknowledge that the journey towards effective treatments for Alzheimer’s is still in its early days,” adds Dr. Fillit. “As we continue our relentless pursuit of effective treatments, accessible diagnostics, and new prevention approaches, we will continue to build upon the momentum gained from Leqembi’s approval to ensure we bring new drugs to market that target the many underlying causes of Alzheimer’s.”