FDA advisory committee backs donanemab for early-stage Alzheimer’s

Key takeaways:

• The FDA panel voted 11-0 in favor of donanemab as an effective treatment for early-stage Alzheimer’s.
• The committee also voted unanimously that clinical benefits of treatment outweighed risks.

An FDA advisory panel on Monday voted in favor of donanemab as a treatment for early symptomatic Alzheimer’s disease.

In a press conference on June 10, the Peripheral and Central Nervous System Drugs Advisory Committee ruled 11-0 that donanemab (Eli Lilly & Co.), a once-per-month, 30-minute infusion treatment, was effective in treating individuals with mild cognitive impairment and mild dementia.

The committee also ruled unanimously that the benefits of the investigational therapeutic outweighed the risks, which included the life-threatening condition of amyloid related imaging abnormalities.

“I voted yes, based on the data and on the value that the best interest of the patient is the only interest to be considered,” Nilufer Ertekin-Taner, MD, PhD, a committee member and neurogeneticist at the Mayo Clinic in Jacksonville, Florida, said after the vote.

The affirmative vote came with a caution that more data is required from underserved populations as well as from those with conditions such as Down syndrome and others of autosomal dominance.

In early March, the FDA said it would convene the advisory committee meeting to further discuss safety, efficacy and dosing regimen of donanemab from the phase 3 double-blind TRAILBLAZER-ALZ 2 study, which enrolled 1,736 older adults with early symptomatic AD who presented with telltale neuropathology such as tau or amyloid plaque found by positron emission tomography. Individuals in the TRAILBLAZER-ALZ 2 trial demonstrated greater disease progression compared with participants in similar trials of other amyloid plaque-targeting therapies.

“For the group that was included (in the clinical trial), the risks can be safely clarified,” Cynthia Carlsson, MD, MS, a committee member and director of the Wisconsin Alzheimer’s Institute, said after the second round of voting.

The Alzheimer’s Drug Discovery Foundation issued its own statement regarding the FDA’s action on donanemab, stating that future approval would “expand the arsenal of available drugs needed to treat Alzheimer’s with combination therapy.”

“If approved, donanemab will expand the first class of disease-modifying drugs, serving as the building blocks for future generations of drugs,” Howard Fillit, MD, co-founder and chief science officer at the ADDF, stated in a related release. “Anti-amyloids are not a silver bullet, but they offer opportunities for patients to modify the course of the disease while the field works towards developing more novel therapies that target the underlying biology.”

Editor's Note: This story was updated on June 11, 2024 to include a quote from the Alzheimer's Drug Discovery Foundation.