FDA approves Eli Lilly’s new Alzheimer’s drug donanemab

Amyloid plaque-targeting therapy to be marketed as Kisunla after slowing cognitive and functional decline by up to 35% in early-stage Alzheimer’s.

Pharma giant Eli Lilly’s has received FDA approval for its new drug, donanemab as a treatment for early symptomatic Alzheimer’s disease. The amyloid plaque-targeting drug, which will be marketed as Kisunla, is approved for use in patients with mild cognitive impairment and those in the mild dementia stage of Alzheimer’s with confirmed amyloid pathology.

Amyloid plaques, formed by the excessive buildup of amyloid protein in the brain, are linked to the memory and thinking issues associated with Alzheimer’s disease. Kisunla helps the body remove these plaques, thereby slowing the decline in abilities such as remembering new information, planning and organizing, and managing daily tasks.

In its pivotal Phase 3 TRAILBLAZER-ALZ 2 study, Kisunla demonstrated slowing cognitive and functional decline by up to 35% compared with placebo over 18 months, and reduced the risk of progressing to the next clinical stage by up to 39%.

“Kisunla demonstrated very meaningful results for people with early symptomatic Alzheimer’s disease, who urgently need effective treatment options,” said Anne White, president of Lilly Neuroscience. “We know these medicines have the greatest potential benefit when people are treated earlier in their disease, and we are working hard in partnership with others to improve detection and diagnosis.”

According to Lilly, Kisunla is the first amyloid plaque-targeting therapy that utilizes a limited-duration treatment regimen based on the removal of amyloid plaques. The company reports that nearly half of the study’s participants completed their treatment within 12 months due to its approach that involves once-monthly 30-minute infusions to reduce amyloid plaques, achieving an average reduction of 84% from baseline by the end of the study.

Kisunla’s treatment regimen aimed to reduce amyloid plaques to minimal levels, as indicated by visually negative scans using amyloid positron emission tomography (PET). Upon reaching these levels, participants could complete their treatment and switch to placebo. The study reported substantial plaque reduction: 61% at 6 months, 80% at 12 months, and 84% at 18 months.

Cost considerations for Kisunla are influenced by the duration of treatment. With each vial priced at $695.65, the total cost varies based on how quickly a patient achieves the minimal amyloid plaque levels. Lilly claims the drug’s potential to stop treatment after a limited-duration course, along with once-monthly 30-minute infusions, can result in lower out-of-pocket costs and fewer infusions compared with other therapies.

“This approval marks another step forward in evolving the standard of care for people living with Alzheimer’s disease that will ultimately include an arsenal of novel treatments, providing much needed hope to the Alzheimer’s community,” said Dr Howard Fillit, Chief Science Officer at the Alzheimer’s Drug Discovery Foundation. “As a physician, I am encouraged by the potential to stop treatment, which could reduce out-of-pocket costs and infusion burden for eligible patients. Diagnosing and treating Alzheimer’s sooner than we do today has the potential to meaningfully slow disease progression, giving patients invaluable time to maintain their independence for longer.”

Despite its benefits, Kisunla can cause amyloid-related imaging abnormalities (ARIA), a side effect common to amyloid plaque-targeting therapies. ARIA, often detected via MRI, typically presents as temporary brain swelling or small brain bleeding spots. While serious and life-threatening ARIA events are rare, Lilly says that Kisunla can also lead to allergic reactions and headaches, particularly during or shortly after infusions.

Lilly is continuing to investigate donanemab in various clinical trials, including TRAILBLAZER-ALZ 3, focusing on preventing symptomatic Alzheimer’s in preclinical patients, TRAILBLAZER-ALZ 5, a registration trial in China and Korea, and TRAILBLAZER-ALZ 6, which aims to expand understanding of ARIA using novel MRI sequences and blood-based biomarkers.

Photograph: iLexx/Envato, Eli Lilly