Lecanemab Receives Full FDA Approval as First Alzheimer’s Treatment Targeting Disease Process

The FDA has announced the full approval of Lecanemab (Leqembi) as the first Alzheimer’s treatment that specifically targets the disease process. Teresa Buracchio, MD, acting director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, describes this development as a significant milestone in the treatment of Alzheimer’s disease. The approval expands the drug’s Medicare coverage, providing more patients with access to this potentially effective treatment.

A Long Journey

Babak Tousi, MD, from the Cleveland Clinic, reflects on the long road to achieving this milestone in Alzheimer’s treatment. While the benefits of Lecanemab may be modest, Tousi emphasizes that they are still noteworthy. The drug has shown the potential to slow down the progression of the disease, offering a glimmer of hope for Alzheimer’s patients. Additionally, the journey towards developing Lecanemab has led to a deeper understanding of the disease, improved diagnostic accuracy, and a better understanding of disease progression and biomarkers.

Accelerated Approval and Confirmatory Study

In January, Lecanemab received accelerated approval to treat Alzheimer’s disease. However, as part of the accelerated approval pathway, drug manufacturers Eisai and Biogen were required to conduct a postmarketing trial to verify the drug’s clinical benefit. This confirmatory study, known as the phase III CLARITY AD trial, demonstrated that Lecanemab resulted in less cognitive and functional decline in early-stage Alzheimer’s disease. Despite these positive outcomes, adverse events were associated with the drug.

Risks and Safety Concerns

Adverse events observed during the CLARITY AD trial included amyloid-related imaging abnormalities (ARIA) with edema or effusions (ARIA-E) and ARIA with hemosiderin deposition (ARIA-H). A total of 13% of patients who received Lecanemab experienced ARIA-E, while 17% experienced ARIA-H. Three subgroups of patients, namely APOE4 homozygotes, individuals with underlying cerebral amyloid angiopathy, and those who require concomitant treatment with anticoagulant agents, appeared to have a higher risk of adverse events. The prescribing information for Lecanemab includes a boxed warning to inform patients and caregivers about the potential risks associated with ARIA. It also recommends testing for APOE4 status before initiating treatment and exercising caution in patients taking anticoagulants or those with other risk factors for intracerebral hemorrhage.

Positive Expert Opinions

The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee recently voted 6-0 in favor of Lecanemab’s clinical benefit in early Alzheimer’s disease. Merit Cudkowicz, MD, a member of the advisory committee from Harvard Medical School, commended the robustness of the CLARITY AD findings, highlighting the positive outcomes on primary and key secondary measures. Cudkowicz also noted the early onset of the drug’s effect, which appeared within six months and seemed to intensify over time.

The full FDA approval of Lecanemab marks a significant milestone in the treatment of Alzheimer’s disease. This groundbreaking drug targets the underlying disease process and has shown clinical benefit in slowing down cognitive and functional decline. While there are safety concerns, the approval provides hope for Alzheimer’s patients and signifies a deeper understanding of the disease.