In a significant development for Alzheimer’s treatment, the Food and Drug Administration’s Peripheral and Central Nervous System Drugs Advisory Committee has unanimously endorsed the approval of Eli Lilly’s new Alzheimer’s drug, donanemab. The committee’s decision, made on June 10, 2024, comes after a thorough evaluation of the drug’s efficacy and safety profile.
The advisory panel voted 11-0 in favor of donanemab, asserting that the medication demonstrates effectiveness in slowing the progression of Alzheimer’s disease during its early stages. The committee concluded that the benefits of the drug outweigh its potential risks, marking a crucial step forward in the fight against this devastating neurodegenerative disorder.
Dean Follmann, a committee member and statistician at the National Institutes of Health, emphasized the strong evidence supporting donanemab’s efficacy. “While it doesn’t stop the disease, slowing it down is very meaningful to patients,” Follmann stated, highlighting the significance of even modest improvements in managing Alzheimer’s progression.
One of the notable aspects of the committee’s recommendation is the flexibility it proposes for treatment duration. The panel advocated for allowing doctors and patients to discontinue monthly infusions if tests indicate that most amyloid plaques, a characteristic marker of Alzheimer’s, have been eliminated from the brain. Preliminary data suggests that the majority of patients could potentially cease treatment within a year.
If approved by the FDA, donanemab would join Leqembi, another Alzheimer’s drug, on the market. Leqembi, produced by Eisai in collaboration with Biogen, is currently administered every two weeks indefinitely. In contrast, donanemab’s potential for a finite treatment duration represents a significant advancement in Alzheimer’s care.
The path to this recommendation has been unusually long for donanemab. Eli Lilly initially submitted an application for ‘accelerated approval’ in January 2023, but the FDA requested additional safety data. The company then applied for traditional approval in mid-2023. The FDA’s decision to convene its advisory committee in early 2024 before making a final decision underscores the careful consideration given to this new treatment option.
During the day-long discussion, the committee heard from doctors and patients advocating for the drug’s approval. Dr. Reisa Sperling, director of the Center for Alzheimer Research and Treatment at Brigham and Women’s Hospital in Boston, stressed the importance of slowing the disease’s progression. Myra Garcia, a 65-year-old Alzheimer’s patient from Upland, California, shared her positive experience as a participant in the clinical trial for donanemab.
Despite the strong endorsement, the committee members extensively deliberated on the risks associated with the drug. Of particular concern were patients with the APOE4 gene, which significantly increases the risk of both Alzheimer’s and complications from the drug. However, Colette Johnston, the patient representative on the committee, argued for access to the drug even for higher-risk patients, emphasizing the dire need for treatment options.
Dr. Sperling concurred with this sentiment, stating, “This is a fatal disease. We need to be a little more aggressive.” The committee recommended that, if approved, doctors and patients should be provided with comprehensive educational materials detailing the drug’s risks.
Looking ahead, the committee urged researchers to investigate the long-term consequences of discontinuing treatment, including the potential re-emergence of amyloid plaques and the possible need for restarting donanemab therapy.
The FDA is expected to act on the committee’s recommendation in the coming months. This decision could potentially provide a new ray of hope for millions of Alzheimer’s patients and their families, offering a treatment option that not only slows the disease’s progression but also presents the possibility of finite treatment duration.
As the medical community awaits the FDA’s final decision, the unanimous endorsement by the advisory committee marks a significant milestone in Alzheimer’s research and treatment, potentially ushering in a new era in the management of this challenging disease.
