An independent panel advising the U.S. Food and Drug Administration (FDA) has unanimously agreed that the benefits of Eli Lilly’s experimental Alzheimer’s treatment, donanemab, outweigh its risks. This decision marks a crucial step toward the FDA’s final verdict on the drug, which is highly anticipated following divergent trial designs.
Key Support from FDA Panel
The panel’s unanimous vote clears a significant hurdle for donanemab, indicating that the trial data effectively demonstrate the drug’s benefits for patients in the early stages of Alzheimer’s. Dawn Brooks, Eli Lilly’s development leader for donanemab, expressed satisfaction with the panel’s recognition of the drug’s positive benefit-risk profile, stating that the company now looks forward to the FDA’s final review.
Unique Trial Design and Efficacy
The FDA had convened this meeting to address unique aspects of Lilly’s trial design, which differed from those of other treatments like Eisai and Biogen’s Leqembi, a drug approved after a similar advisory committee review. Both donanemab and Leqembi target toxic beta-amyloid plaques in the brains of Alzheimer’s patients, aiming to slow disease progression. However, Lilly’s trial design included an innovative approach by measuring tau protein levels—associated with brain cell death—to select patients more likely to benefit from the treatment during the 76-week trial period. This distinct methodology led to the exclusion of patients with very low or no tau levels from the pivotal trial.
Panel’s Insights and Recommendations
The panelists acknowledged the suggestive data from the tau-based selection, with some recommending that tau testing should not be a prerequisite for treatment due to its limited availability, which could hinder access for rural and underserved populations. Additionally, the panel highlighted the need for educating doctors on the elevated risks for patients with two copies of the APOE4 gene, which is linked to a higher risk of Alzheimer’s.
Safety Concerns and Comparison to Leqembi
In Lilly’s extensive clinical trial, donanemab, administered via monthly infusions, demonstrated a 29% slowdown in the progression of memory and cognitive issues, closely aligning with the 27% reduction observed in Leqembi’s trials. However, donanemab was associated with notable safety concerns, including brain swelling and bleeding, occurring in 24% and 31% of patients, respectively, with three reported deaths. In comparison, Eisai and Biogen’s Leqembi showed brain swelling in 12.6% of participants and brain bleeding in 17.3%. Leqembi carries the FDA’s strongest “boxed” warning about these risks, which applies to all drugs in this category.
Innovative Trial Design
Several panel members praised Lilly’s innovative trial design, which allowed participants to discontinue treatment once brain imaging confirmed the clearance of amyloid plaques. However, they noted this approach might complicate treatment decisions for physicians regarding when to stop and potentially restart the drug.
Future Trials and Market Potential
Lilly is planning further trials to evaluate donanemab in genetically predisposed patients, including those with Down syndrome. Analysts like Michael Yee from Jefferies anticipate that the panel’s positive vote will likely lead to FDA approval, potentially introducing two competing drugs in the market, which could benefit patients in the long run.
Market Reaction
Following the panel’s vote, shares of Eli Lilly rose by 1.8%, closing at $865. This development highlights the market’s optimism regarding donanemab’s potential approval and its implications for Alzheimer’s treatment.
Conclusion
The unanimous support from the FDA advisory panel for Eli Lilly’s donanemab signifies a significant advancement in Alzheimer’s therapeutics. The innovative trial design and promising efficacy of donanemab offer hope for patients in the early stages of Alzheimer’s, despite the accompanying safety concerns. As the FDA moves toward its final decision, the medical community and patients alike are hopeful for a new and effective treatment option.
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Source: Biz News