The U.S. Food and Drug Administration's (FDA) advisory panel has unanimously voted in favor of the new Alzheimer's drug, donanemab, suggesting its benefits in slowing cognitive decline outweigh the associated risks. The decision follows a comprehensive study involving 1,700 patients, revealing that those receiving monthly intravenous infusions of donanemab experienced a 35% slower decline compared to participants given a placebo. However, the trial also reported severe side effects, including three deaths linked to brain swelling or bleeding.
Donanemab, developed by Eli Lilly, targets amyloid plaques in the brain—a key factor contributing to Alzheimer's disease. The drug's efficacy varied among patients with different levels of tau protein, and the FDA is now tasked with making a final approval decision later this year. If approved, donanemab would become the second drug in the U.S. market capable of significantly slowing cognitive decline in Alzheimer's patients.
Despite the promising clinical trial results, safety concerns loom over donanemab. The deaths recorded during the study were attributed to brain swelling or bleeding, with one incident linked to a stroke—an increasingly common risk among Alzheimer's patients. This echoes concerns faced by similar drugs targeting amyloid plaques, such as Leqembi, which was approved by the FDA last year.
“I thought the evidence was very strong in the trial showing the effectiveness of the drug,” – panel member Dean Follmann, a statistician from the National Institutes of Health.
The FDA panel's discussion also highlighted the potential need for brain scans to determine patient eligibility for donanemab based on tau protein levels. However, some experts argue against this requirement due to practical and accessibility concerns.
“Imposing a requirement for tau imaging is not necessary and would raise serious practical and access concerns to the treatment,” – Dr. Thomas Montine of Stanford University.
Lilly's approach to administering donanemab differed from previous studies by ceasing treatment once amyloid plaque levels reached minimal thresholds. The company presented limited data on the optimal duration of treatment and the timeframe for restarting therapy if needed. This led to deliberations on whether the drug's benefits should apply broadly to all Alzheimer's patients or only those mirroring the study group.
Economic implications also emerged as a point of contention among experts. The high costs associated with donanemab treatment and monitoring are significant factors in the broader conversation about accessibility and healthcare expenditure.
“It’s a huge cost savings for the society, we’re talking about expensive treatment, expensive surveillance,” – Dr. Tanya Simuni of Northwestern University.
Donanemab's trial outcomes align closely with those of Leqembi, demonstrating modest yet notable improvements in cognitive function for early-stage Alzheimer's patients. The FDA's upcoming decision will determine whether donanemab joins Leqembi as a viable option for combating this debilitating disease in the U.S.
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