The US Food and Drug Administration declined to grant accelerated approval for the experimental Alzheimer’s drug donanemab and requested more information from the drug’s maker, Eli Lilly and Company.
The company, which announced the development on Thursday, plans to submit Phase 3 clinical trial data later this year for traditional FDA approval.
Findings from an earlier Phase 2 trial, published in May in the New England Journal of Medicine, suggested that treatment with donanemab may result in “modestly less cognitive and functional decline” in patients with early Alzheimer’s disease, but the drug has been linked to some co-morbidities. adverse events such as brain swelling or effusion, which are mostly asymptomatic.
In that trial, researchers looked at the drug’s effect on the accumulation of amyloid beta plaque and tau proteins, which are hallmarks of Alzheimer’s disease.
“We look forward to our upcoming confirmatory TRAILBLAZER-ALZ 2 Phase 3 results and FDA submission of what we always see as the most impactful next steps for patients,” said Anne White, executive vice president and president of Lilly Neuroscience at Eli Lilly.
“We expect that this study will confirm the benefit and safety profile we observed in the TRAILBLAZER-ALZ Phase 2 study, and we believe that patients and physicians will be well served by fully presenting the Phase 3 data in addition to the Phase 2 data.” treatment decisions,” he said. “We are committed to working with the FDA to ensure the fastest possible path to get this potential drug to patients in need.”
The experimental intravenous drug donanemab is aimed at slowing cognitive decline in Alzheimer’s patients, not a cure. Alzheimer’s disease is the most common type of dementia. The debilitating disease currently affects more than 6 million adults in the United States.
The Alzheimer’s Drug Discovery Foundation released a statement Thursday in response to the FDA’s decision, saying it was due to a “limited number of patients” who remained on donanemab for at least 12 months, the length of time the FDA wants to see for evaluation. drug safety.
“This drug accomplished its goal—removing amyloid plaques from the brain—and because of the trial’s innovative design, treatment was stopped for patients after biomarker PET scans confirmed the plaques were gone. In some cases, this happened in less than 12 months, so fewer patients took the drug for a full year, “said Dr. Howard Fillitt, co-founder and chief scientist of the Alzheimer’s Drug Discovery Foundation. .
“ADDF applauds the design of the TRAILBLAZER-ALZ trial, which marks a major advance in the field of Alzheimer’s and promises many more treatments to come. We look forward to reviewing phase 3 data later this year,” said Fillitt. “Amyloid-clearing drugs like these are part of the solution, but there remains a critical need to develop a new generation of drugs that target all aspects of the biology of aging.” causes disease.”
Donanemab is just one of more than 300 Alzheimer’s treatments in clinical trials, according to the Alzheimer’s Association. Earlier this month, the FDA granted accelerated approval for the Alzheimer’s disease drug lecanemab, marketed as Legembi, one of the first experimental dementia drugs that appeared to slow the progression of cognitive decline.
The Alzheimer’s Association also issued a statement Thursday in response to the FDA’s determination that more information is needed to make a decision on donanemab.
The FDA’s action “demonstrates the agency’s serious approach in reviewing individual therapies,” and the association “appreciates” the FDA’s process.
“The Donanemab Phase 2 Alzheimer’s trial showed a significant slowing of decline in memory, thinking and daily function in people living with early Alzheimer’s compared to placebo,” he said. “On behalf of our constituents, we look forward to reading the Phase 3 trial data, which is expected later this year, and to the outcome of ongoing discussions between the company and the FDA.”