An advisory committee to the Food and Drug Administration has recommended that the federal agency approve the Alzheimer's drug donanemab. Andrew Harnick/AP Hide caption
Toggle caption Andrew Harnick/AP
Advisers to the Food and Drug Administration unanimously recommended that the agency approve the Alzheimer's disease drug donanemab.
The Peripheral and Central Nervous System Drugs Advisory Committee voted 11-0 at a meeting Monday that donanemab is effective in slowing the progression of Alzheimer's disease in the early stages and that the drug's benefits outweigh the risks.
All 11 committee members also recommended that doctors and patients be given the option to stop the monthly infusions if tests show that most of the amyloid plaque that characterizes Alzheimer's disease has been removed from the brain.
Studies suggest that most patients could potentially stop taking the Eli Lilly drug within a year.
The evidence that donanemab works is “very strong,” says Dean Folman, a statistician and member of the National Institutes of Health panel, and while donanemab can't stop the disease, it slows it down enough that “it's meaningful for patients.”
If the FDA follows the committee's recommendation, donanemab would join a similar drug sold under the brand name Leqembi that was approved by the FDA in 2023.
During the day-long discussion, committee members noted that if approved, donanemab would have distinct advantages over Lukembi: The older drug is given every two weeks indefinitely, while the newer one is given monthly and can be stopped in some patients.
Lukembi, made by Eisai in partnership with Biogen, costs more than $26,000 a year. Eli Lilly has not disclosed the cost of donanemab.
Donanemab, which targets amyloid plaques that build up in the brains of people with Alzheimer's disease, has had an unusually long approval process.
Eli Lilly applied for “accelerated approval” from the FDA in January 2023, but the FDA said it needed more safety data.
Both donanemab and Requenvi can cause swelling and bleeding in the brain. These effects usually do not cause symptoms but can be serious and have been linked to several deaths.
After initially being rejected by the FDA, Eli Lilly filed for traditional approval in mid-2023, when a decision would normally have been made by the end of the year.
Instead, the agency said it would convene an advisory committee in early 2024 before making a decision.
At its meeting on Monday, the committee heard from doctors and patients who had called for the drug's approval.
“It is critical that we do everything we can to slow the progression of this terrible, relentless disease,” said Dr. Leisa Sperling, director of the Alzheimer's Research and Treatment Center at Brigham and Women's Hospital in Boston and a former consultant to several pharmaceutical companies.
“Donanemab has been extremely helpful to me,” said Mayra Garcia, 65, an Alzheimer's patient from Upland, Calif. “I've been on the clinical trial for a couple of years now and haven't had any problems.”
The committee discussed at length the risks associated with donanemab.
Members were particularly concerned about people who have two copies of a gene called APOE4, which greatly increases their risk of developing Alzheimer's disease but also of suffering from brain hemorrhage and swelling when given donanemab.
But Colette Johnston, patient representative on the committee, said the drug should also be made available to higher-risk patients.
“There are certainly risks,” she says, “but once you've been diagnosed with Alzheimer's, there's nothing but risks.”
Sperling agreed.
“Alzheimer's disease needs to be taken seriously,” she says, “and serious illness requires aggressive treatment.”
The committee recommended that if donanemab is approved, educational materials should be made available to doctors and patients clearly explaining the drug's risks.
Members also said researchers need to learn more about the long-term effects of stopping the treatment — for example, whether amyloid plaques will come back after several years, and if so, whether doctors should give patients donanemab again.
The FDA is expected to act on the committee's recommendations in the coming months.
