June 9, 2021 – The FDA’s Monday approval of a new drug to treat Alzheimer’s disease has met with both praise and criticism. As the first Alzheimer’s disease drug approved since 2003, advocates are applauding the decision. Critics say the approval was based on insufficient evidence that the drug works and balks at a price of $ 56,000 per year.
Here’s what else you need to know.
How the drug works
The new drug, aducanumab (Aduhelm), is a monoclonal antibody that reduces the buildup of amyloid plaques in the brain. These plaques, along with the tangles known as tau and other changes in the brain, cause memory loss and eventually the inability to perform simple tasks like dressing. .
The drug is administered intravenously once a month.
According to Biogen, the annual price of herbal teas is around $ 56,000. People probably won’t pay that amount, says Lon Schneider, MD, director of the California Alzheimer Disease Center. On Tuesday morning, Biogen promised multiple ways to help patients access the drug. The company will provide service coordinators to provide one-on-one support, he says.
Details of how Medicare and insurance plans will cover the drug will take some time, Schneider says. But he says even people without insurance will likely be able to negotiate the price down.
A spokesperson for the Centers for Medicare and Medicaid Services said, “CMS is reviewing the FDA decision on aducanumab and will have more information soon. “
The best patients? Advice for families
The new drug is certainly not intended for all people with Alzheimer’s disease, says Julia Biernot, MD, behavioral neurologist at the University of Maryland School of Medicine in Baltimore. “It’s important to know that it will most likely be indicated in patients with mild Alzheimer’s disease or mild cognitive impairment, as opposed to more advanced disease. And there may be potential side effects that need to be addressed. discussed with patients and their families. “
The most common, according to Biogen, is a condition known as ARIA – amyloid-related imaging abnormalities, found in 41% of patients in one study. These problems include temporary swelling of the brain and small areas of bleeding.
While she calls the approval an “overall exciting development,” Biernot also warns families that treatment would likely be needed indefinitely.
“There is no profile for the best patient,” says Schneider. Clinical studies recruited people with mild cognitive impairment or MCI, a precursor to Alzheimer’s disease, and mild Alzheimer’s disease. “This is the group in which the drug has been tested,” he says. “Not necessarily tested and proven effective, but tested.”
“I am thinking of people with symptoms, with an MCI due to Alzheimer’s disease or with mild Alzheimer’s disease, who want to try the monthly infusions for a year and a half. [the schedule used in the trials], we would be happy to support them, ”says Schneider. He stresses, however, “Families should do their own research and talk to their doctor.
Fast Track FDA Approval
The FDA has granted fast-track approval based on clinical trials that have shown reduction of plaque in the brain in people receiving the drug compared to those in the control group or the placebo group. The approval came after the FDA evaluated three separate studies of the drug involving nearly 3,500 patients.
The approval came as a surprise to many, as the FDA’s own advisory committee last November voted 8 to 1 against approving the drug, citing the lack of strong evidence the drug works. An advisory board member resigned following the FDA’s decision, STAT reported.
“The FDA approved this based on the amyloid plaques reducing antibodies,” Schneider said. Reducing the plaque, however, doesn’t prove there is a clinical benefit, such as preventing memory deterioration, he says.
The companies marketing the drug, Biogen and Eisai, are to conduct ongoing studies to verify that the drug has a clinical benefit. If this new trial does not show a benefit, the FDA can withdraw the approval.
Another expert, James E. Galvin, MD, professor of neurology at the Miller School of Medicine at the University of Miami, compares the approval of the new drug based on its ability to remove plaque to an approved cancer drug because it can shrink a tumor. “If you have an anticancer drug that shrinks a tumor, you have proof that it works, that it engages its target. It may or may not mean a clinical effect, or a small clinical effect.” Galvin was a researcher on one of the aducanumab clinical trials and an advisor to Biogen.
Like other experts, Galvin says the approval of aducanumab could pave the way for the approval of future Alzheimer’s drugs that will be even more effective. “The first drug approved isn’t always the best.”