June 9, 2021 -- The FDA's approval on Monday of a new drug to treat Alzheimer's disease was met with both praise and criticism. As the first Alzheimer's drug approved since 2003, advocates applaud the decision. Critics say the approval was based on inadequate proof that the drug works and balk at the price tag of $56,000 a 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, as well as tangles known as tau and other changes in the brain, are what lead to memory loss and eventually the inability to perform simple tasks like dressing oneself.
The drug is given intravenously once a month.
According to Biogen, the annual price of the infusions is about $56,000. People won't likely be paying that amount, says Lon Schneider, MD, director of the California Alzheimer Disease Center. By Tuesday morning, Biogen promised multiple ways to help patients access the drug. The company will provide service coordinators to provide one-on-one support, it says.
Specifics about how Medicare and insurance plans will cover the drug will take some time to work out, Schneider says. But he says even people without insurance will likely be able to negotiate down the price.
A spokesperson for the Centers for Medicare and Medicaid Services said, “CMS is reviewing the FDA’s decision regarding aducanumab and will have more information soon.”
Best Patients? Advice for Families
The new drug is certainly not meant for everyone with Alzheimer's, says Julia Biernot, MD, a behavioral neurologist at the University of Maryland School of Medicine, Baltimore. "It's important to know that it is most likely going to be indicated in patients who have 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 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 in the brain and small areas of bleeding.
While she calls the approval "an exciting development overall," Biernot also cautions families that the treatment would probably be needed indefinitely.
"There isn't a profile for the best patient," Schneider says. The clinical studies enrolled people with mild cognitive impairment or MCI, a precursor to Alzheimer's, and with mild Alzheimer's. "That's the group in which the drug has been tested," he says. "Not necessarily tested and shown to be effective, but tested."
"I think people who come with symptoms, with MCI due to Alzheimer's or with mild Alzheimer's, who wish to try the monthly infusions for a year and a half [the schedule used in the trials], we would be happy to support them," Schneider says. He stresses, however: "Families need to do their own research and talk to their doctor."
FDA's Accelerated Approval
The FDA granted accelerated approval based on clinical trials that showed the reduction of plaque in the brain in those given the drug compared to those in the control or 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 lack of strong evidence that the drug works. One member of the advisory board resigned over the FDA decision, STAT reported.
"The FDA approved this based on the antibody reducing amyloid plaques," Schneider says. Reduction of the plaque, however, does not prove that there is a clinical benefit, such as preventing deterioration of memory, he says.
The companies marketing the drug, Biogen and Eisai, must do ongoing studies to verify that the drug has a clinical benefit. If this new trial does not show a benefit, the FDA can withdraw approval.
Another expert, James E. Galvin, MD, professor of neurology at the University of Miami Miller School of Medicine, compares the approval of the new drug based on its ability to remove plaque to a cancer drug approved because it can shrink a tumor. "If you have a cancer drug that shrinks a tumor, you have proof that it works, it engages its target. That may or may not mean a clinical effect, or a small clinical effect." Galvin was an investigator on one of the clinical trials for aducanumab and is an advisor to Biogen.
Like other experts, Galvin says the aducanumab approval may pave the way for future Alzheimer's drugs being approved that are even more effective. "The first medicine approved is not necessarily always the best."