5 Things to Know About the New Alzheimer’s Drug
For the first time in a long time, the FDA approved a new med for Alzheimer’s. Strong opinions (and some opposition) followed.
On June 7, the FDA approved a drug called Aduhelm (aducanumab) to help slow cognitive decline from Alzheimer’s dementia. This announcement represented a huge breakthrough in the Alzheimer’s community—it’s the first new Alzheimer’s drug to receive FDA approval since 2003, and the first to target the underlying course of disease rather than just alleviating symptoms.
If you or a loved one is one of the six million Americans living with Alzheimer’s, you probably have a lot of feelings and questions about this big news. Aducanumab’s approval has garnered significant scientific and media attention (and generated a bit of controversy in the process). But the question everyone wants an answer to? How will patients get access to this drug?
Let’s go over the basics:
1. Aduhelm (aducanumab) is the first Alzheimer’s treatment of its kind.
This drug is an anti-amyloid antibody therapy. It is an intravenous treatment that targets something called beta amyloid, a protein compound that accumulates in the brain and is one of the hallmarks of Alzheimer’s disease progression. “This therapy was approved by the FDA because it demonstrated that it clears beta amyloid,” says Heather Snyder, PhD, vice president of medical and scientific relations for the Alzheimer’s Association in Chicago. The reduction in amyloid plaque is believed to help slow the progression of dementia.
Previous Alzheimer’s treatments have been designed to help with symptoms such as memory loss or brain fog, but nothing has ever been approved to slow the course of the disease itself. “Aducanumab is the first treatment really targeting the underlying biology,” Snyder says. That’s what makes this medication so revolutionary in the field.
2. It was approved through an accelerated process because it fills an “unmet medical need” to treat a formerly untreatable condition.
“An accelerated approval allows the FDA to approve a drug for a serious condition that fills an unmet medical need, based on what they would call a surrogate or intermediate endpoint,” Snyder explains. This means that the endpoint (in this case, the reduction of beta amyloid in the brain) is thought to provide a benefit to patients but is not 100% proven to do so.
Because of the significant burden Alzheimer’s presents to so many people, and the lack of available treatment options like this, the FDA decided to approve aducanumab through the fast track. This comes with the expectation that the drug company (Biogen) will provide updates on the safety and efficacy of the drug as it rolls it out to more people, thus producing more concrete evidence about how well it works.
3. The FDA’s decision has been met with strong opinions—some positive, some negative.
Not everyone is happy about the approval of aducanumab—in fact, the FDA’s advisory committee declined to endorse the drug back in November, citing a lack of evidence that it actually slows disease progression. Three FDA members of the committee have resigned since the FDA announced it was moving forward with approval. Despite this, prominent groups such as the Alzheimer’s Association have praised the FDA’s decision, citing the immediate potential benefit to Alzheimer’s patients and their families.
Here’s why not everyone is so sure about aducanumab: It has only been tested in a total of 3,482 Alzheimer’s patients with mild to moderate cognitive impairment, and not all studies have shown a positive benefit from the drug. “The trials were complicated in that they were stopped early,” Snyder says. “One of the trials was positive, and the other was not. Within the scientific community, because this is not the usual path that we might see a therapy go through, there are a lot of questions in that regard.” There is also some uncertainty about whether reducing beta amyloid will actually help preserve memory and cognition, as well as concern about a side effect called ARIA—amyloid-related imaging abnormalities (a type of swelling in the brain). In one of the trials, 34% of people on the highest dose of aducanumab experienced ARIA, but 80% of those cases were asymptomatic. Of the 20% who experienced ARIA symptoms such as headache and confusion, those symptoms were considered temporary (though there is obviously more research needed into the potential long-term risks versus benefits of this drug).
Snyder notes that the Alzheimer’s Association regards this FDA approval as a step in the right direction: "We’ve evaluated and think the FDA made the right call in moving forward with approving this drug while also requiring further study.”
4. Access is still a major issue.
Biogen has priced aducanumab at a steep $56,000 per year (yes, you read that right). The company expects to start shipping out the drug to 900 treatment centers in the next few weeks. And as of now, we don’t know much about whether private insurance policies will cover the treatment, and if they do, what the out-of-pocket cost will be to patients. The drug is expected to be covered under Medicare, but patients may still be responsible for some of the cost on their own.
On June 12, the Alzheimer’s Association released a statement calling the price of aducanumab “unacceptable” and calling for equitable, sustainable access to the new drug. Since this drug is specifically targeting beta amyloid, Snyder notes, physicians will need to do diagnostic testing to confirm the presence of beta amyloid in order to prescribe it. This may end up further increasing the patient’s medical bills.
5. Further research will likely tell us more—but for now, talk to your doctor.
The exciting thing about aducanumab (besides being a breakthrough form of treatment) is the hope it presents for further study in Alzheimer’s research and treatment. “History has told us that the approval of a first drug in a new category invigorates the field,” Snyder says. “As our understanding of the disease grows … that innovation and that excitement [can be generated] into new ideas and new potential treatments going forward.”
There are other clinical trials in the works targeting different aspects of the biology of Alzheimer’s disease. “This is a complex disease and will likely need a multi-pronged approach in terms of treatment,” Snyder says. Hopefully, this drug is only the first of more like it to come.
For now, the best thing Alzheimer’s patients and caregivers can do is have an honest conversation with their physician. “Having the conversation with their healthcare provider regarding whether it is an option for them is certainly an important next step,” Snyder says. Your doctor can keep you updated on whether you might be a good fit for aducanumab, and if so, how to access it in the safest and most affordable way.
Alzheimer’s Statistics: Alzheimer’s Association. (n.d.) “Facts and Figures.” https://www.alz.org/alzheimers-dementia/facts-figures
FDA Approval of Aducanumab: U.S. Food and Drug Administration. (2021.) “FDA’s Decision to Approve New Treatment for Alzheimer’s Disease.” https://www.fda.gov/drugs/news-events-human-drugs/fdas-decision-approve-new-treatment-alzheimers-disease
Beta Amyloid: Alzheimer’s Association. (2017.) “Beta-amyloid and the amyloid hypothesis.” https://www.alz.org/national/documents/topicsheet_betaamyloid.pdf
Alzheimer’s Association Statement on FDA Approval: Alzheimer’s Association. (2021.) “Alzheimer's Association Welcomes FDA Approval of Aducanumab.” https://www.alz.org/news/2021/alzheimers-association-fda-approval-aducanumab
Alzheimer’s Association Statement on Access: Alzheimer’s Association. (2021.) “Next Steps for New Alzheimer's Treatment.” https://www.alz.org/news/2021/next-steps-for-new-alzheimers-treatment