En español | The Food and Drug Administration (FDA) approved Biogen's aducanumab on June 7, making it the first Alzheimer's medication available in nearly 20 years and the only one that could slow the progression of the disease.
Aducanumab (which will go by the brand name Aduhelm) is not a cure for Alzheimer's, which affects 6.2 million Americans, and it does not reverse it. But data shows the drug is successful at breaking up sticky plaques of protein (amyloid) that accumulate in the brain — a hallmark of Alzheimer's disease. And health officials expect that if the drug can help remove those plaques, it may help slow the rate of cognitive decline in people who are in the early stages of the disease. Other Alzheimer's drugs on the market simply attempt to tackle the disease's debilitating symptoms.
The FDA's approval, however, comes with a condition: The drug's maker, Biogen, which co-developed aducanumab with Eisai, must do further studies to prove the drug works the way it's intended. In the meantime, a few million people suffering from the disease could have access to aducanumab, although the company did announce that the therapy is expected to cost $56,000 a year, and it's not yet known whether public or private insurers will pay for it.
The federal agency that oversees Medicare has initiated a review process to decide if it will cover the drug for beneficiaries — a decision that could cost billions. A Kaiser Family Foundation analysis found that if 500,000 Medicare beneficiaries went on aducanumab, total spending for the drug in one year would be nearly $29 billion. If 1 million beneficiaries receive aducanumab, spending will exceed $57 billion a year.
Patient advocacy groups welcomed the FDA’s June 7 decision. Kristen Clifford, chief program officer for the Alzheimer's Association, called the announcement a “landmark moment” — one that signifies “the beginning of a new future” for people living with Alzheimer's and their families.
"We know that slowing decline, particularly with early diagnosis, could add days, weeks, months, maybe even years of active life for individuals and families,” Clifford said. At the same time, the Alzheimer’s Association has called on Biogen to lower the price of its drug.
First Alzheimer’s drug since 2003 is not without controversy
The historic decision did not come without controversy. Back in November 2020, an FDA advisory committee expressed concern over the clinical trial data supporting aducanumab's effectiveness and essentially voted against approval of the drug.
The concern centered on two identically designed studies that yielded different results. One showed that participants with early or mild Alzheimer’s who were given high doses of aducanumab saw their symptoms decline. They were better able to handle everyday activities, like shopping and paying bills; their memory also improved, Biogen said. But the other study didn’t show the same positive outcomes, and the divergent results have made the drug contentious among experts.
The FDA doesn't have to accept its advisory committee’s vote, but it usually does. This time, however, the agency approved aducanumab through an accelerated process designed to be used for a drug that is significantly better than existing treatments for a serious or life-threatening illness.
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And while patients who qualify will have immediate access to the drug, Biogen will be required to conduct additional research to prove this treatment benefits patients. If the data doesn't hold up, the FDA can withdraw its approval of the drug.
Approval offers hope for more ‘precious’ moments
Aducanumab's approval gives Arthena Caston hope for the future. The 56-year-old Georgia resident was diagnosed five years ago with mild cognitive impairment (MCI), a precursor to Alzheimer's disease. She has also watched several family members suffer from the illness, and she is hoping this new treatment will forestall future generations from having to deal with its immense burden.
"I don't want my grandchildren to even know what the word Alzheimer’s means,” says Caston, who serves on the Alzheimer's Association board of directors, which supported the drug's approval leading up to the FDA's decision. “I hope this medicine is going to be the one that's going to say, ‘You know what, you don't have to worry about that.’ ”
What's more, she believes the treatment, which she's interested in taking, will give her the chance to watch her young grandchildren grow up. “I just want more time with my new babies. That's the first thing I think about: more time,” she says.
Jeffrey Cummings, a research professor in the Department of Brain Health at the University of Nevada, Las Vegas, says “the most important thing” about aducanumab's approval is the time it gives back to patients as it slows the deterioration of brain function. In one of the trials, participants who received high doses of aducanumab declined 22 percent more slowly than those who didn't get the treatment.
“That means that your grandma, who is really enjoying you and your children, will have that enjoyment for two or three more years before she enters that dementia phase of the illness,” which is the last stage of Alzheimer’s disease, Cummings explains.
"That's really important,” adds Cummings, who is a leading expert of Alzheimer’s clinical trials. “You know, that’s life. And it's towards the end of life, so those moments become even more precious.”
Other experts have been more critical of the drug since its June 7 approval.
Is the health system ready for this breakthrough treatment?
Not everyone with Alzheimer’s disease will qualify for the drug. One month after aducanumab’s approval, the FDA revised the drug’s prescribing label, narrowing its intended use to people in the early stages of the disease — about 2 million Americans.
And because the new drug targets amyloid plaques, the presence of the protein clumps will need to be confirmed. Currently, the only way to know for sure if amyloid plaques are present is with expensive brain scans and spinal taps that some patients may not be able to access. Simpler blood tests that doctors can also use to detect amyloid and other signs of Alzheimer's disease have proved promising but are not yet widely available.
Another obstacle: Aducanumab, a monoclonal antibody, is an infusion therapy, not a pill. Patients in the trials received the drug intravenously every four weeks at infusion centers, which makes the treatment less available to individuals who lack transportation or don't live near a site where it's offered.
"People who live in urban areas and can get scans and have a center near them where they could get infusions are [not going to have a problem getting aducanumab]. But that's not who we want to reach — we want to reach all of the patients who would be qualified for this drug,” Cummings says. “So there's going to have to be an adjustment of the entire health care system to accommodate a really exciting new drug.”
The treatment also comes with some side effects, including temporary swelling in the brain and tiny brain bleeds. This means patients on the therapy will need regular brain scans to ensure safety, Ronald Petersen, director of the Mayo Clinic Alzheimer's Disease Research Center, told AARP in a previous interview — a step that will require more time, technology and money from patients and providers.
Approval energizes Alzheimer's research
Aducanumab's approval will be a boon for Alzheimer's research, Cummings predicts. “Seeing the fact that we can sort of crack the code of Alzheimer's disease is just so meaningful, because it will attract funding, which will allow us to test more drugs, which will allow us to eventually make more approvals.”
A total of 126 different therapies are currently being tested in clinical trials, according to a recent analysis of the Alzheimer's Association research pipeline, authored by Cummings. Like aducanumab, some go after amyloid, including three promising monoclonal antibodies that are in the third and final phase of testing. Others explore different elements of the disease. And many of them — likely even a combination — could be the key to ending Alzheimer's.
In the meantime, researchers have made tremendous strides in understanding how people can reduce their risks for dementia with nonpharmacological interventions. Everything from diet and exercise to blood pressure control can help to preserve brain health and cognitive functioning throughout the aging process.
"AARP continues to support encouraging adults to reduce their risks for cognitive decline as they age and the development of new innovative research,” says Sarah Lenz Lock, senior vice president for policy and brain health at AARP and executive director of the Global Council for Brain Health.
Editor’s Note: This story, originally published June 7, has been updated to reflect new information.
Rachel Nania writes about health care and health policy for AARP. Previously she was a reporter and editor for WTOP Radio in Washington, D.C. A recipient of a Gracie Award and a regional Edward R. Murrow Award, she also participated in a dementia fellowship with the National Press Foundation.