On November 6, FDA’s panel of outside experts concluded that the clinical data did not support the approval of Biogen’s Alzheimer’s drug, aducanumab. Ten of 11 panelists voted that the data provided by Biogen was not enough to demonstrate aducanumab could slow cognitive decline in people with Alzheimer’s.
Now the FDA must determine whether they will proceed with approving the drug.
The Food and Drug Administration (FDA) plans to meet with an expert panel in early November to help determine if a controversial drug to treat Alzheimer’s disease should get approved for consumer use. The drug, known as aducanumab, was studied in clinical trials last year that were halted. The drug’s maker, Biogen, later released data suggesting the drug is actually effective at slowing the progression of Alzheimer’s disease.
The committee of experts will convene through an online conferencing platform on November 6, but background materials and pre-recorded presentations will be available to the public at least two business days beforehand, according to the FDA. The FDA has also established a docket where members of the public can write in with data, information, and views. Amit Sachdev, MD, medical director in the department of neurology at Michigan State University, tells Get Meds Info the expert panel is “a standard part of the approval process.”
Aducanumab has a storied history. Biogen halted two phase 3 clinical trials for the drug in March 2019, after determining the drug did not meet its goals of treating Alzheimer’s. The company also discontinued the development of aducanumab. But, on October 22, 2019, Biogen revealed that they were applying for FDA marketing approval of the drug.
In a press release, Biogen explained that the company re-analyzed data from the trial to include patients who had continued to take the drug beyond the trial’s December 26, 2018 cut-off date through March 21, 2019. One trial showed a “significant reduction in clinical decline,” the company said.
“Patients who received aducanumab experienced significant benefits on measures of cognition and function such as memory, orientation, and language,” the announcement continued. “Patients also experienced benefits on activities of daily living including conducting personal finances, performing household chores such as cleaning, shopping, and doing laundry, and independently traveling out of the home.”
Biogen also said in November 2019 that the company should not be required to do another trial.
The drug has gotten mixed responses from the medical and Alzheimer’s communities. The Alzheimer’s Association said in a press release that it is “encouraged” that Biogen will pursue FDA approval of aducanumab, adding, “all currently pursued treatments that are considered safe should be continued to determine their efficacy.”
But some took to social media to say the drug’s approval feels rushed.
More than 5 million Americans have Alzheimer’s disease, according to the Alzheimer’s Association. The disease is the sixth leading cause of death in the U.S., and there is currently no medical treatment to stop its development. If approved, aducanumab would become the first therapy authorized to slow the decline of Alzheimer’s disease.
What This Means For You
Aducanumab is a monoclonal antibody, meaning it’s a lab-created version of an antibody. An antibody is a protein component of the immune system that circulates in the blood. Antibodies recognize foreign substances in the body and work to neutralize them.
Aducanumab, which is given by IV, selectively binds to select amyloid proteins. In Alzheimer’s disease, abnormal levels of certain amyloid proteins clump together to form plaques that collect between neurons and disrupt cell function. Research is still ongoing to better understand how, and at what stage of Alzheimer’s, certain forms of amyloids influence the disease and its progression.
In one clinical trial, aducanumab showed a substantial reduction of amyloid plaques.
“This finding was robust and unquestionable; plaques were markedly decreased in all cortical brain regions examined,” according to a report published in The Lancet. “However, clinical effects, assessed using four clinical scales after six and 12 months of treatment, were far less certain.” Still, the report says, “this early study demonstrated aducanumab to be a robust amyloid plaque buster.”
What Do Experts Think?
Overall, they’re excited about the possibility of offering the drug to patients. “There are some people who will benefit,” Sachdev says.
Scott Kaiser, MD, a geriatrician and director of Geriatric Cognitive Health for Pacific Neuroscience Institute at Providence Saint John’s Health Center in California, tells Get Meds Info that the drug is “very exciting.”
“Therapies to address Alzheimer’s disease have been elusive,” he says. “There’s currently no meaningful disease-modifying pharmaceutical therapy. The fact that there’s anything that can provide benefit is a huge feat. As somebody who takes care of patients with dementia every day, if you could delay the progression of symptoms, maintain people’s independence for a greater amount of time, that’s a significant factor in people’s lives.”
If approved, aducanumab “offers hope to patients and families where there are very few options,” Kaiser says. Kaiser admits that it’s “unusual” for a company to change a decision to halt a trial. But, he adds, “when you look at the evidence, it does seem quite clear that there are benefits.” The big question, Kaiser says, is whether more studies need to be done to determine whether the drug is safe. “That’s the job of this committee,” he says.
While the drug is promising, Sachdev points out that, “there is a concern that the medication works too late in the disease process.”
While Sachdev says he’s hopeful aducanumab may help some Alzheimer’s patients in the future, he also stresses the importance of healthy living. “Graceful aging often involves some loss of memory. It’s hard to know who will develop problems with daily activities and who will not,” he says. “For most people, a new medication will not be the right answer. The best approach is to foster a healthy brain by fostering a healthy body.”