Shipments of the medication are expected to go out in just a couple of weeks. Hospitals are on tap to administer treatment when needed. And there is serious division in the FDA, especially around the drug’s effectiveness.
Aducanumab, to be sold under the brand name Aduhelm, was approved to treat patients with Alzheimer’s disease — against the recommendation of an FDA advisory committee that concluded last year there is not enough evidence to support approval of the treatment. The FDA has not approved a novel therapy for Alzheimer’s disease since 2003.
Patients now have many questions about aducanumab. At Memorial Healthcare in Michigan, the hospital’s Institute for Neuroscience has been closely following the drug’s development and already has a waiting list of potential patients who may meet the criteria to receive the treatment.
“What’s really exciting is that aducanumab is the first new FDA-approved Alzheimer’s treatment in nearly 20 years, and we’re optimistic this will spark a wave of new research and innovation in this space. Patients are excited for that, too, and if aducanumab is the first step toward that brighter future, patients are eager to be part of it.”
For now, here are some basic questions patients and their families might have about aducanumab, answered by doctors from across the United States.
Who is the drug for?
Aducanumab is approved for patients with Alzheimer’s disease.
“The FDA authorization of Aduhelm is fairly broad, indicating it is for Alzheimer’s disease as a whole,” Aburashed said. However, the pharmaceutical company Biogen and its Japanese partner Eisai tested aducanumab, administered through intravenous infusion, in patients with mild cognitive impairment, the impaired thinking and memory loss that often develops into dementia.
So, “the clinical development program targeted early mild cognitive impairment and mild cases of Alzheimer’s disease as opposed to more progressive cases,” Aburashed said. “At Memorial Healthcare Institute for Neuroscience, we intend to be very focused on this subset of patients for treatment consideration, given some of the risks surrounding the drug.”
“Ultimately, there are many factors to consider when deciding to treat a patient with a newly approved medication, especially when there is some uncertainty about its clinical benefit,” she added. “There may be a subgroup of patients that benefits from this drug. My own decision to prescribe it will be made in collaboration with the patient, and based on the patient’s age, imaging findings to rule out contraindications, type and severity of symptoms, risk factors and overall diagnostic impression.”
Why is there some controversy?
Aducanumab’s road to FDA approval has been rocky, due to halted trials and a FDA advisory panel not entirely convinced of the drug’s effectiveness.
In March 2019, two Phase 3 clinical trials of aducanumab were discontinued because a futility analysis found the trials were unlikely to meet their primary goals at completion.
“Studies were halted and the drug stopped development,” Dr. Lon Schneider, Della Martin Chair of Psychiatry and Neuroscience and director of the California Alzheimer’s Disease Center at the University of Southern California, told CNN on Tuesday.
Then, “a little bit later, the company analyzed all of its data, which included extra patients who were still being treated while the futility analysis was under way and they found that one of the studies was flat-out negative,” Schneider said. “But the other study was nominally statistically significant in the cognitive outcomes, and from that the FDA was supportive of the company essentially putting in a new drug…