This past Wednesday, the Penn Memory Center held a community event for PMC patients and families led by co-directors Drs. Jason Karlawish and David Wolk to discuss the Food and Drug Administration’s (FDA) decision to approve Aduhelm (aducanumab). The focus was the impact of the approval on the PMC’s clinical and research operations.
Drs. Karlawish and Wolk emphasized that the clinical benefit of Aduhelm is uncertain. One study testing it was positive; the other negative. An FDA advisory board’s and statistician’s reviews of these data concluded that it should not be approved as safe and effective. The FDA then used a different set of regulations and evidence to approve the drug for sale, but with conditions and caveats. These are discussed below.
Aduhelm has been tested only on people with early-stage Alzheimer’s disease. It will not be appropriate for all patients. Patients interested in Aduhelm need to discuss the drug’s uncertainties, costs, risks, and other factors with their care team. Eligibility for a prescription will be determined based on the results of tests for amyloid, and the safety of the drug based on the results of an MRI, and possibly as well testing for the APOE4 gene.
Dr. Wolk remarked that after some 20 years as a researcher and clinician, this is the first Alzheimer’s drug to be approved by the FDA.
“I do think it’s important that we, as a field, appreciate the context of what’s happened here… the fact that a crazy idea of giving an immune based drug to remove or target amyloid in the brain can be given and you can actually then see with a scan removal of that amyloid from the brain is really remarkable… I think this a hopeful time,” said Dr. Wolk.
It is not known precisely when Aduhelm will be available to PMC patients or the criteria insurers, like Medicare, will use to determine the kinds of patients that it is a reasonable and necessary treatment.
We will update the PMC community as we receive more information.
The FDA approved Aduhelm using an Accelerated Approval pathway. This has two parts. First, the approval recognizes that the drug clears amyloid from the brain and that this may be associated with benefit to a patient’s health and well being. Second, another study of the drug is needed to confirm this clinical benefit. Biogen has not yet announced the details of this study.
PMC has been a testing site of aducanumab in the past, and Drs. Karlawish and Wolk said they hope PMC will be a site for the confirmatory trial.
To answer the many questions about Aduhelm, a Q+A page has been added to the PMC website. The page will be continuously updated.