Biogen and Eisai have secured accelerated approval from the US Food and Drug Administration (FDA) for Aduhelm (aducanumab-avwa) as the first and only new treatment for Alzheimer’s disease since 2003.
Alzheimer’s is a progressive brain disease that cannot be reversed. It gradually destroys the thinking skills and memory, and over time, the ability to do simple tasks.
First-of-its-kind to obtain FDA approval for Alzheimer’s, Aduhelm is a human monoclonal antibody that can address the fundamental pathology of the disease by lowering amyloid beta plaques in the brain.
FDA Center for Drug Evaluation and Research director Patrizia Cavazzoni said: “Alzheimer’s disease is a devastating illness that can have a profound impact on the lives of people diagnosed with the disease as well as their loved ones.
“Currently available therapies only treat symptoms of the disease; this treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s.”
The accelerated approval by the FDA is based on the data obtained from clinical trials, which showed the drug’s effect on mitigating amyloid beta plaques, a biomarker that may predict clinical benefit and in this case a decrease in clinical decline.
Aduhelm’s efficacy was assessed by researchers in Phase III EMERGE (Study 1) and ENGAGE (Study 2) clinical trials and Phase Ib PRIME (Study 3) trial.
These trials were conducted in patients with early stages of Alzheimer’s and with confirmed incidence of amyloid pathology.
Biogen stated that the patients who received the treatment had experienced a consistent dose-and time-dependent effect on reducing amyloid beta plaque.
As part of FDA approval, the company has planned to carry out a controlled trial to confirm Aduhelm’s clinical benefit in Alzheimer’s patients.
The continued approval for the drug will be based on validation of its clinical benefit in confirmatory trials.
The safety information of Aduhelm includes a warning for amyloid-related imaging abnormalities (ARIA), which is the most common temporary swelling in brain areas.
ARIA generally resolve over time and does not have symptoms, though some people may have headache, confusion, dizziness, vision changes or nausea, among others.
In April this year, Biogen and Bio-Thera Solutions announced a commercialisation and license agreement to develop, manufacture and commercialise BAT1806.