The approval is significant in many ways. Aduhelm is the first novel therapy approved for Alzheimer’s disease since 2003. Perhaps more significantly, Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer’s disease, the presence of amyloid beta plaques in the brain. The clinical trials for Aduhelm were the first to show that a reduction in these plaques—a hallmark finding in the brain of patients with Alzheimer’s—is expected to lead to a reduction in the clinical decline of this devastating form of dementia.
Neurimmune discovered aducanumab together with a team of researchers at the University of Zurich. In 2007 Neurimmune and Biogen entered into a collaborative development and license agreement for aducanumab's development and commercialization. Since October 2017 Biogen and Eisai have collaborated on the development and commercialization of aducanumab globally.
“The data of three separate clinical studies unequivocally show that aducanumab’s biological activity removed amyloid from brains of patients with Alzheimer’s disease. Based on our understanding of the disease, substantial amyloid removal is required to slow disease progression.” said Roger Nitsch, CEO of Neurimmune. “Our outstanding 14-year collaboration with Biogen succeeded in bringing this new treatment option to patients and their physicians.”
Adhelm was approved using the Accelerated Approval pathway, under which the FDA approves a drug for a serious or life-threatening illness that may provide meaningful therapeutic benefit over existing treatments when the drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients and there remains some uncertainty about the drug’s clinical benefit. The approval was contentious. In a long article on the FDA website Patrizia Cavazzoni, Director, FDA Center for Drug Evaluation and Research, explains the decision.
(Press release / SK)