The U.S. Food and Drug Administration (FDA) approved Aduhelm (aducanumab) developed by Biogen Inc. to treat people with Alzheimer’s disease. The approval was through the FDA’s 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.
Aduhelm, a human monoclonal antibody, is the first novel therapy approved for Alzheimer’s disease since 2003. The treatment reduces the presence of amyloid beta plaques in the brain that are a hallmark of Alzheimer’s disease. A reduction in the plaques is expected to reduce the clinical decline of Alzheimer’s disease.
The drug was developed by Biogen. The late-stage development program for Aduhelm consisted of two phase 3 clinical trials. One study met the primary endpoint, showing reduction in clinical decline. The second trial did not meet the primary endpoint. In all studies in which it was evaluated, however, Aduhelm consistently and very convincingly reduced the level of amyloid plaques in the brain in a dose- and time-dependent fashion.
FDA will continue to monitor Aduhelm as it reaches the market. As a condition of the Accelerated Approval, the FDA is requiring Biogen to conduct a post-approval clinical trial to verify the drug’s clinical benefit. If the drug does not work as intended, the FDA may remove it from the market.
This was reported by the FDA and Biogen on June 7, 2021.