The U.S. Food and Drug Administration (FDA) approved Aduhelm (aducanumab) for the treatment of Alzheimer’s, a debilitating disease affecting 6.2 million Americans.
The agency granted approval of the drug even though its advisory committee and other experts voiced concerns about the drug’s efficacy and safety, based on the evidence produced during clinical trials, according to the New York Times.
The FDA said that Aduhelm is the first new treatment approved for Alzheimer’s since 2003. “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,” said Patrizia Cavazzoni, MD, Director of the FDA’s Center for Drug Evaluation and Research.
The FDA said Aduhelm was approved using the accelerated approval pathway, which can be used for a drug that provides a meaningful therapeutic advantage over existing treatments for a serious disease. Under that pathway, the FDA said it is requiring the drug’s developer, Biogen, to conduct a new randomized, controlled clinical trial to verify the drug’s clinical benefit. If the trial fails to verify clinical benefit, the FDA said it may withdraw approval of the drug.
Researchers evaluated Aduhelm’s efficacy in three separate studies representing a total of 3,482 patients. The studies consisted of double-blind, randomized, placebo-controlled dose-ranging studies in patients with Alzheimer’s disease. Patients receiving the treatment had significant dose-and time-dependent reduction of amyloid beta plaque, while patients in the control arm of the studies had no reduction of amyloid beta plaque.
Amyloid beta plaque was quantified using positron emission tomography (PET) imaging to estimate the brain levels of amyloid beta plaque in a composite of brain regions expected to be widely affected by Alzheimer’s disease pathology compared to a brain region expected to be spared of such pathology.
The prescribing information for Aduhelm includes a warning for amyloid-related imaging abnormalities (ARIA), which most commonly occurs as temporary swelling in areas of the brain that usually resolves over time and does not cause symptoms. However, some people may have symptoms such as headache, confusion, dizziness, vision changes, or nausea. Another warning for Aduhelm is for a risk of hypersensitivity reactions, including angioedema and urticaria.
