Aducanumab for the treatment of Alzheimer's disease.

Alzheimer's disease (AD) is the most frequent neurodegenerative condition, the most common cause of dementia, and a leading cause of disability and death globally. Mounting evidence supported accumulation of amyloid β (Aβ) as the primary cause of AD pathology and sprouted a number of candidate treatments engaging Aβ from its production to its clearance, yet no amyloid-based drug candidate had been proven effective. Alternative pathomechanisms have been proposed, but still current treatments are limited to symptomatic therapy. Aducanumab (BIIB-037) is a fully human monoclonal IgG1 antibody that selectively binds aggregated forms of Aβ, inhibits its template activity and promotes clearance of Aβ deposits. Three early terminated trials in humans are available. Overall, conflicting results exist over measures of clinical efficacy, despite an objective decrease in Aβ burden. Amyloid-related imaging abnormalities emerge as the most significant treatment-related adverse event. Here, we provide a comprehensive review of the available evidence on aducanumab, a drug that recently received a debated accelerated approval for the treatment of mild AD by the U.S. Food and Drug Administration (FDA).