The science does not yet support regulatory approval of amyloid‐targeting therapies for Alzheimer's disease based solely on biomarker evidence
Year: 2025
Type: article
Abstract: To rapidly develop safer and more effective amyloid-targeting therapies for Alzheimer's disease (AD) and to facilitate drug approval for prevention in asymptomatic amyloid-positive individuals, Aisen et al.recently proposed in a perspective article that traditional regulatory approval-not accelerated approval-could be considered based on "robust amyloid lowering and reasonable safety". 1 Because this is a critical goal for our field, we reviewed existing evidence and argue that, for now, at least three rationales underscore the necessity for AD trials to still use meaningful clinical endpoints ensuring that clinicians can responsibly prescribe adequately safe and efficacious treatments. 1. Amyloid load reduction is an unvalidated surrogate for clinical benefit.Amyloid-targeting antibodies have demonstrated efficacy in both amyloid reduction and slowing cognitive decline due to
Source: Alzheimer s & Dementia
DOI: 10.1002/alz.70068
Institutions: Centre National de la Recherche Scientifique, Centre Hospitalier Universitaire de Bordeaux, Institut des Maladies Neurodégénératives, Washington University in St. Louis, Mayo Clinic, University of Geneva, University Hospital of Geneva, University of California San Diego, University of California, Irvine, University of Southern California, University of Pennsylvania, Inserm, Sorbonne Université, Pitié-Salpêtrière Hospital, Institut du Cerveau
Language: English
Subfield: Physiology
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,200
Funder: National Institute on Aging
OpenAlex ID: W4409526736