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Risk of Non-arteritic Anterior Ischemic Optic Neuropathy (NAION) Following Glucagon-Like Peptide-1 Receptor Agonist (GLP-1 RA) Use in Patients with Type 2 Diabetes Mellitus

    Basic Details
    Date Posted
    Status
    In progress
    Medical Product
    glucagon-like peptide-1 receptor agonists (GLP-1 RAs)
    Health Outcome(s)
    non-arteritic anterior ischemic optic neuropathy
    Description

    Recently, the safety of glucagon-like peptide 1 receptor agonists (GLP-1 RAs) have been questioned with respect to an increased risk of non-arteritic anterior ischemic optic neuropathy (NAION), a form of optic neuropathy which can cause acute blindness in adults. This concern was raised by a single referral-center study focused on semaglutide, and subsequent observational studies produced conflicting results. Given the severe and irreversible nature of NAION and the growing popularity of GLP-1 RAs, we aim to evaluate the previously observed safety signal with respect to NAION using a principled approach to study design and analysis. Therefore, we aim to conduct a non-randomized cohort study in the HealthVerity aggregated de-duplicated claims data source (January 2018 – June 2024) of the FDA Sentinel System’s Real World Evidence Data Enterprise (RWE-DE) Commercial Network, augmented with several outcome validity-enhancing activities leveraging data linkages between insurance claims and electronic health records (EHRs) from two data sources: Mass General Brigham (MGB) and HealthVerity. This investigation is designed using the “PRocess guide for INferential studies using healthcare data from routine ClinIcal Practice to evaLuate causal Effects of Drugs” (PRINCIPLED) framework, which is a standard Sentinel process to conduct causal inferential studies of medication outcomes. This protocol outlines the specification and emulation of the target trial to answer the study question of interest.

    The Sentinel Initiative has posted a new protocol/surveillance plan for public comment. We invite stakeholders and the general public to review the document and provide feedback. The public comment period is between June 12, 2025 - June 25, 2025.

    Additional Details
    FDA Center
    CDER
    Time Period
    January 1, 2018 – June 30, 2024
    Analysis Type
    Retrospective Inferential
    Population / Cohort
    Individuals 18 years of age and older
    Data Sources
    Sentinel Real-World Evidence Data Enterprise (RWE-DE)