A real-world analysis of the FDA Adverse Event Reporting System identified 4,326 adverse event reports associated with alglucosidase alfa in Pompe disease. The pharmacovigilance review highlights both known complications and rare autoinflammatory signals. The scale of the FAERS capture provides an expanded safety signal landscape for enzyme replacement therapy, where patient heterogeneity and long-term exposure remain central concerns. For clinicians and pharmacovigilance teams, the actionable outcome is a prioritized review of autoinflammatory patterns and known adverse-event clusters to guide monitoring and reporting. The study reinforces the ongoing role of FAERS in identifying signals that may not surface in pre-approval trials or may require much larger exposure denominators.
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