Real-world safety assessment of burosumab: a pharmacovigilance study utilizing the FDA adverse event reporting system

OBJECTIVE: This study aims to evaluate the adverse events (AEs) associated with burosumab in real-world clinical practice using data from the United States Food and Drug Administration (FDA) Adverse Event Reporting System (FAERS), and to provide evidence for optimizing its clinical safety management. METHODS: Data for this study were retrieved from the FAERS database via OpenVigil 2.1, covering the period from the first quarter of 2018 to the third quarter of 2024. Disproportionality analyses were performed to quantify adverse event signals associated with burosumab, employing four established methods: Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Multi-Item Gamma Poisson Shrinker (MGPS), and Bayesian Confidence Propagation Neural Network (BCPNN). RESULTS: A total of 5054 adverse events (AEs) associated with burosumab were identified. Commonly reported AEs included injection site reactions, musculoskeletal pain, restless leg syndrome, skeletal deformities, and corrective orthopedic surgeries necessitated by severe bone deformities. Additionally, several concerning AEs were observed, such as hyperparathyroidism, tertiary hyperparathyroidism, nephrocalcinosis, and dental and periodontal complications. CONCLUSION: This study provides an initial evaluation of burosumab’s safety in real-world practice, identifying noteworthy adverse event signals that broaden current understanding and support more informed prescribing decisions for patients with XLH.