Evaluating adverse events of pegvaliase-pqpz in phenylketonuria treatment: A comprehensive safety assessment.

Objectives: In 2018, the Food and Drug Administration approved pegvaliase-pqpz as an enzyme replacement therapy to lower blood phenylalanine levels in adults with phenylketonuria. However, its potential side effects have not been fully explored in a real-world setting. This study aimed to examine pegvaliase-pqpz-related adverse events and investigate their associated risk factors to offer important insights into clinical drug applications. Methods: We extracted reports on adverse events from Food and Drug Administration Adverse Event Reporting System database from 2018Q2 to 2024Q3. The analyses included demographic data, time-to-onset, sex, age, dosage, reporting personnel, and subgroup signals. The Reporting Odds Ratio, Proportional Reporting Ratio, Bayesian Confidence Propagation Neural Network, and Multi-item Gamma Poisson Shrinker were used for disproportionality analysis. A screening procedure was implemented for designated medical events, focusing on medication-related adverse events. Results: A total of 6264 reports were identified. Arthralgia was the most frequently reported adverse events. Lymphadenopathy, serum sickness, and tunnel vision were identified as unexpected signals. Weibull distribution analysis revealed that the incidence of adverse events decreased over time. Conclusions: Our study provides a deeper insight into the safety profile of pegvaliase-pqpz, thereby aiding healthcare professionals in clinical surveillance and risk identification.