Real-world safety of Symdeko: insights from the food and drug administration adverse event reporting system.

ABSTRACT:

Background

Symdeko, a cystic fibrosis transmembrane conductance regulator (CFTR), is widely utilized for treating cystic fibrosis (CF). Although its safety has been validated in numerous clinical trials, the real-world safety profile still needs further investagation.

Methods

This study analyzed adverse event (AE) reports related to Symdeko from the FDA Adverse Event Reporting System (FAERS) spanning Q1 2018 to Q3 2024. Four disproportionality analysis methods were employed: Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Multi-Item Gamma Poisson Shrinker (MGPS), and Bayesian Confidence Propagation Neural Network (BCPNN). A descriptive analysis of the time to onset (TTO) of AEs was conducted, and the Weibull distribution was used to predict temporal variation

SUBMITTER: Wei S 

PROVIDER: S-EPMC12611701 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature