Pharmacovigilance of obeticholic acid: An analysis of the Food and Drug Administration Adverse Event Reporting System database.

Aims: Obeticholic acid (OCA) is a farnesoid X nuclear receptor agonist that was approved by the Food and Drug Administration in 2016 for the treatment of primary biliary cholangitis. The aim of this study was to investigate the adverse events (AEs) associated with OCA.

Methods: We extracted data from the Food and Drug Administration Adverse Event Reporting System (FAERS) database from the second quarter of 2016 to the first quarter of 2025. Disproportionality analyses were conducted to assess risk signals of AEs associated with OCA through 4 algorithms, encompassing reporting odds ratio (ROR), proportional reporting ratio, Bayesian confidence propagation neural network and empirical Bayesian geometric mean.

Results: Out of 14 859 866 reports from the FAERS database, 6177 reports with OCA as principal suspect AEs were identified. Across 27 organ systems, we identified 187 significant disproportionate preferred terms, which mainly concentrated on gastrointestinal, hepatobiliary, skin and subcutaneous tissue disorders. We also observed unlisted adverse reactions, such as oesophageal varices (ROR 58.39 [95% confidence interval 43.67-78.08]) and hepatic fibrosis (ROR 74.95 [95% confidence interval 58.59-95.88]). Most AEs occurred within 30 days or after 1 year of medication, and were more frequent in female patients.

Conclusion: This real-world study identified OCA-associated AEs, especially some not recorded in the drug insert, and revealed delayed AEs beyond 1 year. These findings highlight the importance of active monitoring for patient medication safety, and we recommend that the monitoring duration be no less than 1 year.