Adverse events associated with obeticholic acid: a real-world, pharmacovigilance study

Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease predominantly affecting middle-aged women. While ursodeoxycholic acid (UDCA) is the first-line treatment, 30–40% of patients do not respond adequately, necessitating alternative therapies like Obeticholic Acid (OCA), an FXR agonist. The long-term safety of OCA remains insufficiently studied. This study utilized the US FDA Adverse Event Reporting System (FAERS) to evaluate OCA safety through large-scale data mining, using disproportionality analyses (ROR, PRR, BCPNN, and MGPS) to identify adverse event signals. From Q2 2016 to Q1 2024, 5,864 reports linked to OCA usage were identified among 13,245,871 AE reports. Significant signals across 27 System Organ Classes were found, with pruritus (12.54%), fatigue (4.16%), and nausea (1.64%) being the most prevalent adverse events. Severe hepatic events like liver failure were rare (0.6%). Median time to onset of AEs was 178 days. The most common outcomes reported were important medical events (18.6%), hospitalization (17.8%), and death (6.5%). This study provides key insights into the safety profile of OCA, highlighting the importance of monitoring for pruritus and hepatic complications, particularly within the first six months of treatment.