Background and Importance Obeticholic acid (OCA) is an orphan drug for patients with primary biliary cholangitis (PBC), a rare autoimmune disease, who do not respond adequately to treatment with ursodeoxycholic acid (UDCA) or do not tolerate it.

Aim and Objectives To evaluate the efficacy and safety of OCA in patients with PBC.

Material and Methods Descriptive and retrospective study. Data from patients who received OCA from January-2021 April-2023 were analysed. Demographic variables (sex and age); previous treatment with UDCA; plasma values of liver markers: alkaline phosphatase (ALP), gamma-glutamyl transferase (GGT), total bilirubin (Bt), aspartate aminotransferase (AST) and alanine aminotransferase (ALT), at the start of treatment with OCA, at 6 months and at 12 months, were collected from the electronic medical records programme. Adverse effects described since the start of treatment were also collected. Treatment response was defined as an ALP value less than 1.67 times the upper limit of normality (ULN), a Bt value within the normal range and a decrease from baseline ALP value of at least 15%, according to the pivotal drug trial.

Results Thirty patients (87% women) were evaluated. The median age was 66 years. All patients except one (3%) were on treatment with UDCA. Median values and percentile 25–75 are shown.

A reduction of ALP>15% was achieved in 15 (50%) and 16 patients (53%) at 6 and 12 months, respectively. 29 patients (97%) had bilirubin in the normal range at 6 months, and all (100%) at 12 months. ALP<1.67xULN was obtained in seven (23%) and 11 (37%) patients at 6 and 12 months, respectively. Overall, four patients (13%) fulfilled the three pivotal trial conditions at 6 months and eight patients (26%) at 12 months. Adverse reactions reported were: pruritus in 14 patients (47%) and fatigue in one (3%).

Conclusion and Relevance Based on clinical trial endpoints, OCA achieved modest results at 6 months, which doubled 1 year after initiation of treatment. Further studies are needed to assess long-term benefit.

Conflict of Interest No conflict of interest.