Obeticholic Acid for Primary Biliary Cholangitis in Patients with Inadequate Response to Ursodiol

Obeticholic acid (OCA), a selective farnesoid X receptor agonist, has improved liver tests in patients with primary biliary cholangitis (PBC) in phase II studies (NEJM JW Gastroenterol Jun 2015 and Gastroenterology 2015; 148:751). In the current phase III trial, researchers assessed its safety and efficacy among patients with PBC who had inadequate response (alkaline phosphatase ≥1.67 times the upper limit of normal [ULN] or abnormal total bilirubin <2 x ULN) or intolerance to ursodiol.

Investigators randomized 216 patients (91% women, 94% white) to receive a 10-mg dose daily of OCA, 5 mg titrated to 10 mg if inadequate response, or placebo for 12 months. The primary endpoint was normalization of total bilirubin and reduction in alkaline pho…