Patients with primary biliary cholangitis (PBC) who do not have a biochemical response to obeticholic acid (OCA) may be at elevated risk of experiencing adverse effects, according to findings recently published in Alimentary Pharmacology & Therapeutics.
In September 2025, OCA, sold as Ocaliva, was taken off the U.S. market by Intercept Pharmaceuticals following reports of liver injury and death. OCA was initially approved in 2016 as a second-line therapy for individuals with PBC who did not respond to ursodeoxycholic acid (UDCA), the main treatment for the disease.
Previous studies have found particular laboratory results that can identify patients at higher risk of side effects from UDCA. Here, the authors demonstrate that those same criteria can be applied to individuals treated with OCA.
Biochemical response was determined by measuring levels of bilirubin, alanine aminotransferase (ALT) and alkaline phosphatase (ALP), three major indicators of liver function.
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The study included 336 individuals with PBC who began OCA at medical centers in the United Kingdom, Italy and Canada between 2017 and 2019. Of the participants, 45% discontinued OCA within two years after they began taking it. Itchiness was the most common reason for discontinuation, followed by declining liver function.
While patients receiving the drug for 12 months had a biochemical response rate of 37%, those who took OCA for 48 months had a response rate of 55%. “Whilst response rates increase over time, discontinuation rates underscore the need for newer treatment paradigms,” the authors emphasized.
Over four years, 64 participants experienced a clinical event, defined as worsening liver function, hepatocellular carcinoma, liver transplant referral or death. Further analysis revealed that patients with cirrhosis and those who did not respond to OCA within one year had a higher risk of experiencing a clinical event.
The authors also assessed 44 patients who received fibrates alongside OCA and 74 patients who switched from OCA to a fibrate. They found that those taking a combination therapy had higher biochemical response rates at four years than those who switched.
“Future studies from our group will continue to explore the effects of OCA in the real-world setting, including combination treatment approaches with elafibranor and seladelpar, alongside other therapies that are on the horizon for PBC,” the authors concluded.
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