Obeticholic Acid Withdrawn From US Market for PBC; FDA Puts Clinical Trials on Hold
The US Food and Drug Administration (FDA) has placed a clinical hold on all US clinical trials involving obeticholic acid, and Intercept Pharmaceuticals announced on September 11 that it will voluntarily withdraw its obeticholic acid product, Ocaliva, from the US market for the treatment of primary biliary cholangitis (PBC).
The agency granted accelerated approval to obeticholic acid in 2016 for the treatment of PBC in adults with an inadequate response to or intolerance of ursodeoxycholic acid (UDCA). FDA later restricted the use of obeticholic acid in patients with PBC and advanced cirrhosis of the liver after finding that the drug can cause significant liver injury in this population.
“Based on its review of postmarket clinical trial data, on December 12, 2024, the FDA announced identified cases of serious liver injury among patients being treated for primary biliary cholangitis, or PBC, with Ocaliva, active ingredient obeticholic acid, who did not have cirrhosis of the liver,” the agency stated in its Drug Safety Podcast. “This risk was notably higher for patients taking Ocaliva compared with a placebo,” FDA stated.
Clinicians are advised to conduct frequent liver test monitoring to identify worsening liver function and ensure appropriate discontinuation of obeticholic acid. However, the agency cautioned, “Based on the current data, it is not clear if this monitoring will be sufficient to address the risk of serious liver injury. Discontinue Ocaliva treatment with any evidence of liver disease progression or if efficacy is not established.”
References:
Serious liver injury being observed in patients without cirrhosis taking Ocaliva (obeticholic acid) to treat primary biliary cholangitis. Drug Safety Podcast transcript. US Food and Drug Administration; September 11, 2025. Accessed September 15, 2025. https://www.fda.gov/drugs/fda-drug-safety-podcasts/serious-liver-injury-being-observed-patients-without-cirrhosis-taking-ocaliva-obeticholic-acid-treat
Intercept announces voluntary withdrawal of Ocaliva® for primary biliary cholangitis (PBC) from the US market; UC clinical trials involving obeticholic acid placed on clinical hold. News release. Intercept Pharmaceuticals; September 11, 2025. Accessed September 15, 2025. https://www.interceptpharma.com/about-us/news/?id=3148535
