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Serious Liver Injury Being Observed In Patients Without Cirrhosis Taking Ocaliva (obeticholic acid) to Treat Primary Biliary Cholangitis

Based on its review of postmarket clinical trial data, the U.S. Food and Drug Administration (FDA) identified cases of serious liver injury among patients being treated for primary biliary cholangitis (PBC) with Ocaliva (obeticholic acid) who did not have cirrhosis of the liver. We previously identified that PBC patients with advanced cirrhosis were at risk of serious liver injury when taking Ocaliva and updated the prescribing information to restrict its use in these patients. FDA’s review of this required clinical trial found that some cases of liver injury in patients without cirrhosis resulted in liver transplant. This risk was notably higher for patients taking Ocaliva compared with a placebo, a pill without any active medicine. 

FDA restricted the use of Ocaliva in patients who have PBC with advanced cirrhosis of the liver in 2021 because it can cause serious harm in those patients, adding a new Contraindication to the Ocaliva prescribing information and patient Medication Guide. However, our recent review of case reports submitted to FDA* found that some patients with PBC and advanced cirrhosis were still taking the medicine despite these restrictions.

We are notifying health care professionals and patients of this new safety information, and that frequent liver test monitoring is necessary to identify worsening liver function and ensure appropriate discontinuation of Ocaliva. The agency will continue to monitor the medicine’s safety and will follow up if additional information becomes available.

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