FDA Warns of Serious Liver Injury With Obeticholic Acid Among Patients With PBC Without Cirrhosis 

The  U.S. Food and Drug Administration (FDA) said on December 12 that its review of postmarketing clinical trial data identified cases of serious liver injury among patients being treated for primary biliary cholangitis (PBC) with obeticholic acid who did not have cirrhosis of the liver. The agency's review of this required clinical trial found that some of these cases resulted in liver transplant. The risk of serious liver injury was notably higher for patients taking obeticholic acid compared with a placebo.

In 2021, FDA restricted the use of obeticholic acid in patients who have PBC with advanced cirrhosis of the liver and added a new contraindication to the drug's 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," the agency stated in its safety communication.

"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 statement continued.

FDA further instructed clinicians to monitor liver tests frequently for their patients being treated with obeticholic acid, but cautioned that "it is not clear if this monitoring will be sufficient to address the risk of serious liver injury."