FDA Observes Serious Liver Injury After Treatment with Ocaliva

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. FDA 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.

The agency’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.

The use of Ocaliva in patients who have PBC with advanced cirrhosis of the liver was restricted by FDA 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, its 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.

FDA urges healthcare professionals and patients to review this new safety information, discontinue Ocaliva treatment with any evidence of liver disease progression or if efficacy is not established and understand signs and symptoms of worsening liver injury in patients receiving Ocaliva. Patients should contact their medical provider immediately if they develop any of the symptoms.

The agency also emphasizes that frequent liver test monitoring is necessary to identify worsening liver function and ensure appropriate discontinuation of Ocaliva. FDA will continue to monitor the medicine’s safety and will follow up if additional information becomes available.

Healthcare professionals and patients should report side effects involving Ocaliva to the FDA MedWatch program.