FDA Restricts Use of Obeticholic Acid Among Patients With PBC

The U.S. Food and Drug Administration (FDA) on May 28 announced restrictions on the use of obeticholic acid among patients with primary biliary cholangitis (PBC) and advanced cirrhosis of the liver after some patients developed liver failure, in some cases requiring liver transplantation.

The agency stated, “Based on the original clinical trials, FDA believes the benefits of Ocaliva outweigh the risks for PBC patients who do not have advanced cirrhosis. We will continue to monitor and evaluate the clinical benefit and adverse events of Ocaliva and will communicate any new information to the public if it becomes available.”

A new Contraindication has been added to the prescribing information and patient Medication Guide for obeticholic acid, warning against its use to treat patients with PBC and advanced cirrhosis. The FDA said it had also revised the Boxed Warning for the drug to include this information along with related warnings about this risk.

Obeticholic acid was approved in May 2016 and has been shown to improve alkaline phosphatase (ALP) among patients who did not respond to ursodeoxycholic acid (UDCA). Clinical trials for obeticholic acid suggested survival and disease-related symptoms may be improved by this decrease in ALP.

—Rebecca Mashaw

Due to risk of serious liver injury, FDA restricts use of Ocaliva in primary biliary cholangitis (PBC) patients with advanced cirrhosis. Rockville, MD: US Food and Drug Administration; May 28, 2021. https://www.fda.gov/drugs/drug-safety-and-availability/due-risk-serious-liver-injury-fda-restricts-use-ocaliva-primary-biliary-cholangitis-pbc-patients