Originally published by our sister publication Gastroenterology & Endoscopy News

The FDA has issued a warning of serious liver injury for patients taking obeticholic acid (Ocaliva, Intercept) for primary biliary cholangitis, even those without cirrhosis. This notice follows a 2021 contraindication of the medication for patients with PBC and advanced cirrhosis.

“Healthcare professionals should monitor liver tests frequently for patients taking obeticholic acid to detect and address worsening liver function early,” the FDA wrote in a news release, adding that providers should advise patients taking obeticholic acid to monitor and immediately report specific symptoms such as a swollen abdomen, jaundice, bloody or black stools, or mental status changes. General symptoms such as stomach pain, nausea, vomiting or diarrhea, loss of appetite or weight loss, new or worsening tiredness, weakness, fever and chills, lightheadedness, and less frequent urination should also be reported if they persist longer than a few days. 

The drug should be discontinued immediately on any evidence of liver disease progression or if efficacy is not established, the FDA advised.

Obeticholic acid was granted accelerated approval in 2016 for patients with PBC who didn’t respond to ursodeoxycholic acid because it was found to lower alkaline phosphatase. 

The FDA reported that since they restricted use of obeticholic acid for patients with advanced cirrhosis in 2021, they identified 20 cases reported to the FDA Adverse Event Reporting System (FAERS), including seven patients requiring liver transplant, eight instances where a patient was evaluated or listed for transplant, and six liver-related deaths. The FDA noted its recent review of FAERS indicated some patients with PBC and advanced cirrhosis are continuing to take the medication.

—GEN Staff

Based on an FDA press release.