Latest news about obeticholic acid in 2025

Based on a review of postmarketing clinical trial data, the U.S. Food and Drug Administration (FDA) announced on December 12, 2024, cases of severe liver injury in patients with primary cholangitis (PBC) who were treated with obeticholic acid but had no history of cirrhosis. Previous studies have shown that patients with advanced cirrhosis are at risk for severe liver injury when taking obeticholic acid, so the FDA has updated prescribing information to limit the drug's use in these patients.

A recent FDA review found that some cases of liver injury led to liver transplantation, even in patients without cirrhosis, and that this risk was significantly increased in patients treated with obeticholic acid compared with placebo. Based on these findings, the FDA has limited the application scope of obeticholic acid in patients with advanced cirrhosis in 2021, adding a new contraindication, warning that it may cause serious harm to such patients.

Despite the restrictions, a recent review of case reports submitted to the FDA found that some patients with PBC and advanced cirrhosis are still using the drug. Therefore, the FDA reminds health care professionals and patients to pay attention to this new safety information and emphasizes the importance of frequent liver testing to promptly identify worsening liver function and ensure that obeticholic acid is discontinued if necessary. It is unclear whether existing surveillance measures are sufficient to prevent the development of severe liver injury. If any evidence of progression of liver disease is found, or if symptoms do not improve, use of this drug should be discontinued. At the same time, medical staff need to explain the symptoms of worsening liver damage to patients and instruct them to seek medical treatment immediately when relevant symptoms occur.

In its liver safety assessment of patients eligible for obeticholic acid treatment, the FDA found that patients receiving obeticholic acid had a higher risk of liver transplantation and death than those receiving placebo. Seven of the 81 patients who received obeticholic acid required a liver transplant, compared with only one of the 68 patients who received a placebo. In addition, four patients who received obeticholic acid died, compared with one in the placebo group. This further emphasizes the importance of continued monitoring of liver health and immediate discontinuation of the drug if there are signs of progression of cirrhosis.

References:https://www.fda.gov/drugs/fda-drug-safety-podcasts/serious-liver-injury-being-observed-patients-without-cirrhosis-taking-ocaliva-obeticholic-acid-treat