FDA Issues Urgent Safety Warning on Ocaliva, Highlights Risk of Serious Liver Injury in Primary Biliary Cholangitis Patients

This update builds upon a previous safety communication from May 26, 2021, which restricted the use of Ocaliva in PBC patients with advanced cirrhosis due to the risk of severe liver-related complications. However, following an in-depth review of postmarket clinical trial data, the FDA has now identified serious liver injuries in patients without cirrhosis as well. Some of these cases have progressed to liver transplantation, emphasizing the severity of this risk.

Ocaliva, approved in May 2016, is a prescription medication that has been shown to improve a liver test called alkaline phosphatase (ALP) in PBC patients who did not respond to the standard treatment, ursodeoxycholic acid (UDCA). Although initial trials showed promising results, a recent postmarket clinical trial revealed a higher incidence of liver-related complications in patients receiving Ocaliva compared to those receiving a placebo. Specifically, risks of liver transplant and death were higher in the treatment group. Among these findings, 7 out of 81 patients receiving Ocaliva needed a liver transplant, compared to just 1 in the placebo group. Additionally, four patients in the Ocaliva group died, compared to just one in the placebo group.

Between May 26, 2021, and September 18, 2024, the FDA identified 20 cases of serious liver complications in patients treated with Ocaliva. These included liver transplants, evaluations for liver transplantation, and liver-related deaths. Notably, three of these cases involved patients for whom Ocaliva should have been discontinued based on the progression of liver disease, underscoring the importance of regular liver monitoring.

The FDA urges healthcare providers to monitor liver tests frequently in patients taking Ocaliva to detect signs of worsening liver function early. Additionally, healthcare providers should discontinue Ocaliva at the first evidence of liver disease progression or if the medication is no longer effective. Patients are advised to contact their healthcare provider immediately if they experience symptoms such as a swollen belly, yellowing of the skin or eyes, bloody or black stools, mental confusion, or other signs of liver injury.

The FDA also encourages patients, caregivers, and healthcare providers to report side effects related to Ocaliva or other medications to the FDA MedWatch program. These reports are vital for ongoing monitoring and ensuring public health and drug safety.

This recent communication reflects the FDA’s commitment to continuously evaluate and communicate risks associated with widely used treatments. Ongoing vigilance is essential to ensure the safety of patients with chronic conditions like PBC.