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

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.