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FDA Restricts Use of Ocaliva in Primary Biliary Cholangitis Patients with Advanced Cirrhosis

MedWatch - The FDA Safety Information and Adverse Event Reporting Program

AUDIENCE: Patient, Health Professional, Pharmacy ISSUE: The FDA is restricting the use of the liver disease medicine Ocaliva (obeticholic acid) in patients having primary biliary cholangitis (PBC) with advanced cirrhosis of the liver because it can cause serious harm. Some PBC patients with cirrhosis who took Ocaliva, especially those with evidence of advanced cirrhosis, developed liver failure, sometimes requiring liver transplant. In the five years since Ocaliva’s accelerated approval, FDA identified 25 cases of serious liver injury leading to liver decompensation or liver failure associated with Ocaliva in PBC patients with cirrhosis, both in those without clinical signs of cirrhosis (compensated) or in those with clinical signs of cirrhosis (decompensated). Many of these PBC patients had advanced cirrhosis before starting Ocaliva. FDA added a new Contraindication, FDA’s strongest warning, to the Ocaliva prescribing information and patient Medication Guide stating that Ocaliva should not be used in PBC patients with advanced cirrhosis. FDA also revised the Boxed Warning, our most prominent warning, to include this information along with related warnings about this risk. Based on the original clinical trials, FDA believes the benefits of Ocaliva outweigh the risks for PBC patients who do not have advanced cirrhosis. FDA 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. For more information about this Drug Safety Communication, click on the red button "Read Alert" below. BACKGROUND: PBC is a rare, chronic disease affecting the ducts in the liver that carry bile, which helps with digestion. RECOMMENDATIONS: Patients:

  • Patients with PBC who have cirrhosis and are taking Ocaliva should talk to your health care professional about these new warnings. Contact your prescriber immediately if you develop any symptoms, which may be signs of worsening liver injury or development of advanced cirrhosis.

Health Care Professionals:

  • Health care professionals should determine before starting Ocaliva whether a patient with PBC has advanced cirrhosis as the medicine is contraindicated in these patients. Advanced cirrhosis is defined as cirrhosis with current or prior evidence of hepatic decompensation (e.g., encephalopathy, coagulopathy) or portal hypertension (e.g., ascites, gastroesophageal varices, persistent thrombocytopenia).

  • Routinely monitor patients during Ocaliva treatment for progression of PBC with laboratory and clinical assessments to determine whether the medicine needs to be discontinued. Permanently discontinue Ocaliva in patients with cirrhosis who progress to advanced cirrhosis.

  • Also monitor patients for clinically significant liver-related adverse reactions that may manifest as development of acute-on-chronic liver disease with nausea, vomiting, diarrhea, jaundice, scleral icterus, and/or dark urine. Permanently discontinue Ocaliva in patients developing these symptoms.

Health professionals and patients are encouraged to report adverse events or side effects related to the use of these products to the FDA's MedWatch Safety Information and Adverse Event Reporting Program:

  • Complete and submit the report online.

  • Download form or call 1-800-332-1088 to request a reporting form, then complete and return to the address on form, or submit by fax to 1-800-FDA-0178.

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