What does Ocaliva treat?

• Ocaliva (generic name: obeticholic acid) is used in certain patients to treat primary biliary cholangitis (PBC), previously known as primary biliary cirrhosis.
• It is used in adults without cirrhosis OR with compensated cirrhosis who do not have evidence of portal hypertension.
• Ocaliva is given either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA or as a single agent (monotherapy) in patients unable to tolerate UDCA.
• It is NOT used in patients with decompensated cirrhosis or a prior decompensated event, compensated cirrhosis with evidence of portal hypertension (for example, ascites, gastroesophageal varices, persistent thrombocytopenia), or complete biliary obstruction.

Primary biliary cholangitis (PBC) is a long-lasting and rare disease that causes the small bile ducts in the liver to become inflamed and destroyed. This causes bile to build-up in the liver, which damages the liver cells over time. Cirrhosis, or scarring of the liver occurs, and the liver eventually loses its ability to function.

Symptoms of cholestasis include:

• itching (pruritus)
• fatigue (extreme tiredness)
• dark-colored urine
• stomach pain
• yellowing of the eyes or skin (jaundice)
• pale-colored stools.

Ocaliva is classified as a first-in-class farnesoid X receptor (FXR) agonist for the treatment of PBC. FXR is a nuclear receptor expressed in the liver and intestine and a key regulator of bile acid, inflammatory, fibrotic and metabolic pathways.

Ocaliva was given accelerated FDA approval in May 2016 and was the first new treatment for PBC in nearly 20 years.

In May 2021, the FDA updated the Boxed Warning and restricted the use of Ocaliva in patients with PBC with advanced cirrhosis due to an increased risk of serious liver injury.

How is Ocaliva used?

Ocaliva is an oral pill that can be taken with or without food. Your doctor will determine your dose and schedule for taking Ocaliva based on your liver function. Follow your doctor's dosing instructions exactly.

Ocaliva can be used alone or with ursodeoxycholic acid (UDCA) in adults with an inadequate UDCA response or unable to tolerate UDCA. UDCA was approved by the FDA in 1997 but not all patients respond to this treatment.

This is not all the information you need to know about Ocaliva (obeticholic acid) for safe and effective use and does not take the place of talking to your doctor about your treatment. Review the full Ocaliva information here, and discuss this information and any questions you have with your doctor or other health care provider.