What does Ocaliva treat?

Key Points

• Ocaliva (generic name: obeticholic acid) is used in certain patients to treat primary biliary cholangitis (PBC), previously known as primary biliary cirrhosis.
• The most recent Ocaliva label (dated 5/2022) states 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 (is contraindicated) 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.

Updated liver warnings for Ocaliva

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.

In Dec. 2024, the FDA issued an update to the May 2021 warning to include a post-marketing report that identified cases of serious liver injury among patients being treated for primary biliary cholangitis (PBC) with Ocaliva who did NOT have cirrhosis of the liver. Some of these cases resulted in liver transplant.

Healthcare providers should:

• Be aware that frequent liver test and clinical monitoring is necessary to identify worsening liver function and ensure appropriate discontinuation of Ocaliva. Based on the current data, it is not clear if this monitoring will be sufficient to address the risk of serious liver injury.
• Explain the signs and symptoms of worsening liver injury to patients receiving Ocaliva and direct them to contact you immediately if they develop any signs or symptoms of worsening liver injury.
• Discontinue Ocaliva treatment with any evidence of liver disease progression or if efficacy is not established.

Patients should:

Discuss the safety risk and the benefits of continuing treatment with Ocaliva with their healthcare providers. Discuss any concerns you may have, including about possible alternative treatments.

Contact their health care professional immediately if they develop any of the following symptoms which may indicate worsening liver injury.

• Swollen bell
• Yellow eyes or skin
• Bloody or black stools
• Coughing up or vomiting blood
• Mental status changes such as confusion, slurred speech, mood swings, changes in personality, or increased sleepiness or difficulty waking up

Any of these general symptoms if they are severe or do not go away after a few days:

• Belly pain
• Nausea, vomiting, or diarrhea
• Loss of appetite or weight loss
• New or worsening tiredness
• Weakness
• Fever and chills
• Lightheadedness
• Less frequent urination

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.

*Ocaliva was approved under FDA accelerated approval based on a reduction in alkaline phosphatase (ALP), one indicator of liver function. An improvement in survival or disease-related symptoms has not been established in studies. Continued approval for this use may depend upon verification and description of clinical benefit in additional clinical trials.

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 your healthcare provider's instructions. Review the full product information and discuss this information and any questions you have with your doctor or other health care provider.