Previously, Ocaliva (obeticholic acid) was approved as a prescription treatment for patients with primary biliary cholangitis (PBC), a rare and chronic liver disease. However, Market Screener reports that biopharmaceutical company Intercept Pharmaceuticals, Inc. (“Intercept”) recently updated the Ocaliva Prescribing Information to show that the treatment is contraindicated for patients with PBC and decompensated cirrhosis, prior decompensation, or compensated cirrhosis with portal hypertension.

Ocaliva

In 2016, the FDA approved Ocaliva, a farnesoid X receptor (FXR) agonist, as a monotherapy for adult patients with PBC without cirrhosis, or patients with compensated cirrhosis without portal hypertension. The FDA approval also suggested that Ocaliva could be used in conjunction with ursodeoxycholic acid for patients.

Let’s break this down. Compensated cirrhosis means that some liver scarring is evident but the liver still functions fairly well. Additionally, compensated cirrhosis means that a patient is still somewhat asymptomatic. Next, portal hypertension means high blood pressure (hypertension) within the portal vein, which brings blood from digestive organs to the liver. Both decompensated cirrhosis, and portal hypertension, mean that a patient’s PBC is progressing and worsening.

Ocaliva Prescribing Information

Altogether, the prescribing information was changed based on information submitted to the FDA’s Adverse Event Reporting System. This data was later published, highlighting how patients with PBC and cirrhosis, who were later treated with Ocaliva, saw worse liver function, increased liver damage, and, in some cases, liver failure.

Thus, Ocaliva is now contraindicated for patients with PBC with cirrhosis, or patients who have decompensated cirrhosis or portal hypertension. Additionally, the added warning and precaution information should help doctors identify patients who could be at risk of hepatic decompensation.

Generally, many patients with PBC should still be able to receive treatment. However, it is important to protect patient health and improve patient outcomes, so understanding the prescribing changes is crucial for medical professionals.

Adverse Reactions

Common side effects associated with Ocaliva treatment include:

• Abdominal, throat, and joint pain
• Rashes
• Fatigue
• Dizziness
• Constipation
• Eczema
• Pruritus (intense itching)
• Changes in thyroid function

However, patients experiencing symptoms related to hepatic decompensation, such as worsening jaundice (yellowing of the skin and eyes), thrombocytopenia (low platelet count), or abdominal swelling, should report these symptoms to:

• Intercept
  • 1-844-782-ICPT
• FDA
  • www.fda.gov/medwatch
  • 1-800-FDA-1088

Patients experiencing hepatic decompensation or liver failure were seen in patients taking 10-50mg Ocaliva. In many cases, symptoms appeared within 1-4 months of treatment.

Primary Biliary Cholangitis (PBC)

PBC is characterized by slow, progressive bile duct damage. Normally, the liver produces bile, which plays a role in fat and fat-soluble vitamin digestion. But when the bile ducts are damaged, bile is unable to properly flow out of the liver. As a result, bile accumulation causes scarring (fibrosis) and inflammation. As the condition progresses, PBC can result in cirrhosis (advanced scarring) and liver failure. Risk factors include being female, being between 30-60 years old, smoking, or having a prior history of PBC.

In the early stages, patients may experience mild symptoms, such as:

• Severe fatigue
• Dry eyes and mouth
• Pruritus (intense itching), particularly on the bottom of the feet

In later stages of PBC, symptoms include:

• Jaundice
• Chronic diarrhea
• Underactive thyroid
• Xanthomas (fatty deposits) under the skin, especially around the eyes
• Bone, muscle, and joint pain
• Abdominal distention due to fluid (ascites)
• Hyperpigmentation (skin darkening)
• Swelling of the lower extremities
• Unintended weight loss
• Internal bleeding in the throat and stomach
• Bone fragility