The FDA has issued a safety warning for Ocaliva (obeticholic acid), a prescription drug used to treat a chronic liver condition called primary biliary cholangitis (PBC). New findings show that Ocaliva can cause serious liver injury, even in patients without advanced liver disease (cirrhosis).

Why the FDA Issued This Warning

Recent clinical trial data revealed:

• Higher risks of liver transplants and deaths in patients taking Ocaliva compared to those on a placebo.
  • 7 out of 81 patients on Ocaliva needed a liver transplant.
  • 4 deaths were reported in the Ocaliva group, compared to just 1 death in the placebo group.

The risk of serious liver complications was found to be significantly higher, even for those without severe liver disease.

In 2021, the FDA had already restricted Ocaliva for patients with advanced cirrhosis due to similar safety concerns. Despite these restrictions, reports show that some patients with severe liver disease continued to take the medication.

What Patients and Healthcare Providers Need to Know

The FDA is now urging healthcare providers to:

• Monitor liver tests frequently in patients taking Ocaliva.
• Stop Ocaliva immediately if there are signs of worsening liver function or if it’s not effective.
• Educate patients about symptoms of liver injury and the need to seek immediate care.

Symptoms of Liver Injury

Patients should contact their healthcare provider immediately if they experience any of the following specific symptoms:

• Swollen belly
• Yellow skin or eyes (jaundice)
• Black or bloody stools
• Coughing up or vomiting blood
• Confusion, slurred speech, mood swings, personality changes, or extreme drowsiness

Additionally, general symptoms of liver problems, if severe or persistent, include:

• Abdominal pain
• Nausea, vomiting, or diarrhea
• Loss of appetite or weight loss
• Fatigue or weakness
• Fever and chills
• Lightheadedness
• Urinating less frequently

Background on Ocaliva

Ocaliva was approved in 2016 for PBC patients who do not respond to other treatments like ursodeoxycholic acid (UDCA). While it was initially approved based on improved liver test results, the recent data raises concerns about its safety.

FDA Findings in Detail

The FDA reviewed data from postmarket clinical trials and found:

• In patients eligible for Ocaliva, the risk of liver transplants and deaths was higher compared to placebo users.
• Patients who experienced these complications had not been diagnosed with advanced cirrhosis at the start of treatment.
• Despite 2021 restrictions, the FDA identified 20 new cases of liver-related complications (transplants or deaths) between May 2021 and September 2024.

The FDA emphasizes the importance of ongoing liver monitoring to detect early signs of damage and urges immediate discontinuation of Ocaliva if liver disease worsens.

What Patients Should Do

If you are taking Ocaliva:

• Talk to your doctor about this safety warning and whether the benefits still outweigh the risks.
• Be aware of the symptoms listed above and report any to your healthcare provider immediately.
• Ask about potential alternative treatments if you have concerns.

Report Side Effects

If you or someone you know experiences side effects while taking Ocaliva, report them to the FDA’s MedWatch program:

• Online: MedWatch Reporting
• Phone: Call 1-800-332-1088 to request a form and submit it by mail or fax.

For more information, read the full FDA Drug Safety Communication here.