FDA Warns of Ocaliva Liver Injury Risks Linked to Autoimmune Disorder Drug

Federal regulators rejected full approval of Ocaliva in November following an analysis of post-marketing data by a committee of scientific advisors.

The findings of a new study indicate that patients taking the autoimmune liver disease drug Ocaliva faced a nearly 400% increased risk of developing liver disease.

The U.S. Food and Drug Administration (FDA) issued a drug safety communication on December 12, warning that Ocaliva can lead to serious liver injury among patients who do not already have liver disease and increases the likelihood of needing a liver transplant and death.

Ocaliva (obeticholic acid) is an Intercept Pharmaceuticals drug used to treat patients with primary biliary cholangitis, which is an autoimmune disease that causes inflammation and scarring in the liver’s bile ducts. Over time, this damage can lead to progressive liver dysfunction, cirrhosis, and potentially liver failure, making early intervention and appropriate therapy critical for patient outcomes.

It is estimated that roughly 9,000 individuals across the U.S. are diagnosed with primary biliary cholangitis, with the disease predominantly affecting middle-aged women. While the exact cause of PBC remains unclear, genetic predisposition and environmental factors are thought to play a role in triggering the autoimmune response.

Ocaliva Liver Injury Concerns

Federal regulators approved Ocalivain through an accelerated approval process in 2015,  to be used in combination with ursodeoxycholic acid (UDCA) or as a second-line therapy for those who have had an inadequate response to UDCA alone, aiming to slow disease progression and improve liver function over time.

However, the FDA required Intercept Pharmaceuticals to conduct postmarketing clinical trials to further evaluate the drug’s long-term safety and effectiveness. As data from these ongoing studies and real-world patient experiences began to emerge, the agency issued multiple warnings regarding the potential for serious liver injury associated with Ocaliva in patients being treated for primary biliary cholangitis.

The FDA first issued a warning regarding the risk of serious liver injury with Ocaliva in September 2017. In early 2018, the FDA required the addition of a boxed warning, the most prominent type of FDA warning, to Ocaliva’s prescribing information to highlight these severe risks.

Another official warning focusing on liver injury was released by the FDA in May 2021, reminding healthcare professionals to adhere strictly to the recommended dosing, especially in patients with more advanced disease.

According to the warnings and documented side effects of liver disease progression, once a person begins to suffer from liver failure, they may begin to experience other symptoms like a build-up of fluid in the brain, seizures, coma, bleeding disorders, fluid retention and liver cancer.

As a result of the mounting Ocaliva side effects reported throughout the postmarketing studies, the FDA decided against giving Ocaliva full approval, after a scientific advisory committee voted against it, according to a November 12 press release by Intercept.

According to the FDA, postmarketing data showed that patients taking Ocaliva face a significantly higher risk of needing a liver transplant or dying compared to those taking a placebo, according to the FDA.

Among 81 Ocaliva patients considered at lower initial risk, seven ultimately required a liver transplant, while only one of the 68 placebo patients did. Notably, that single placebo patient had switched to Ocaliva for two years before needing the transplant, suggesting Ocaliva’s role in the injury.

Overall, the data indicates patients taking Ocaliva face a 377% increased risk of needing a liver transplant or dying. A total of four patients taking Ocaliva have died, compared to one patient in the placebo group.

FDA Ocaliva Recommendations

The FDA indicates doctors should conduct frequent liver tests and monitor the results to identify liver disease early and worsening liver function in patients taking Ocaliva. However, researchers said it is unclear if monitoring is sufficient to prevent serious liver injury.

The agency also recommends doctors explain the signs and symptoms of liver injury to Ocaliva patients, including symptoms such as yellow eyes or skin, bloody or black stool, coughing up or vomiting blood, change in mental status like confusion, slurred speech, increased sleepiness, stomach pain, nausea, vomiting, diarrhea, fever, weakness, and frequent urination.

The FDA requests any side effects linked to taking Ocaliva, such as liver disease and liver failure, should be reported to the FDA’s MedWatch Adverse Event Reporting Program.

2025 Ocaliva Recall Lawsuit Update

On September 11, 2025, Intercept Pharmaceuticals withdrew Ocaliva from the U.S. market at the FDA’s request, following years of safety concerns that began with a Boxed Warning in February 2018 over fatal dosing errors. The withdrawal came after mounting reports of liver failure in patients who should never have received standard dosing due to impaired liver function.

For former users left with liver injuries, lawyers are now investigating the potential for Ocaliva lawsuits, which may help obtain financial compensation from the drug makers, for failing to adequately warn about the potential side effects of Ocaliva.