Ocaliva Liver Side Effects Are Higher for Patients Who Didn’t Respond to Drug: Study

Study adds new information about the potential liver risks after an Ocaliva recall was initiated last month, following a determination that the potential side effects outweigh the benefits provided by the treatment.

A new study indicates that patients who experienced the most severe liver injuries from the recently recalled drug Ocaliva were also the least likely to respond to its treatment benefits.

Ocaliva (obeticholic acid) is an Intercept Pharmaceuticals drug approved for the treatment of primary biliary cholangitis (PBC), a rare autoimmune disease that destroys the liver’s bile ducts over time. The ducts become inflamed and eventually collapse, causing liver damage that can eventually result in cirrhosis.

After the drug was approved through an accelerated approval program in 2006, postmarketing studies and adverse event reports have linked Ocaliva side effects to severe liver injury and liver failure risks, which have resulted in a series of Ocaliva warnings over the past few years. 

The FDA first issued a safety communication about Ocaliva dosing errors in September 2017, and by February 2018, it required a black box warning with updated dosing and monitoring instructions, followed by additional use restrictions and warnings

Last year, the agency decided against full permanent approval of Ocaliva after a scientific advisory board voted against it. The FDA then issued another Ocaliva warning in December 2024, indicating that the drug has been linked to a 377% increased risk of needing a liver transplant or dying, when compared to those who did not take the drug.

Following a request from the agency, Intercept announced an Ocaliva recall last month, indicating federal regulators had determined the drug’s benefits did not outweigh its risks.

On September 22, researchers with the U.K.’s National Institute for Health and Social Rare Research (NIHR) published a study in Alimentary Pharmacology and Therapeutics, reporting that it was patients who had a biochemical non-response, meaning their disease did not react to the treatment, who faced extremely high risks of liver injury and cirrhosis from Ocaliva.

The researchers collected data from 336 patients who started using Ocaliva between August 2017 and 2019, and followed up with those patients until June 2024, looking at biochemical non-response and clinical events, which included hepatic decompensation, need for liver transplantation, liver cancer and death.

According to the findings, 64 of those patients suffered clinical events over a four-year period. The researchers determined that if a patient showed no biological response to the drug, meaning their PBC was not improving, they were four and a half times more likely to suffer a clinical event, and 20 times more likely to develop cirrhosis. They also had more than double the risk of hyperbilirubinaemia, which can cause jaundice.

The researchers noted response rates did increase over time. However, that was often too late, with Ocaliva liver side effects and other factors resulting in a 45% discontinuation rate over those same four years.

“Whilst response rates increase over time, discontinuation rates underscore the need for newer treatment paradigms.”

– National Institute for Health and Social Rare Research (NIHR), Non-Response to Obeticholic Acid Is Associated With Heightened Risks of Developing Clinical Events in Primary Biliary Cholangitis

The study was published just 11 days after the Ocaliva recall was announced due to potential liver side effects.  

With the complete removal of the drug from the market expected to be completed later this year, Intercept is recommending patients prescribed Ocaliva consult with their healthcare providers before making any medication changes. The company indicates it will provide additional information as it works through the transition process with the FDA.

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.

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