FDA finds serious liver injury observed in patients without cirrhosis patients taking Ocaliva

Based on its review of post-market clinical trial data, the US Food and Drug Administration (FDA) identified cases of serious liver injury among patients being treated for primary biliary cholangitis (PBC) with Ocaliva (obeticholic acid) who did not have cirrhosis of the liver.

Ocaliva is marketed by Intercept Pharmaceuticals, which was acquired for around $800 million by Italy’s Alfasigma last year. Last month, the FDA has issued a Complete Response Letter (CRL) that addresses the supplemental New Drug Application (sNDA) for Ocaliva seeking full approval for the treatment of indicated patients with PBC. The drug gained FDA accelerated approval for PBC in 2016. Ocaliva was also pulled off the European market last month.

The FDA said it has previously identified that PBC patients with advanced cirrhosis were at risk of serious liver injury when taking Ocaliva and updated the prescribing information to restrict its use in these patients. The FDA’s review of this required clinical trial found that some cases of liver injury in patients without cirrhosis resulted in liver transplant. This risk was notably higher for patients taking Ocaliva compared with a placebo, a pill without any active medicine.