Ocaliva Faces FDA Scrutiny for Serious Liver Injury and Approval Denials

Ocaliva, developed by Intercept Pharmaceuticals, initially secured FDA approval in 2016 for the treatment of primary biliary cholangitis (PBC), a rare liver condition. However, the drug has faced significant challenges due to safety concerns, including serious liver injury and a higher likelihood of death or liver transplantation in patients treated with Ocaliva[1][2]. These safety risks have led to failed attempts to expand its approved uses, notably for metabolic dysfunction-associated steatohepatitis (MASH), resulting in a program halt and workforce reduction at Intercept[1]. Despite efforts to address these concerns, the drug continues to face scrutiny, unable to secure traditional approval, while also having its marketing authorization revoked by European authorities as new alternatives enter the market[1][2].