FDA Raises Safety Concerns with Ocaliva Amid Liver Injury Risks and Failed Approvals

Ocaliva, developed by Intercept Pharmaceuticals, has faced a tumultuous journey marred by significant safety concerns raised by the FDA. Originally approved for primary biliary cholangitis (PBC) in 2016, the drug has been linked to serious liver injury cases, prompting rigorous scrutiny and a recent advisory committee downvote[1][2]. Attempts to broaden its application to metabolic dysfunction-associated steatohepatitis (MASH) resulted in failures and subsequent program abandonment in 2020 and 2023. This led to significant setbacks, including staff reductions and a Complete Response Letter detailing safety issues[1][2]. The FDA's latest warnings reiterate the need for vigilant liver function monitoring for patients, highlighting the critical balance between Ocaliva's potential therapeutic benefits and its considerable risks[2].