Ursodiol

Advanz said it is considering next steps, including options to enable continued access for patients. Credit: Yavdat via Shutterstock. Advanz Pharma’s liver disease drug Ocaliva (obeticholic acid) will no longer be available in Europe after the General Court of the European Union upheld a decision by the European Commission (EC) to revoke its conditional marketing authorisation (CMA). Developed by UK pharmaceutical company Advanz Pharma, the drug originally received conditional marketing authorisation (CMA) in 2016, allowing it to be prescribed to patients with primary biliary cholangitis (PBC) while additional data was collected. However, in June 2024, the European Medicines Agency (EMA) reassessed the drug’s benefit-risk profile and recommended revocation of its CMA. Advanz Pharma temporarily secured a suspension of the EC’s decision last month while pursuing legal action to annul the revocation. However, the General Court has declined to extend the suspension, meaning Ocaliva is now fully withdrawn from the European Union and European Economic Area markets. While Advanz Pharma continues to challenge the EC’s decision, with a final ruling expected in 2025, the revocation is effective immediately. The revocation applies to all European Union member states and European Economic Area countries, including Iceland, Liechtenstein, and Norway. Ocaliva has been previously shown to reduce the blood levels of alkaline phosphatase (ALP) and bilirubin in patients with PBC – indicating an improvement in liver condition. However, the CHMP concluded that the drug was no more effective than a placebo after evaluating data from a confirmatory trial (NCT02308111) alongside real-world data. A similar stance has been echoed by the US Food and Drug Administration (FDA). The drug was granted accelerated approval in 2016, but a black box warning was added two years later after reports of severe liver injury linked to incorrect dosing. This month, the FDA denied full approval for the drug following a recommendation from its advisory committee , which also concluded that the benefits did not outweigh the risks. Advanz acquired the drug’s rights in Europe after acquiring non-American subsidiaries of Intercept Pharmaceuticals , which developed the drug, for an upfront payment of $405m in July 2022. Intercept, which still has the US rights, said Ocaliva will remain on the US market under accelerated approval and is talking with the FDA about what’s next. PBC is a rare progressive autoimmune condition that damages the bile ducts in the liver. Ocaliva functions as a selective and potent farnesoid X receptor (FXR) agonist, regulating bile acid and metabolic pathways to improve patient outcomes. It has been prescribed in combination with ursodeoxycholic acid (UDCA) for patients with inadequate UDCA responses or as a standalone therapy for those unable to tolerate UDCA.