Advanz Pharma scored a win for its liver disease treatment Ocaliva after the General Court of the European Union temporarily suspended the European Commission’s decision to revoke the conditional marketing authorization for the treatment.
The drug, which is approved for the rare liver disease primary biliary cholangitis — a condition that causes the body’s immune system to attack and injure bile ducts — will continue to be available “until further notice,” according to the company.
“This enables immediate continuity of supply until the next decision by the General Court,” Advanz CEO Steffen Wagner said in a statement. “Ocaliva — an FXR agonist — has a different mechanism of action from other treatments, making it an important option for patients with PBC, a condition where treatment options are already very limited.”
The decision from the general court comes just a few days after the European Commission took away the drug’s authorization following a June recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use, which reassessed the benefit-risk profile of the drug.
Ocaliva is also facing upcoming competition in the US and in Europe. Gilead secured accelerated approval for Livdelzi (seladelpar) in August, as did Ipsen’s treatment Iqirvo.
Following the EC’s decision, Leerink Partners analysts estimated that in 2024, there are around 100,000 patients in the EU living with PBC and about 130,000 patients in the US. The analysts added in a note that they expect EMA approval for Iqirvo in the third quarter of this year and Livdelzi in early 2025 in PBC, “and see both drugs as clearly more effective in driving biochemical response and generally better tolerated than Ocaliva.”
Alfasigma, which has the US rights to the drug, is also facing an FDA adcomm next week to discuss the full approval. Ocaliva won an accelerated approval to treat PBC in 2016. Since its approval, the FDA added a black box warning after it was being incorrectly dosed as a daily treatment as opposed to a weekly treatment, potentially increasing the risk for more severe liver damage. The treatment has also failed to expand into other indications.