Ocaliva: EC Withdrawal of Approval Temporarily Suspended

The European Commission (EC) revoked the conditional marketing authorization (CMA) of Advanz Pharma’s Ocaliva (obeticholic acid) in Europe for the second-line treatment of patients with primary biliary cholangitis (PBC) in September 2024. However, Advanz Pharma has secured a temporary suspension of the EC’s decision, allowing Ocaliva to remain on the market for now. The EC’s decision to revoke the Ocaliva marketing authorization has been suspended with immediate effect by Order of the President of the General Court of the European Union, as per a statement issued by Advanz Pharma.

Ocaliva is indicated for the treatment of Primary Biliary Cholangitis (PBC), a chronic liver disease and autoimmune condition in which there is a gradual destruction of the bile ducts in the liver. This may lead to scarring and liver failure, and may increase the risk of liver cancer. Ocaliva is available in tablet form and has a recommended starting dose of 5 mg once daily. Ocaliva was approved by the EC in December 2016.

The EC’s decision on Ocaliva followed a recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in June 2024. The CHMP recommended revoking the Marketing Authorization of Ocaliva because its benefits are no longer considered to outweigh its risks.

However Advanz Pharma, the MA holder for Ocalvia said it disagreed with the EC decision. It leaves thousands of patients with PBC who are responding well to Ocalvia at increased risk of disease progression. Advanz Pharma argues that the EC decision did not adequately consider the totality of available data supporting the efficacy and safety of Ocalvia and the wealth of positive real-world evidence (RWE) from more than seven years of clinical use. Advanz Pharma said it is considering all options to ensure continued access to Ocalvia for patients who need it.

Related readings:

• EMA recommendation revoking conditional marketing authorisation for Ocaliva
• ADVANZ PHARMA’s response to European Commission revocation of MA for Ocaliva
• Temporary suspension of the EC decision on Ocaliva