BioMarin Pulls EU Hemophilia Gene Therapy Filing, But Is Not Giving Up

Agios’s MAA For Tibsovo also withdrawn

BioMarin plans to resubmit its marketing application for Roctavian to the European Medicines Agency.

Gene therapy
Several gene therapies for hemophilia are under development • Source: Shutterstock

BioMarin Pharmaceutical has withdrawn its EU marketing authorization application (MAA) for Roctavian (valoctocogene roxaparvovec), the gene therapy it is developing for severe hemophilia A that also recently suffered a setback in the US.

The company has pulled the filing because it cannot provide the data that the European Medicines Agency has asked for during the current regulatory review of the MAA

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