European Medicines Agency Approves Two Novel Therapies: Hemophilia B Gene Therapy and Enzyme Replacement for Congenital Thrombotic Thrombocytopenic Purpura

The European Medicines Agency has granted conditional marketing authorization for Durveqtix, a gene therapy for severe and moderately severe hemophilia B, and Adzynma, an enzyme replacement therapy for congenital thrombotic thrombocytopenic purpura, addressing unmet medical needs in these rare conditions.