• FDA grants breakthrough therapy designation to Helimbra for hemophilia A without inhibitors

    11 monthes ago - By Healio

    The FDA granted breakthrough therapy designation to emicizumab-kxwh for the treatment of patients with hemophilia A without factor VIII inhibitors, according to the agent's manufacturer.
    Emicizumab-kxwh (Helimbra; Chugai Pharmaceutical, Roche/Genentech) - a bispecific factor IXa- and factor X-directed antibody - is designed to bring together factor IXa and factor X to activate the natural coagulation cascade and restore the blood clotting process.
    “Helimbra is the first medicine to show superior efficacy compared factor VIII prophylaxis, the standard of care for
    Read more ...