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

    7 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
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