Swiss biotech, Novimmune received Breakthrough Therapy Designation from U.S FDA for its drug candidate NI-0501 for the treatment of patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory disease, or with recurrent or progressive disease during conventional therapy. HLH is a hyperinflammatory syndrome characterized by uncontrolled and aberrant activation of the immune system and a life-threatening cytokine storm presenting with non-remitting fever, pancytopenia, coagulopathy, and hemophagocytosis potentially leading to death. NI-0501 is a fully human, anti-interferon-gamma (IFNγ) monoclonal antibody discovered and developed by Novimmune. The biological activity of IFNγ, which is considered to have a pivotal pathogenic role in HLH, is neutralized by NI-0501, the first targeted therapy for this disease. NI-0501 received orphan drug designation from both the FDA and the EMA in 2010.