Novimmune's rare immune system disorder candidate NI-0501 gets Breakthrough status

The US Food and Drug Administration has granted Breakthrough Therapy designation to Swiss biotech firm Novimmune’s NI-0501 for the treatment of patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory disease, or with recurrent or progressive disease during conventional therapy.

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. The designation is intended to expedite the development and review of new therapies

“The FDA’s designation of NI-0501 as a Breakthrough Therapy recognizes the potential of NI-0501 to address an important unmet medical need in a disease with still high mortality, and for which there are no approved treatments,” said Novimmune chairman and chief executive Eduard Holdener.