Novimmune, a Swiss biotech company, announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to 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 (IFN?) monoclonal antibody discovered and developed by Novimmune. The biological activity of IFN?, which is considered to have a pivotal pathogenic role in HLH, is neutralized by NI-0501, the first targeted therapy for this disease.

“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 officer, Eduard Holdener. “We look forward to working closely with the regulatory authorities to bring NI-0501 to patients suffering from this devastating disease as fast as possible,” added Novimmune chief medical officer, Cristina de Min.

The Breakthrough Therapy designation is intended to expedite the development and review of new therapies for serious or life threatening conditions, which have shown encouraging early clinical results demonstrating substantial improvement on a clinically significant endpoint over available therapies.

This Breakthrough Therapy designation for NI-0501 was granted by the FDA on the basis of clinical data from a phase 2 study in children with primary HLH. Preliminary results from the phase 2 study have been presented as a late breaking abstract at the American Society of Hematology meeting in Orlando, 8th December 2015.

NI-0501 received orphan drug designation from both the FDA and the EMA in 2010.

Novimmune has been awarded by the Seventh Framework Programme (FP7) of the European Commission a €6 million grant to the “FIGHT HLH” consortium of institutions to support clinical testing of Novimmune’s anti-interferon gamma drug candidate, NI-0501.

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. Two forms of HLH are described: primary HLH is genetically determined and is typically diagnosed in infants and young children, while secondary HLH occurs on a background of a rheumatic/inflammatory disease, a malignancy or an infection.

Mortality in patients with primary and secondary HLH remains high, despite the use of aggressive immune-chemotherapy.