Gamifant (emapalumab-lzsg) is approved for the treatment of the rare immune disease HLH in both adults and children.
People suffering from the rare inherited immune disease primary hemophagocytic lymphohistiocytosis (HLH) suffer from too many activated immune cells, resulting in symptoms ranging from fever to enlargement of the liver or spleen, decreased production of red blood cells and neurological problems.
The FDA has recently approved the first treatment specifically developed for primary HLH. Gamifant (emapalumab-lzsg) was approved based on results of a phase II/III clinical trial. Out of 27 patients (82% with a genetically confirmed primary HLH diagnosis) treated with Gamifant and dexamethasone through intravenous infusion over 1 hour twice per week until hematopoietic stem cell transplant, 63% achieved either a complete or partial response or HLH improvement. In addition, 70% were able to proceed to hematopoietic stem cell transplant.
Gamifant, which was developed by Novimmune SA, targets the cytokine interferon gamma and is a new approach to treating primary HLH. It received Priority Review and Breakthrough Therapy and Orphan Drug designations. Novimmune SA licensed the global rights to Gamifant exclusively to Sobi in July 2018.