First Treatment for Primary Hemophagocytic Lymphohistiocytosis Approved by FDA

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.

David Alvaro, Ph.D.

David is Scientific Editorial Director for That’s Nice and the Pharma’s Almanac content enterprise, responsible for directing and generating industry, scientific and research-based content, including client-owned strategic content. Before joining That’s Nice, David served as a scientific editor for the multidisciplinary scientific journal Annals of the New York Academy of Sciences. He received a B.A. in Biology from New York University and a Ph.D. in Genetics and Development from Columbia University.