Emapalumab for primary HLHFebruary 5, 2019
Emapalumab has been cleared for the treatment of primary haemophagocytic lymphohistiocytosis (HLH).
Emapalumab (Gamifant) is the first and only treatment for HLH, according to Swedish Orphan Biovitrum AB and Novimmune SA, a Swiss biotech company.
Emapalumab-lzsg is an interferon gamma blocking antibody for the treatment of paediatric and adult patients with HLH. The drug is a monoclonal antibody (mAb) that binds to and neutralises interferon gamma (IFNy).
Primary HLH is an ultra-rare syndrome of hyperinflammation with high morbidity and mortality and for which there was previously no approved drug.
The FDA approval is based on data from the pivotal phase 2/3 study which enrolled patients with primary HLH. The study’s primary endpoint in patients with either refractory, recurrent, or progressive disease during conventional HLH therapy or who were intolerant of conventional HLH therapy was achieved, with a clinically meaningful and statistically significant proportion of patients demonstrating an overall response at the end of treatment. In addition, 70 percent of patients proceeded to haematopoietic stem-cell transplantation (HSCT).