FDA approves Gamifant to treat patients with primary haemophagocytic lymphohistiocytosis .- SOBI

Swedish Orphan Biovitrum AB (publ) (Sobi) announced that the FDA has approved Gamifant (emapalumab-lzsg), an interferon gamma blocking antibody for the treatment of paediatric (new born and older) and adult patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy.

Primary HLH is an ultra-rare syndrome of hyperinflammation with high morbidity and mortality and for which there was previously no approved drug. Gamifant represents a major advance in the treatment of these patients through a targeted mode of action. The FDA approval is based on data from the pivotal phase II/III study which enrolled patients with primary HLH. The study 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 per cent of patients proceeded to haematopoietic stem-cell transplantation (HSCT). The most commonly reported adverse reactions ( greater than 20 per cent) were infections, hypertension, infusion-related reactions and fever. Results from the pivotal study will be presented at forthcoming international meetings.

SOBI acquired world rights to the drug from Novimmune SA.