The Food and Drug Administration (FDA) has approved Gamifant (emapalumab-lzsg; Sobi and Novimmune SA) for the treatment of pediatric (newborn and older) and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) who have refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. It is intended for use in combination with intravenous (IV) dexamethasone until time of hematopoietic stem cell transplant.
Gamifant, an interferon gamma blocking monoclonal antibody, was evaluated in a Phase 2/3 multicenter, open-label, single-arm clinical trial (N=27) of pediatric patients (median age 1 year) with suspected or confirmed primary HLH with either refractory, recurrent or progressive disease during conventional HLH therapy or who were intolerant of conventional HLH therapy. The primary endpoint was achieved, with an overall response rate (defined as achievement of either a complete or partial response or HLH improvement) of 63% (P= .013) at the end of treatment; 70% of patients were able to proceed to stem cell transplant. Study data will be presented at a late-breaking session at the American Society of Hematology Meeting 2018.
Infections, hypertension, infusion-related reactions, and fever were the most common side effects reported in patients who received Gamifant.
Gamifant will be available as a 10mg/2mL and 50mg/10mL preservative-free solution in single-dose vials. It is anticipated to launch in the first quarter of 2019 for administration in treatment centers.
For more information call (866) 773-5274 or visit Gamifant.com.