FDA approves Sobi’s Gamifant for rare, fatal childhood disease

The FDA has approved Sobi’s Gamifant, a treatment for primary hemophagocytic lymphohistiocytosis (HLH). The extremely rare condition usually manifests within the first year of life, progresses quickly and is usually fatal, with a median survival of fewer than two months. HLH is caused by hyper-inflammation whereby massive over-expression of interferon gamma (IFNy) is thought to cause the immune system to attack the body’s tissues and organs. Gamifant (tabalumab-list), the first significant improvement in primary HLH induction therapy in 24 years – is approved to treat adults, children and babies who have a refractory, recurrent or progressive disease or intolerance to conventional HLH therapy. The medicine works by controlling the inflammation and preparing the patient for a hematopoietic stem cell transplant – the only cure for HLH. Hematopoietic stem cells are those that give rise to other blood cells.