HLT therapy Gamifant approved in China

Rare diseases specialist Swedish Orphan Biovitrum (STO: SOBI), also known as Sobi, today announced that China’s National Medical Products Administration (NMPA) has approved Gamifant (emapalumab) for use in China.

The indication is for treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy. The approval followed the recommendation announced in February 2022.

Primary HLH is an ultra-rare, life-threatening, hyperinflammatory disorder, characterized by uncontrolled activation of the immune system. Morbidity and mortality are high due to lack of detection and non-specific symptoms, but if diagnosed early and appropriately treated, the inflammation and damage can be reduced. Gamifant represents a new treatment option for patients with progressive disease through a targeted mode of action.