The Center for Drug Evaluation (CDE) has recommended approval of Gamifant to the National Medical Products Administration of China (NMPA).
The indication is for treatment of adult and paediatric (newborn and older) patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy.
“We are gratified that CDE has recommended Gamifant as potentially the first approved treatment for primary HLH in China,” says Norbert Oppitz, Head of International at Sobi. “If approved by the NMPA, this will be welcome news for those impacted by primary HLH across China.”
Primary haemophagocytic lymphohistiocytosis (HLH) is an ultra-rare, rapidly progressive, often-fatal syndrome of hyperinflammation in which hyperproduction of interferon gamma (IFNy) is thought to drive immune system hyperactivation, ultimately leading to organ failures. Diagnosis is challenging due to the variability in signs and symptoms, which may include fevers, swelling of the liver and spleen, severe low red and white blood cell counts, bleeding disorders, infections, neurological symptoms, organ dysfunction and organ failure.
Primary HLH can rapidly become fatal if left untreated, with median survival of less than two months. The immediate goal of treatment is to quickly control the hyperinflammation and to prepare for haematopoietic stem-cell transplant. The current conventional treatment prior to transplant includes steroids and chemotherapy and are not specifically approved to treat primary HLH.