The U.S. Food and Drug Administration (FDA) has approved Gamifant (emapalumab-lzsg) for the treatment of patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in Still’s disease.
Still’s disease is a rare systemic inflammatory disorder characterized by inflammatory polyarthritis, daily fever, and maculopapular rash. Patients also typically have elevated serum ferritin levels. The exact cause of disease is unknown. MAS is a form of HLH that frequently occurs in patients with Still’s disease. HLH/MAS is a disorder of interferon gamma (IFNγ) driven hyperinflammation characterized by high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly.
Emapalumab is an anti-interferon gamma monoclonal antibody that binds to and neutralizes IFNγ, reducing hyperinflammation. The approval follows positive results from pooled data of two studies (NCT05001737 and NCT03311854). In these studies, 54% of patients experienced a complete response at week 8, and 82% achieved clinical MAS remission at week 8. Safety and tolerability were favorable and consistent with previous studies. The most common adverse events were viral infections, such as cytomegalovirus infection or reactivation, and rash.
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To learn more about Still’s disease and other rare autoimmune conditions, visit https://checkrare.com/diseases/autoimmune-and-auto-inflammatory-disorders/