The US Food and Drug Administration (FDA) has approved Sobi’s Gamifant (emapalumab) to treat adults and children with haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) associated with Still’s disease, including juvenile idiopathic arthritis (sJIA), where there is a reduced response or intolerance to glucocorticoids, or cases of recurrent MAS.
Gamifant functions as an anti-interferon gamma (IFNγ) monoclonal antibody.
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It is offered in three strengths:10mg/2 ml, 50mg/10ml and 100mg/20ml vials, administered intravenously.
Sobi CEO Guido Oelkers stated: “With our expertise in primary hemophagocytic lymphohistiocytosis, we understand the urgency of managing MAS quickly to improve patient outcomes.
“Gamifant is already an established therapy making a meaningful difference for patients with primary HLH, and with this approval, we are excited about the opportunity to positively impact patients affected by MAS in Still’s disease.”
The FDA approval was based on pooled data from two pivotal studies: the Phase III EMERALD trial and NI-0501-06.
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By GlobalDataA complete response (CR) at week eight was observed in 54% of patients, while 82% achieved clinical MAS remission at the same interval. Safety and tolerability profiles were consistent with those seen in earlier clinical trials.
HLH/MAS can be a severe complication within rheumatic diseases such as systemic sJIA and adult-onset Still’s disease. This rare disorder is characterised by hyperinflammation driven by IFNy, leading to symptoms like persistent high fever, elevated ferritin levels, coagulopathies, cytopenias and hepatosplenomegaly.
In February 2025, the company obtained FDA approval for Gamifant’s supplemental biologics licence application.
Gamifant is also authorised in the US for patients with primary HLH with refractory, persistent or progressive disease or intolerant to standard therapy.
