Sobi announced that the U.S. Food and Drug Administration (FDA) approved Gamifant (emapalumab-lzsg) for the treatment of adult and pediatric (newborn and older) patients with hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.
“With our expertise in primary hemophagocytic lymphohistiocytosis, we understand the urgency of managing MAS quickly to improve patient outcomes,” said Guido Oelkers, Chief Executive Officer at Sobi. “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 approval is based on results of the pooled data from two pivotal studies, the Phase 3 study (NCT05001737) and NI-0501-06 (NCT03311854). 54 percent (21/39) of patients had a complete response at Week 8, and 82% (32/39) achieved clinical MAS remission (VAS ≤1 cm) at Week 8. Safety and tolerability were consistent with previous clinical studies. In patients with HLH/MAS in Still’s disease, the most common adverse events (≥20%) were viral infections, including cytomegalovirus infection or reactivation, and rash.
“MAS in Still’s disease is a serious and potentially life-threatening complication, marked by severe hyperinflammation and, in some cases, multi-organ failure,” said Alexei A. Grom, MD, Professor of Pediatrics, Research Director Division of Rheumatology, Cincinnati Children’s Hospital Medical Center. “Many patients affected by MAS—both young children and adults—face significant unmet medical needs. With Gamifant now as the first FDA-approved treatment for MAS, we have a new therapeutic option that helps control hyperinflammation and reduce our reliance on high-dose glucocorticoids.”
MAS, a form of HLH, is a severe complication of rheumatic diseases, occurring most frequently in Still’s disease including systemic juvenile idiopathic arthritis and adult-onset Still’s disease. HLH/MAS is a rare systemic disorder of interferon gamma (IFNy) driven hyperinflammation with common clinical manifestations such as high persistent fever, elevated ferritin, cytopenias, coagulopathies, and hepatosplenomegaly.
Gamifant, an interferon gamma (IFNγ)–blocking antibody, is the first and only FDA approved treatment for adult and pediatric (newborn and older) patients with primary HLH with refractory, recurrent, or progressive disease or intolerance with conventional HLH therapy.