Sobi has announced that the US Food and Drug Administration (FDA) has accepted the supplemental biologics license application (sBLA) for Gamifant (emapalumab-lzsg).
This is for treating adult and paediatric patients with hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome (MAS) in Still’s disease.
The treatment is for patients with an inadequate response to or intolerance of glucocorticoids, or those with recurrent MAS.
The application has been granted priority review, with a Prescription Drug User Fee Act (PDUFA) date of 27 June 2025.
In May 2024, Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi), received FDA Fast Track designation for emapalumab-lzsg.
The sBLA is based on pooled data from two studies, EMERALD (NCT05001737) and NI-0501-06 (NCT03311854), which enrolled 39 patients.
A total of 53% of patients achieved a complete response at week 8, while 85% achieved a complete response at any time during the studies.
Weekly mean glucocorticoid doses were reduced by 70.1% after two weeks of treatment.
Sobi chief medical officer and R&D head Lydia Abad-Franch said: “HLH/MAS in Still’s disease is a serious and potentially fatal complication where patients can experience intense hyperinflammation and even multiple organ failure.
“There is no approved therapy for HLH/MAS today. Gamifant (emapalumab-Izsg) selectively neutralises interferon gamma (IFN-γ), a key driver of hyperinflammation, and if approved, may also help reduce the need for high-dose glucocorticoids in these patients.”
Gamifant is a monoclonal antibody that targets and neutralises IFN-γ.
Initially approved by the FDA in 2018, it is used to treat adult and paediatric patients with primary HLH who have refractory, recurrent, or progressive disease, or who are intolerant of conventional HLH therapies.
Recently, Sobi and Apellis Pharmaceuticals announced that the European Medicines Agency (EMA) has validated an indication extension application for Aspaveli (pegcetacoplan).
The application is for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), rare chronic kidney diseases with no approved treatments.
