US FDA Grants Priority Review to Sobi's Supplemental Biologics Licence Application for Gamifant
Friday, February 28, 2025
Sobi® has announced that the U.S. Food and Drug Administration (FDA) has accepted the supplemental Biologics License Application (sBLA) for Gamifant® (emapalumab-lzsg). The application seeks approval for use in adult and paediatric patients with haemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) associated with Still's disease, who have had an inadequate response or intolerance to glucocorticoids or have experienced recurrent MAS. The application has been granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target date set for 27 June 2025.
HLH/MAS is a severe complication of rheumatic diseases, commonly occurring in Still's disease, including systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). It is a rare systemic disorder of hyperinflammation, leading to persistent high fever, elevated ferritin levels, cytopenias, coagulopathies, and hepatosplenomegaly. Without appropriate treatment, the condition can progress to multiple organ failure.
The application is supported by pooled data from two clinical studies, EMERALD (NCT05001737) and NI-0501-06 (NCT03311854), involving 39 patients. Results showed that 53% of patients achieved a complete response by Week 8, while 85% achieved a complete response at some point during the studies. Additionally, the mean weekly glucocorticoid dosage was reduced by 70.1% after two weeks of treatment.
Gamifant® (emapalumab-lzsg) is a monoclonal antibody that binds to and neutralises interferon gamma (IFN-γ), a key driver of hyperinflammation. It was first approved by the FDA in 2018 for treating primary HLH in adult and paediatric patients (including newborns) with refractory, recurrent, or progressive disease or intolerance to conventional HLH therapy. The FDA approval for primary HLH was based on data from Phase 2/3 clinical studies (NCT01818492 and NCT02069899).
MAS is recognised as a severe complication of rheumatic diseases such as sJIA and AOSD. It is characterised by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities, and hyperferritinaemia, with the potential to progress to multiple organ failure. As a subtype of HLH, MAS presents significant risks if left untreated.
In the United States, Gamifant® (emapalumab-lzsg) remains the only FDA-approved treatment for primary HLH. It is administered via intravenous infusion over one hour, twice per week, until haematopoietic stem cell transplantation (HSCT) is performed.
Source: prnewswire.com
