FDA approves Novimmune’s lead molecule

Primary HLH is an ultra-rare syndrome of hyperinflammation with high morbidity and mortality and for which there was previously no approved drug. Gamifant represents a major advance in the treatment of these patients through a targeted mode of action.

“The FDA approval of Gamifant is a manifestation of Sobi’s vision to bring transformative treatments to patients with a high medical need,” says Sobi President and CEO Guido Oelkers. “Gamifant will add significant strength to Sobi’s Immunology franchise. We would like to acknowledge the excellent work done by Novimmune to develop and bring this important treatment to approval and Sobi will now focus on ensuring a successful commercialisation of Gamifant for the benefit of HLH patients in the US.”

The FDA approval is based on data from the pivotal phase 2/3 study which enrolled patients with primary HLH. The study primary endpoint in patients with either refractory, recurrent, or progressive disease during conventional HLH therapy or who were intolerant of conventional HLH therapy was achieved, with a clinically meaningful and statistically significant proportion of patients demonstrating an overall response at the end of treatment. In addition, 70 per cent of patients proceeded to haematopoietic stem-cell transplantation (HSCT). The most commonly reported adverse reactions (≥ 20 per cent) were infections, hypertension, infusion-related reactions and fever. Results from the pivotal study will be presented at forthcoming international meetings.

 “We are extremely glad to make a new medicine available for patients suffering from primary HLH,” says Cristina de Min, Chief Medical Officer at Novimmune. “Gamifant is the first drug specifically targeted to neutralise IFNγ. Based on the clinical validation of this new target, additional clinical studies are ongoing or being planned with emapalumab in diseases for which IFNγ is considered pathogenic.”