8/4/2023 0 Comments Hlh up to date![]() ![]() “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. The approval of Gamifant as the first treatment for primary HLH is a significant step in our journey of helping these patients.” Milan Zdravkovic, Chief Medical Officer and Head of Research & Development at Sobi, says: “Primary HLH is a very rare disease with significant morbidity and mortality. Results from the pivotal study will be presented at forthcoming international meetings. ![]() The most commonly reported adverse reactions (≥ 20 per cent) were infections, hypertension, infusion-related reactions and fever. In addition, 70 per cent of patients proceeded to haematopoietic stem-cell transplantation (HSCT). 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. The FDA approval is based on data from the pivotal phase 2/3 study which enrolled patients with primary HLH. 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.” “Gamifant will add significant strength to Sobi’s Immunology franchise, along with Kineret and the recent acquisition of the US rights to Synagis®. “The FDA approval of Gamifant marks an important milestone in Sobi’s vision to bring transformative treatments to patients with a high medical need,” says Sobi President and CEO Guido Oelkers. Gamifant represents a major advance in the treatment of these patients through a targeted mode of action. Primary HLH is an ultra-rare syndrome of hyperinflammation with high morbidity and mortality and for which there was previously no approved drug. Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO: SOBI), an international biopharmaceutical company dedicated to rare diseases, and Novimmune SA, a Swiss biotech company, today announce that the US Food and Drug Administration (FDA) has approved Gamifant® (emapalumab-lzsg), an interferon gamma (IFNγ) blocking antibody for the treatment of paediatric (new born and older) and adult patients with primary haemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy. ![]()
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