Sobi™ (http://www.sobi.com) announced today that the results from the pivotal phase 2/3 study evaluating the efficacy and safety of emapalumab in patients with primary haemophagocytic lymphohistiocytosis (HLH) were published in the
Primary HLH is a rare syndrome that typically presents in infancy but can also be seen in adults and is associated with high morbidity and mortality. This life-threatening disease is characterised by immune dysregulation and uncontrolled hyperinflammation. The treatment objective is to suppress the hyperinflammation and control the acute features of the disease in order to successfully bring patients to haematopoietic stem cell transplantation (HSCT).
"The publication of the results in this highly respected medical journal is a testament to the medical importance of the emapalumab findings for patients with primary HLH," says
The results with emapalumab in primary HLH published in the
Professor
This pivotal clinical study is the first study in primary HLH to prospectively assess and report treatment responses using predefined comprehensive objective clinical and laboratory criteria. Preclinical data have shown the central role of interferon gamma (IFNγ) in the pathogenesis of this disease[1]. Emapalumab is a monoclonal antibody that binds to and neutralises IFNγ. It was approved by the
About emapalumab
Emapalumab is a monoclonal antibody that binds to and neutralises interferon gamma (IFNγ). In the US, emapalumab is indicated for paediatric (newborn and older) and adult primary haemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). For more information please see www.gamifant.com including the full US Prescribing Information. Emapalumab is under review for primary HLH by the
About Sobi[TM]
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,400 people across
For more information please contact
Paula Treutiger, Head of Communication & Investor Relations
+ 46 733 666 599
paula.treutiger@sobi.com
+ 46 708 734 095
linda.holmstrom@sobi.com
1. Jordan et al. Blood 2004;104:735-43.
Postal address SE-112 76
Phone: 46 8 697 20 00 www.sobi.com
https://news.cision.com/swedish-orphan-biovitrum-ab/r/results-from-pivotal-phase-2-3-study-of-emapalumab-in-patients-with-primary-hlh-published-in-new-eng,c3105820
https://mb.cision.com/Main/14266/3105820/1243289.pdf
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