Emapalumab's efficacy confirmed by sensitivity analysis presented at ESID2020
"This analysis confirms the methodology and use of clinically objective response criteria in the pivotal study assessing treatment efficacy of emapalumab in primary HLH, where there are currently no validated endpoints for efficacy assessment" said
A high unmet medical need exists among these patients despite today's standard-of-care therapy, glucocorticoids and etoposide, with or without cyclosporine. Conventional therapies are associated with adverse events as cytotoxicity and opportunistic infections, and no decrease in mortality has been shown in the past 20 years using the current standard-of-care therapies.
No standard approach to measuring response rate to treatment has been established due to the rarity of the disease. The pivotal study used clinically objective response criteria to define the primary endpoint of overall response rate to emapalumab in primary HLH. The sensitivity analysis supports the use of the clinically objective ORR as a primary endpoint in studies of primary HLH.
Emapalumab is the first therapy approved by the
Emapalumab is a monoclonal antibody that binds to and neutralises interferon gamma (IFNγ). In the US, emapalumab is indicated for the treatment of adult and paediatric (new-born and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. Primary HLH is 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
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1Locatelli F, Jordan MB, Allen C, et al. Emapalumab in children with primary hemophagocytic lymphohistiocytosis. N Engl J Med 2020;382:1811-22.
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Press release - Emapalumab sensitivity data
SOURCE Swedish Orphan Biovitrum AB