|Emapalumab's efficacy confirmed by sensitivity analysis presented at ESID2020|
|By: PR Newswire Association LLC. - 17 Oct 2020||Back to overview list
STOCKHOLM, Sweden, Oct. 17, 2020 /PRNewswire/ -- Sobi™ today presented results from the sensitivity analysis from the pivotal phase 2/3 study (NCT01818492) of emapalumab in patients with primary HLH at the 19th meeting of the European Society of Immunodeficiencies (ESID). Analyses of the efficacy of emapalumab in primary HLH utilising various definitions of treatment response all support the study primary endpoint of a 63 per cent overall response rate (ORR) in patients with insufficient response to standard of care, as published in NEJM in May 20201.
"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 Ravi Rao, Head of R&D and Chief Medical Officer at Sobi. "We remain committed to making emapalumab accessible to patients affected by this severe condition around the world."
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 US Food & Drug Administration (FDA) for primary HLH. Emapalumab is under review by the European Medicines Agency (EMA).
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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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SOURCE Swedish Orphan Biovitrum AB
Copyright 2020 PR Newswire Association LLC.
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