Emapalumab's efficacy for primary HLH is confirmed by sensitivity analysis presented at ESID2020.- Swedish Orphan Biovitrium

Sobi presented results from the sensitivity analysis from the pivotal phase II/III 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 2020. 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).