Efficacy of EHL N9-GP for on-demand treatment of bleeding episodes in hemophilia B: analysis of pivotal trial data
Year: 2019
Type: article
Abstract: Miguel A Escobar,1 Christopher E Walsh,2 David L Cooper,3 Guy Young41Department of Internal Medicine, University of Texas Health Science Center at Houston—McGovern Medical School, Houston, TX, USA; 2Icahn School of Medicine at Mount Sinai, New York, NY, USA; 3Clinical Development and Medical Affairs - Biopharm, Novo Nordisk Inc, Plainsboro, NJ, USA; 4Hemostasis and Thrombosis Center, Children’s Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USAThe safety and efficacy of N9-GP (nonacog beta pegol; Novo Nordisk A/S, Bagsværd, Denmark), a recombinant glycoPEGylated factor IX(FIX) with extended half-life (EHL),1 was investigated in the multinational, Phase 3, paradigm 2 trial (NCT01333111), previously reported by Collins et al in Blood.2 The trial was conducted in accordance with the Declaration of Helsinki and written informed consent was provided by all participants. Prior to trial initiation, the protocol, the protocol amendments, the consent form, and the patient information sheet were reviewed and approved according to local regulations by appropriate health authorities and by independent ethics committees/institutional review boards (see Table S1). Patients 13–70 years of age with previously treated hemophilia B (≤2% baseline FIX) were allocated to either once-weekly prophylaxis or on-demand (OD) treatment. TheODtreatment was a US Food andDrugAdministration requirement prior to enrolling patients on prophylaxis in the USA.
Source: Journal of Blood Medicine
DOI: 10.2147/jbm.s212690
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $2,320
Funder: Novo Nordisk
Award: G1572433987
OpenAlex ID: W2962856511