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Efficacy of EHL N9-GP for on-demand treatment of bleeding episodes in hemophilia B: analysis of pivotal trial data

Authors Escobar MA, Walsh CE, Cooper DL, Young G

Received 19 April 2019

Accepted for publication 28 June 2019

Published 25 July 2019 Volume 2019:10 Pages 243—250


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 2

Editor who approved publication: Dr Martin Bluth

Miguel A Escobar,1 Christopher E Walsh,2 David L Cooper,3 Guy Young4

1Department 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, USA

The 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.

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