Once-weekly prophylaxis with glycoPEGylated recombinant factor VIII (N8-GP) in severe haemophilia A: Safety and efficacy results from pathfinder 2 (randomized phase III trial)

Haemophilia : the Official Journal of the World Federation of Hemophilia
Nicola CurrySteven R Lentz

Abstract

Turoctocog alfa pegol (N8-GP) is a site-specific, 40 kDa glycoPEGylated recombinant factor VIII (FVIII) product with an extended half-life. The comprehensive main phase of the pivotal pathfinder 2 trial showed N8-GP dosed every 4 days (Q4D) provided favourable safety and efficacy for preventing bleeds in 175 patients with haemophilia A. We investigated the safety and efficacy of N8-GP prophylaxis when administered weekly (Q7D) for 24 weeks to patients with low bleeding rates in the pathfinder 2 extension trial. Patients (≥12 years) with ≤2 bleeds during the preceding 6 months of the pathfinder 2 main phase were eligible for randomization to receive N8-GP 50 IU/kg Q4D or 75 IU/kg Q7D. Safety and efficacy endpoints were incidence of FVIII inhibitors and annualized bleeding rate (ABR), respectively. Fifty-five of 143 (38.5%) patients on prophylaxis who continued into the extension phase were randomized to receive 50 IU/kg Q4D (n = 17) or 75 IU/kg Q7D (n = 38). Nine patients in the Q7D cohort reverted to 50 IU/kg Q4D. No inhibitors were detected. In both cohorts, >50% of patients experienced no bleeds. Median ABR for overall, joint, spontaneous, traumatic and muscle was 0.00 for both cohorts. Overall estimated success rate for trea...Continue Reading

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Jan 29, 2020·Expert Opinion on Investigational Drugs·Pier Mannuccio Mannucci
Jun 10, 2020·Expert Review of Clinical Pharmacology·Massimo Morfini, Emanuela Marchesini
Dec 22, 2020·Seminars in Thrombosis and Hemostasis·Matteo Nicola Dario Di MinnoMassimo Franchini
Feb 5, 2021·Journal of Blood Medicine·Mariasanta NapolitanoHermann Eichler
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Sep 26, 2021·Haemophilia : the Official Journal of the World Federation of Hemophilia·Andreas TiedePratima Chowdary

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Clinical Trials Mentioned

NCT01480180

Software Mentioned

pathfinder

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