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- Title
Pharmacokinetics of a novel extended half-life glyco PEGylated factor IX, nonacog beta pegol (N9- GP) in previously treated patients with haemophilia B: results from two phase 3 clinical trials.
- Authors
Tiede, A.; Abdul-Karim, F.; Carcao, M.; Persson, P.; Clausen, W. H. O.; Kearney, S.; Matsushita, T.; Negrier, C.; Oldenburg, J.; Santagostino, E.; Young, G.
- Abstract
Introduction Nonacog beta pegol (N9- GP) is a glyco PEGylated recombinant factor IX ( FIX) with an extended half-life developed for routine prophylaxis and the prevention and treatment of bleeding episodes in patients with haemophilia B. Aim The aim of this study was to evaluate the pharmacokinetics ( PK) of N9- GP. Methods Data from 41 previously treated haemophilia B patients, enrolled globally (16 adolescents/adults and 25 children; FIX activity ≤0.02 IU mL−1) with no history of FIX inhibitors, were included. N9- GP was administered once-weekly as 10 IU kg−1 or 40 IU kg−1 in adolescents/adults and 40 IU kg−1 in children. Blood was sampled up to 168 h (1 week) post dose. Standard PK was estimated on the basis of plasma FIX activity vs. time ( PK profiles) using non-compartmental methods. Furthermore, a population PK analysis and FIX activity predictions were performed. Results Incremental recoveries were 0.02 ( IU mL−1)/( IU kg−1) in both adolescents/adults and children. The extended half-life resulted in mean trough levels of 0.27 IU mL−1 for adolescents/adults and 0.17 IU mL−1 for children at steady-state after weekly dosing at 40 IU kg−1. The population PK analysis confirmed a mono-exponential decay in FIX activity and allowed for predictions of FIX activity for adolescents/adults above 0.15 IU mL−1 at all times and 6.4 days week−1 in children. Conclusion N9- GP has the potential to shift previously treated haemophilia B patients from a severe/moderate disease state into a mild- or non-haemophilic range for most of the dosing interval, which is expected to reduce the number of bleeding episodes.
- Subjects
PHARMACOKINETICS; HEMOPHILIACS; CLINICAL trials; MEDICAL care; BLOOD coagulation disorders; BLOOD disease treatment
- Publication
Haemophilia, 2017, Vol 23, Issue 4, p547
- ISSN
1351-8216
- Publication type
Article
- DOI
10.1111/hae.13191