Pharmacokinetics, efficacy and safety of BAX326, a novel recombinant factor IX: a prospective, controlled, multicentre phase I/III study in previously treated patients with severe (FIX level <1%) or moderately severe (FIX level ≤2%) haemophilia B

Summary BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmac...

Full description

Saved in:
Bibliographic Details
Published in:Haemophilia : the official journal of the World Federation of Hemophilia 2014-01, Vol.20 (1), p.15-24
Main Authors: Windyga, J., Lissitchkov, T., Stasyshyn, O., Mamonov, V., Rusen, L., Lamas, J. L., Oh, M.-S., Chapman, M., Fritsch, S., Pavlova, B. G., Wong, W.-Y., Abbuehl, B. E.
Format: Article
Language:English
Subjects:
Adolescent
Adult
Aged
BAX326
Bleeding
Blood Coagulation - drug effects
Child
Factor IX - pharmacokinetics
Factor IX - therapeutic use
Female
haemophilia B
Hemophilia B - blood
Hemophilia B - drug therapy
Humans
Male
Middle Aged
Premedication
previously treated patients
Quality of Life
recombinant factor IX
Recombinant Proteins
Severity of Illness Index
Treatment Outcome
Young Adult
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Summary BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12–65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non‐serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence (n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC0–72 h per dose. Twice‐weekly prophylaxis [mean duration 6.2 (±0.7) months; 1.8 (±0.1) infusions per week, 49.5 (±4.8) IU kg−1 per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P 
ISSN:1351-8216
1365-2516
DOI:10.1111/hae.12228