402
Views
5
CrossRef citations to date
0
Altmetric
Drug Profile

Nonacog gamma, a novel recombinant factor IX with low factor IXa content for treatment and prophylaxis of bleeding episodes

, , , , , , , , , , , , , , & show all

References

  • Giannelli F, Green PM, Sommer SS, et al. Haemophilia B: database of point mutations and short additions and deletions-eighth edition. Nucleic Acids Res 1998;26:265-8
  • Bicocchi MP, Pasino M, Rosano C, et al. Insight into molecular changes of the FIX protein in a series of Italian patients with haemophilia B. Haemophilia 2006;12:263-70
  • Mukherjee S, Saha A, Biswas P, et al. Structural analysis of factor IX protein variants to predict functional aberration causing haemophilia B. Haemophilia 2008;14:1076-81
  • Windyga J, Abbuehl BE, Hafeman AE. BAX326 (recombinant coagulation factor IX) for the treatment and prophylaxis of hemophilia B. Expert Rev Hematol 2014;7:333-42
  • Peden A, McCardle L, Head MW, et al. Variant CJD infection in the spleen of a neurologically asymptomatic UK adult patient with haemophilia. Haemophilia 2010;16:296-304
  • Sharp CP, Lail A, Donfield S, et al. Virologic and clinical features of primary infection with human parvovirus 4 in subjects with hemophilia: frequent transmission by virally inactivated clotting factor concentrates. Transfusion 2012;52:1482-9
  • Azzi A, Ciappi S, Zakvrzewska K, et al. Human parvovirus B19 infection in hemophiliacs first infused with two high-purity, virally attenuated factor VIII concentrates. Am J Hematol 1992;39:228-30
  • Ludlam CA and Turner ML. Managing the risk of transmission of variant Creutzfeldt Jakob disease by blood products. Br J Haematol 2006;132:13-24
  • Pipe SW. Recombinant clotting factors. Thromb Haemost 2008;99:840-50
  • Martinowitz U, Shapiro A, Quon DV, et al. Pharmacokinetic properties of IB1001, an investigational recombinant factor IX, in patients with haemophilia B: repeat pharmacokinetic evaluation and sialylation analysis. Haemophilia 2012;18:881-7
  • Inspiration Biopharmaceuticals Announces Clinical Hold of Clinical Trials Evaluating IB1001 for the Treatment and Prevention of Bleeding in Hemophilia B, July 10, 2012, Press Release
  • Dhillon S. Octocog alfa, antihaemophilic factor (recombinant), plasma/albumin free method (Advate(R)): a review of its use in the management of patients with haemophilia A. Drugs 2012;72:987-1007
  • Pipe S. Antihemophilic factor (recombinant) plasma/albumin-free method for the management and prevention of bleeding episodes in patients with hemophilia A. Biologics 2009;3:117-25
  • Santagostino E. PROLONG-9FP clinical development program – phase I results of recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP). Thromb Res 2013;131(Suppl 2):S7-10
  • Valentino LA. The role of Rixubis in the treatment of hemophilia B. Immunotherapy 2014;6:381-94
  • Martinowitz U, Lubetsky A. Phase I/II, open-label, multicenter, safety, efficacy and PK study of a recombinant coagulation factor IX albumin fusion protein (rIX-FP) in subjects with hemophilia B. Thromb Res 2013;131(Suppl 2):S11-14
  • Windyga J, Lissitchkov T, Stasyshyn O, et al. 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. Haemophilia 2014;20:15-24
  • RIXUBIS [package insert]. Baxter Healthcare Corp. Westlake Village, CA. 2014. Available from: www.baxter.com/downloads/healthcare_professionals/products/RIXUBIS_PI.pdf [Last accessed 6 November 2014]
  • Dietrich B, Schiviz A, Hoellriegl W. Preclinical safety and efficacy of a new recombinant FIX for treatment of hemophilia B. Int J Hematol 2013;98:525-32
  • Pickering WM, Gray E. The effect of activated factor IX on the factor IX coagulant and NAPTT activity of a high-purity factor IX concentrate. J Thromb and Haemost 2007;5: abstract: P-T-156
  • Wessler S, Reimer SM, Sheps MC. Biologic assay of a thrombosis-inducing activity in human serum. J Appl Physiol 1959;14:943-6
  • Urasinski T, Stasyshyn O, Andreeva T, et al. Recombinant factor IX (BAX 326) in previously-treated pediatric patients with haemophilia B: a prospective clinical trial. Hemophilia 2014. [Epub ahead of print]
  • Windyga J, Lissitchkov T, Stasyshyn O, et al. Efficacy and safety of a recombinant factor IX (Bax326) in previously treated patients with severe or moderately severe haemophilia B undergoing surgical or other invasive procedures: a prospective, open-label, uncontrolled, multicentre, phase III study. Haemophilia 2014;20:651-8
  • Core SPC for human plasma derived and recombinant coagulation factor IX products. Committee for Proprietary Medicinal Products. London: CPMP/BPWG/1625/99.European Agency for the Evaluation of Medicinal Products (EMEA). 2000. Available from: www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003706.pdf [Last accessed 18 November 2014]
  • Clinicaltrials. Available from: https://clinicaltrials.gov/
  • Wilmot HV, Hogwood J, Gray E. Recombinant factor IX: discrepancies between one-stage clotting and chromogenic assays. Haemophilia 2014;20:891-7
  • Dahlbäck B. Blood coagulation. Lancet 2000;355:1627-32
  • Schmaier AH, Dahl LD, Hasan AA, et al. FactorIXa inhibition by protease nexin-2/amyloid beta-protein precursor on phospholipid vesicles and cell membranes. Biochemistry 1995;34:1171-8
  • Monahan PE, Liesner R, Sullivan ST, et al. Safety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia B. Haemophilia 2010;16:460-8
  • Shapiro AD, Di PJ, Cohen A, et al. The safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia B. Blood 2005;105:518-25
  • Vermylen J, Peerlinck K. Review of the hepatitis A epidemics in hemophiliacs in Europe. Vox Sang 1994;67:Suppl 4;8-11
  • Mariani G, Di PT, Baklaya R, et al. Prospective study of the evaluation of hepatitis C virus infectivity in a high-purity, solvent/detergent-treated factor VIII concentrate: parallel evaluation of other markers for lipid-enveloped and non-lipid-enveloped viruses. The Ad Hoc Study Group of the Fondazione dell’Emofilia. Transfusion 1993;33:814-18
  • Bartolomei CO, Azzi A, Morfini M, et al. Human parvovirus infection in haemophiliacs first infused with treated clotting factor concentrates. J Med Virol 1988;25:165-70
  • Lambert T, Recht M, Valentino LA, et al. Reformulated BeneFix: efficacy and safety in previously treated patients with moderately severe to severe haemophilia B. Haemophilia 2007;13:233-43
  • DiMichele D. Inhibitor development in haemophilia B: an orphan disease in need of attention. Br J Haematol 2007;138:305-15
  • Ewenstein BM, Joist JH, Shapiro AD, et al. Pharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia B. Transfusion 2002;42:190-7
  • Roth DA, Kessler CM, Pasi KJ, et al. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood 2001;98:3600-6
  • Giangrande P. Haemophilia B: christmas disease. Expert Opin Pharmacother 2005;6:1517-24
  • Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP), 20-23 October 2014. 2014. Available from: www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2014/10/news_detail_002191.jsp&mid=WC0b01ac058004d5c1 [Last accessed 6 November 2014]
  • Shapiro AD, Ragni MV, Valentino LA, et al. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients. Blood 2012;119:666-72
  • Ivens IA, Baumann A, McDonald TA, et al. PEGylated therapeutic proteins for haemophilia treatment: a review for haemophilia caregivers. Haemophilia 2013;19:11-20
  • Negrier C, Knobe K, Tiede A, et al. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood 2011;118:2695-701
  • Powell J, Shapiro A, Ragni M, et al. Switching to recombinant factor IX Fc fusion protein prophylaxis results in fewer infusions, decreased factor IX consumption and lower bleeding rates. Br J Haematol 2015;168(1):113-23
  • Bjorkman S. Pharmacokinetics of plasma-derived and recombinant factor IX - implications for prophylaxis and on-demand therapy. Haemophilia 2013;19:808-13

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.