Extended half-life clotting factor concentrates: results from published clinical trials

Haemophilia

Volumn 22, Issue , 2016, Pages 25-30

  Young, G ^a   Mahlangu, J N ^b  

^a UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^b UNIVERSITY OF THE WITWATERSRAND   (South Africa)

Author keywords

extended half life; factor; haemophilia; prophylaxis

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR CONCENTRATE; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT BLOOD CLOTTING FACTOR 9;

AREA UNDER THE CURVE; ARTICLE; BLEEDING DISORDER; DRUG CLEARANCE; DRUG EFFICACY; DRUG FATALITY; DRUG HALF LIFE; DRUG SAFETY; EXTENDED HALF LIFE; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HUMAN; MULTICENTER STUDY (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PUBLISHING; THROMBOSIS; TREATMENT RESPONSE;

EID: 84978832712     PISSN: 13518216     EISSN: 13652516     Source Type: Journal    
DOI: 10.1111/hae.13028     Document Type: Article

References (30)

1. Muto A, Yoshihashi K, Takeda M et al. Anti-factor IXa/X bispecific antibody (ACE910): hemostatic potency against ongoing bleeds in a hemophilia A model and the possibility of routine supplementation. J Thromb Haemost 2014; 12: 206–13.
2. Muto A, Yoshihashi K, Takeda M et al. Anti-factor IXa/X bispecific antibody ACE910 prevents joint bleeds in a long-term primate model of acquired hemophilia A. Blood 2014; 124: 3165–71.
3. Sehgal A, Barros S, Ivanciu L et al. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat Med 2015; 21: 492–7.
4. Chowdary P, Lethagen S, Friedrich U et al. Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial. J Thromb Haemost 2015; 13: 743–54.
5. Lheriteau E, Davidoff AM, Nathwani AC. Haemophilia gene therapy: progress and challenges. Blood Rev 2015; 29: 321–8.
6. Nathwani AC, Reiss UM, Tuddenham EG et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371: 1994–2004.
7. High KH, Nathwani A, Spencer T, Lillicrap D. Current status of haemophilia gene therapy. Haemophilia 2014; 20(Suppl 4): 43–9.
8. Powell JS, Pasi KJ, Ragni MV et al. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med 2013; 369: 2313–23.
9. Santagostino E, Martinowitz U, Lissitchkov T et al. Long acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood 2016; 127(14): 1761–69.
10. Collins PW, Young G, Knobe K et al. Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial. Blood 2014; 124: 3880–6.
11. McCue J, Osborne D, Dumont J et al. Validation of the manufacturing process used to produce long-acting recombinant factor IX Fc fusion protein. Haemophilia 2014; 20: e327–35.
12. Shapiro A. Development of long-acting recombinant FVIII and FIX Fc fusion proteins for the management of hemophilia. Expert Opin Biol Ther 2013; 13: 1287–97.
13. Collins PW, Colberg T, Young G et al. Safety, efficacy and pharmacokinetics of nonacog beta pegol (N9-GP) for prophylaxis and treatment of bleeding episodes in patients with haemophilia B. J Thromb Haemost 2013; 11: 19.
14. ™4) Thromb Res. 2016; 141: 69–76.
15. 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.
16. ®: efficacy and safety in previously treated patients with moderately severe to severe haemophilia B. Haemophilia 2007; 13: 233–43.
17. Mahlangu J, Powell JS, Ragni MV et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood 2014; 123: 317–25.
18. Konkle B, Stasyshyn O, Chowdary P et al. Pegylated, full-length recombinant factor VIII for prophylactic and on-demand treatment of hemophilia A. Blood 2015; 126: 1078–85.
19. Lentz SR, Misgav M, Ozelo M et al. Results from a large multinational clinical trial (guardian 1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy. Haemophilia 2013; 19: 691–7.
20. Boggio LNHW, Wang M, Eyster ME, Michaels LA. Bleeding phenotype with various BAY 94-9027 dosing regimens: sub-analyses from the Protect VIII study. Blood 2014; 124: 126. (abstract).
21. Stidl R, Fuchs S, Bossard M, Siekmann J, Turecek PL, Putz M. Safety of PEGylated recombinant human full-length coagulation factor VIII (BAX 855) in the overall context of PEG and PEG conjugates. Haemophilia 2016; 22: 54–64.
22. Young G, Mahlangu JN, Kulkarni R et al. Safety, efficacy, and pharmacokinetics of recombinant factor VIII Fc fusion protein (rFVIIIFc) in previously-treated children with severe hemophilia a (Kids-ALONG). Blood 2014; 124: 1494.
23. Dumont JA, Low SC, Peters RT, Bitonti AJ. Monomeric Fc fusions: impact on pharmacokinetic and biological activity of protein therapeutics. BioDrugs 2006; 20: 151–60.
24. Dumont JA, Liu T, Low SC et al. Prolonged activity of a recombinant factor VIII-Fc fusion protein in hemophilia A mice and dogs. Blood 2012; 119: 3024–30.
25. Valentino L. Bax 855. Haemophilia 2015; 21: 58.
26. Coyle TE, Reding MT, Lin JC, Michaels LA, Shah A, Powell J. Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A. J Thromb Haemost 2014; 12: 488–96.
27. Carcao M, Zak M, Abdul Karim F et al. Safety, efficacy and pharmacokinetics of nonacog beta pegol (N9-GP) in prophylaxis and treatment of bleeding episodes in previously treated pediatric hemophilia B patients. Blood 2014; 124: 1513.
28. Barnes C, Fischer K, Kulkarni R et al. Safety, Efficacy and Pharmacokinetics of Recombinant Factor IX Fc Fusion Protein in Children With Haemophilia B (Kids B-LONG). Haematology Society of Australia and New Zealand, the Australian & New Zealand Society of Blood Transfusion and the Australasian Society of Thrombosis and Haemostasis (HAA) Annual Scientific Meeting 2015.
29. Kenet G, Chambost H, Male C et al. Efficacy, pharmacokinetics (PK) and safety results of a phase 3 clinical study of recombinant fusion proteinlinking coagulation factor IX with albumin (RIX-FP) in previously treated children with hemophilia B. J Thromb Hemost 2015; 13: 227.
30. Young G, Mahlangu J, Kulkarni R et al. Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A. J Thromb Hemost 2015; 13: 967–77.