Clinical utility and patient perspectives on the use of extended half-life rFIXFc in the management of hemophilia B

Patient Preference and Adherence

Volumn 8, Issue , 2014, Pages 1073-1083

  Miguelino, Maricel G ^a   Powell, Jerry S ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Extended half life FIX; Factor IX; Genetic coagulation defects; Hemophilia B; Prophylaxis

Indexed keywords

ANTITHROMBIN; BLOOD CLOTTING FACTOR 7A; BLOOD CLOTTING FACTOR 9; IMMUNOGLOBULIN FC FRAGMENT; MACROGOL; NEW DRUG; RECOMBINANT BLOOD CLOTTING FACTOR 9; TISSUE FACTOR PATHWAY INHIBITOR;

BIOCHEMISTRY; BIOENGINEERING; BLEEDING; CAUSE OF DEATH; CHEMICAL PHENOMENA; DISEASE SEVERITY; DRUG APPROVAL; DRUG EFFICACY; DRUG HALF LIFE; DRUG INFUSION; DRUG SYNTHESIS; DRUG TARGETING; DRUG TOLERABILITY; DRUG USE; ENDOTHELIUM CELL; HEMOPHILIA B; HUMAN; MAJOR SURGERY; NONHUMAN; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PHYSICAL ACTIVITY; PROTEIN FUNCTION; REVIEW;

EID: 84936935010     PISSN: None     EISSN: 1177889X     Source Type: Journal    
DOI: 10.2147/PPA.S54951     Document Type: Review

References (80)

1. Powell JS, Rodgers GM. Inherited coagulation disorders. In: Greer JP, Arber DA, Glader B, et al, editors. Wintrobe's Clinical Hematology. Philadelphia, PA: Wolters Kluwer; 2014:1143-1185.
2. Giangrande P. Haemophilia B: Christmas disease. Expert Opin Pharmacother. 2005;6(9):1517-1524.
3. Mannucci PM, Mendolicchio L, Gringeri A. Use of prophylaxis to prevent complications of hemophilia. Adv Exp Med Biol. 2001;489:59-64.
4. Nilsson IM, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med. 1992;232(1):25-32.
5. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007;357(6):535-544.
6. Darby SC, Kan SW, Spooner RJ, et al. Mortality rates, life expectancy, and causes of death in people with hemophilia A or B in the United Kingdom who were not infected with HIV. Blood. 2007;110(3):815-825.
7. White GC 2nd, Courter S, Bray GL, Lee M, Gomperts ED. A multicenter study of recombinant factor VIII (Recombinate) in previously treated patients with hemophilia A. Thromb Haemost. 1997;77(4):660-667.
8. Feldman BM, Pai M, Rivard GE, et al. Tailored prophylaxis in severe hemophilia A: interim results from the first 5 years of the Canadian Hemophilia Primary Prophylaxis Study. J Thromb Haemost. 2006;4(6):1228-1236.
9. Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy in haemophilia. Haemophilia. 2001;7(4):392-396.
10. Manco-Johnson MJ, Riske B, Kasper CK. Advances in care of children with hemophilia. Semin Thromb Hemost. 2003;29(6):585-594.
11. Gater A, Thomson TA, Strandberg-Larsen M. Haemophilia B: impact on patients and economic burden of disease. Thromb Haemost. 2011; 106(3):398-404.
12. Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011;365(25):2357-2365.
13. Negrier C, Knobe K, Tiede A, Giangrande P, Moss J. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood. 2011;118(10):2695-2701.
14. 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(6):881-887.
15. Shapiro AD, Ragni MV, Valentino LA, et al. Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a Phase I/IIa study in hemophilia B patients. Blood. 2012;119(3):666-672.
16. Metzner HJ, Pipe SW, Weimer T, Schulte S. Extending the pharmacokinetic half-life of coagulation factors by fusion to recombinant albumin. Thromb Haemost. 2013;110(5):931-939.
17. Peters RT, Low SC, Kamphaus GD, et al. Prolonged activity of factor IX as a monomeric Fc fusion protein. Blood. 2010;115(10):2057-2064.
18. Santagostino E, Negrier C, Klamroth R, et al. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood. 2012;120(12):2405-2411.
19. Schmidt SR. Fusion-proteins as biopharmaceuticals-applications and challenges. Curr Opin Drug Discov Devel. 2009;12(2):284-295.
20. Huang C. Receptor-Fc fusion therapeutics, traps, and MIMETIBODY technology. Curr Opin Biotechnol. 2009;20(6):692-699.
21. Borel Y, Fritsche R, Borel H, Dahlgren U, Dalhman-Hoglund A, Hanson LA. Parenteral and oral administration of tolerogens: protein-IgG conjugates. Ann N Y Acad Sci. 1996;778:80-87.
22. Borel H, Borel Y. A novel technique to link either proteins or peptides to gammaglobulin to construct tolerogens. J Immunol Methods. 1990;126(2):159-168.
23. Roopenian DC, Akilesh S. FcRn: the neonatal Fc receptor comes of age. Nat Rev Immunol. 2007;7(9):715-725.
24. Rath T, Baker K, Dumont JA, et al. Fc-fusion proteins and FcRn: structural insights for longer-lasting and more effective therapeutics. Crit Rev Biotechnol. Epub 2013 Oct 24.
25. Kontermann RE. Strategies for extended serum half-life of protein therapeutics. Curr Opin Biotechnol. 2011;22(6):868-876.
26. Lusson J, Vieau D, Hamelin J, Day R, Chretien M, Seidah NG. cDNA structure of the mouse and rat subtilisin/kexin-like PC5: a candidate proprotein convertase expressed in endocrine and nonendocrine cells. Proc Natl Acad Sci U S A. 1993;90(14):6691-6695.
27. Bond M, Jankowski M, Patel H, et al. Biochemical characterization of recombinant factor IX. Semin Hematol. 1998;35(2 Suppl 2):11-17.
28. Lissitchkov T, Matysiak M, Zavilska K, et al. Head-to-head comparison of the pharmacokinetic profiles of a high-purity factor IX concentrate (AlphaNine®) and a recombinant factor IX (BeneFIX®) in patients with severe haemophilia B. Haemophilia. 2013;19(5):674-678.
29. 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(24):2313-2323.
30. 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(3):233-243.
31. Roth DA, Kessler CM, Pasi KJ, Rup B, Courter SG, Tubridy KL. Human recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentrates. Blood. 2001;98(13):3600-3606.
32. van den Berg HM, Fischer K, van der Bom JG. Comparing outcomes of different treatment regimens for severe haemophilia. Haemophilia. 2003;9(Suppl 1):27-31.
33. Lillicrap D. Improvements in factor concentrates. Curr Opin Hematol. 2010;17(5):393-397.
34. Hoffman M, Whinna HC, Monroe DM. Circulating tissue factor accumulates in thrombi, but not in hemostatic plugs. J Thromb Haemost. 2006;4(9):2092-2093.
35. Carlsson M, Bjorkman S, Berntorp E. Multidose pharmacokinetics of factor IX: implications for dosing in prophylaxis. Haemophilia. 1998;4(2):83-88.
36. Kisker CT, Eisberg A, Schwartz B. Prophylaxis in factor IX deficiency product and patient variation. Haemophilia. 2003;9(3):279-284.
37. Bjorkman S. Prophylactic dosing of factor VIII and factor IX from a clinical pharmacokinetic perspective. Haemophilia. 2003;9(Suppl 1):101-108.
38. Ahnstrom J, Berntorp E, Lindvall K, Bjorkman S. A 6-year follow-up of dosing, coagulation factor levels and bleedings in relation to joint status in the prophylactic treatment of haemophilia. Haemophilia. 2004;10(6):689-697.
39. Weisman R. Biogen Idec prices its newly approved hemophilia drug. The Boston Globe. 2014 April 19.
40. Skinner MW, Myles E. World Federation of Hemophilia: 50 years of advancing treatment for all. Haemophilia. 2013;19(4):475-480.
41. Weimer T, Wormsbacher W, Kronthaler U, Lang W, Liebing U, Schulte S. Prolonged in-vivo half-life of factor VIIa by fusion to albumin. Thromb Haemost. 2008;99(4):659-667.
42. Schulte S. Innovative coagulation factors: albumin fusion technology and recombinant single-chain factor VIII. Thromb Res. 2013;131(Suppl 2):S2-S6.
43. 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-S10.
44. Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2013;123(3):317-325.
45. Scott DW, Pratt KP, Miao CH. Progress toward inducing immunologic tolerance to factor VIII. Blood. 2013;121(22):4449-4456.
46. Gui T, Lin HF, Jin DY, et al. Circulating and binding characteristics of wild-type factor IX and certain Gla domain mutants in vivo. Blood. 2002;100(1):153-158.
47. Abuchowski A, van Es T, Palczuk NC, Davis FF. Alteration of immunological properties of bovine serum albumin by covalent attachment of polyethylene glycol. J Biol Chem. 1977;252(11):3578-3581.
48. Caliceti P, Veronese FM. Pharmacokinetic and biodistribution properties of poly(ethylene glycol)-protein conjugates. Adv Drug Deliv Rev. 2003;55(10):1261-1277.
49. Shen BW, Speigel PC, Chang CH, et al. The tertiary structure and domain organization of coagulation factor VIII. Blood. 2008;111(3):1240-1247.
50. Ngo JC, Huang M, Roth DA, Furie BC, Furie B. Crystal structure of human factor VIII: implications for the formation of the factor IXa-factor VIIIa complex. Structure. 2008;16(4):597-606.
51. Mei B, Pan C, Jiang H, et al. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment. Blood. 2010;116(2):270-279.
52. Horton S, Walsh C, Emery P. Certolizumab pegol for the treatment of rheumatoid arthritis. Expert Opin Biol Ther. 2012;12(2):235-249.
53. Alconcel SNS, Baas AS, Maynard HD. FDA-approved poly(ethylene glycol)-protein conjugate drugs. Polym Chem. 2011;2(7):1442-1448.
54. Ivens IA, Baumann A, McDonald TA, Humphries TJ, Michaels LA, Mathew P. PEGylated therapeutic proteins for haemophilia treatment: a review for haemophilia caregivers. Haemophilia. 2013;19(1):11-20.
55. Tang L, Leong L, Sim D, et al. von Willebrand factor contributes to longer half-life of PEGylated factor VIII in vivo. Haemophilia. 2013;19(4):539-545.
56. 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 Haemostat. 2014;12(4):488-496.
57. Turecek PL, Bossard MJ, Graninger M, et al. BAX 855, a PEGylated rFVIII product with prolonged half-life. Development, functional, and structural characterisation. Hamostaseologie. 2012;32(Suppl 1): S29-S38.
58. Tiede A, Brand B, Fischer R, et al. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost. 2013;11(4):670-678.
59. Novo Nordisk A/S. Novo Nordisk reports positive results from first phase 3 trial with N8-GP, a long-acting factor VIII for treatment of haemophilia A [company announcement]. Bagsværd: Novo Nordisk A/S; 2014 [March 19]. Available from: http://www.novonordisk.com/include/asp/exe_news_attachment.asp?sAttachmentGUID=31c49463-5e29-4262-9e4d-f8c47e96a57b. Accessed July 18, 2014.
60. Sandberg H, Kannicht C, Stenlund P, et al. Functional characteristics of the novel, human-derived recombinant FVIII protein product, human-cl rhFVIII. Thromb Res. 2012;130(5):808-817.
61. Kannicht C, Ramstrom M, Kohla G, et al. Characterisation of the post-translational modifications of a novel, human cell line-derived recombinant human factor VIII. Thromb Res. 2013;131(1):78-88.
62. Schulte S. Challenges for new haemophilia products from a manufacturer's perspective. Thromb Res. Epub 2013 Dec 21.
63. Zollner SB, Raquet E, Muller-Cohrs J, et al. Preclinical efficacy and safety of rVIII-SingleChain (CSL627), a novel recombinant single-chain factor VIII. Thromb Res. 2013;132(2):280-287.
64. Zollner S, Schuermann D, Raquet E, et al. Pharmacological characteristics of a novel, recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP). J Thromb Haemost. 2014;12(2): 220-228.
65. 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 Haemostat. 2014;12(2):206-213.
66. Hilden I, Lauritzen B, Sorenson BB, et al. Hemostatic effect of a monoclonal antibody mAb 2021 blocking the interaction between FXa and TFPI in a rabbit hemophilia model. Blood. 2012;119(24):5871-5878.
67. Agerso H, Overgaard RV, Peterson MB, et al. Pharmacokinetics of an anti-TFPI monoclonal antibody (concizumab) blocking the TFPI interaction with the active site of FXa in cynomolgus monkeys after iv and sc administration. Eur J Pharm Sci. 2014;56:65-69.
68. Gorczyca ME, Nair SC, Jilma B, et al. Inhibition of tissue factor pathway inhibitor by the aptamer BAX499 improves clotting of hemophilic blood and plasma. J Thromb Haemost. 2012;10(8):1581-1590.
69. Prasad S, Lillicrap D, Labelle A, et al. Efficacy and safety of a new-class hemostatic drug candidate, AV513, in dogs with hemophilia A. Blood. 2008;111(2):672-679.
70. Knappe S, Gorczyca ME, Jilma B, et al. Plasmatic tissue factor pathway inhibitor is a major determinant of clotting in factor VIII inhibited plasma or blood. Thromb Haemost. 2013;109(3):450-457.
71. Peraramelli S, Thomassen S, Heinzmann A, et al. Direct inhibition of factor VIIa by TFPI and TFPI constructs. J Thromb Haemost. 2013; 11(4):704-714.
72. Dockal M, Hartmann R, Fries M, et al. Small peptides blocking inhibition of factor Xa and tissue factor-factor VIIa by tissue factor pathway inhibitor (TFPI). J Biol Chem. 2014;289(3):1732-1741.
73. Bolliger D, Szlam F, Suzuki N, Matsushita T, Tanaka KA. Heterozygous antithrombin deficiency improves in vivo haemostasis in factor VIII-deficient mice. Thromb Haemost. 2010;103(6):1233-1238.
74. Peyvandi F, Garagiola I, Seregni S. Future of coagulation factor replacement therapy. J Thromb Haemost. 2013;11(Suppl 1):84-98.
75. Sehgal A, Vaishnaw A, Fitzgerald K. Liver as a target for oligonucleotide therapeutics. J Hepatol. 2013;59(6):1354-1359.
76. Akinc A, Sehgal A, Hettinger J, et al. An RNAi Therapeutic Targeting Antithrombin Increases Thrombin Generation in Nonhuman Primates. Available from: http://www.alnylam.com/web/wp-content/uploads/2012/12/ALNY-ASH-AT3posterXpanel-Dec2012-r.pdf. Accessed 18 July, 2014.
77. Konkle BA, Ebbesen LS, Erhardtsen E, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost. 2007;5(9):1904-1913.
78. Lentz SR, Ehrenforth S, Karim FA, et al. Recombinant factor VIIa analogue in management of hemophilia with inhibitors: results from a multicenter, randomized, controlled trial of vatreptacog alfa. J Thromb Haemost. Epub 2014 Jun 16.
79. Mahlangu JN, Koh PL, Ng HJ, Lissitchkov T, Hardtke M, Schroeder J. The TRUST Trial: Anti-Drug Antibody Formation In a Patient With Hemophilia With Inhibitors After Receiving The Activated Factor VII Product Bay 86-6150. Blood. 2013;122(21):573.
80. Parunov LA, Soshitova NP, Fadeeva OA, et al. Drug-drug interaction of the anti-TFPI aptamer BAX499 and factor VIII: Studies of spatial dynamics of fibrin clot formation in hemophilia A. Thrombo Res. 2014;133(1):112-119.