Efficacy and safety of a recombinant factor VIII produced from a human cell line (simoctocog alfa)

References

• Mannucci PM, Tuddenham EG. The hemophilias–from royal genes to gene therapy. N Engl J Med. 2001;344:1773–1779.
   PubMed Web of Science ®Google Scholar
• Berntorp E, Shapiro AD. Modern haemophilia care. Lancet. 2012;379:1447–1456.
   PubMed Web of Science ®Google Scholar
• White GC, Rosendaal F, Aledort LM, et al. Definitions in hemophilia. Recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 2001;85:560.
   PubMed Web of Science ®Google Scholar
• Franchini M, Mannucci PM. Past, present and future of hemophilia: a narrative review. Orphanet J Rare Dis. 2012;7:24.
   PubMed Web of Science ®Google Scholar
• Mannucci PM, Franchini M. Present and future challenges in the treatment of haemophilia: a clinician’s perspective. Blood Transfus. 2013;11(Suppl 4):s77–81.
   PubMed Web of Science ®Google Scholar
• Franchini M, Mannucci PM. Hemophilia A in the third millennium. Blood Rev. 2013 Jul;27(4):179–184.
   PubMed Web of Science ®Google Scholar
• 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;357:535–544.
   PubMed Web of Science ®Google Scholar
• Gringeri A, Lundin B, von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011;9:700–710.
   PubMed Web of Science ®Google Scholar
• Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388:187–197.
   PubMed Web of Science ®Google Scholar
• Franchini M, Mannucci PM. The safety of pharmacologic options for the treatment of persons with hemophilia. Expert Opin Drug Saf. 2016;15:1391–1400. [Epub ahead of print]
   PubMed Web of Science ®Google Scholar
• Mannucci PM. Back to the future: a recent history of haemophilia treatment. Haemophilia. 2008;14(Suppl. 3):10–18.
   PubMed Web of Science ®Google Scholar
• Coppola A, Santoro C, Tagliaferri A, et al. Understanding inhibitor development in haemophilia A: towards clinical prediction and prevention strategies. Haemophilia. 2010;16(Suppl. 1):13–19.
   PubMed Web of Science ®Google Scholar
• Franchini M, Mannucci PM. Inhibitors of propagation of coagulation (factors VIII, IX and XI): a review of current therapeutic practice. Br J Clin Pharmacol. 2011;72:553–562.
   PubMed Web of Science ®Google Scholar
• Gringeri A, Mantovani LG, Scalone L, et al.; COCIS Study Group. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS study group. Blood. 2003;102:2358–2363.
   PubMed Web of Science ®Google Scholar
• Mannucci PM, Mancuso ME, Franchini M. Tailoring hemostatic therapies to lower inhibitor development in previously untreated patients with severe hemophilia. J Thromb Haemost. 2016;14:1330–1336.
   PubMed Web of Science ®Google Scholar
• Franchini M, Lippi G. Prevention of inhibitor development in hemophilia A in 2016. A glimpse into the future? Thromb Res. 2016;148:96–100.
   PubMed Web of Science ®Google Scholar
• Lillicrap D, Fijnvandraat K, Santagostino E. Inhibitors – genetic and environmental factors. Haemophilia. 2014;20(Suppl. 4):87–93.
   PubMed Web of Science ®Google Scholar
• Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII and neutralizing antibodies in hemophilia A. N Engl J Med. 2016;374:2054–2064.
   PubMed Web of Science ®Google Scholar
• 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;131:78–88.
   PubMed Web of Science ®Google Scholar
• Casademunt E, Martinelle K, Jernberg M, et al. The first recombinant human coagulation factor VIII of human origin: human cell line and manufacturing characteristics. Eur J Haematol. 2012;89:165–176.
   PubMed Web of Science ®Google Scholar
• 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;130:808–817.
   PubMed Web of Science ®Google Scholar
• Winge S, Yderland L, Kannicht C, et al. Development, upscaling and validation of the purification process for human-cl rhFVIII (Nuwiq), a new generation recombinant factor VIII produced in a human cell-line. Protein Expr Purif. 2015;115:165–175.
   PubMed Web of Science ®Google Scholar
• Valentino LA, Negrier C, Kohla G, et al. The first recombinant FVIII produced in human cells –an update on its clinical development programme. Haemophilia. 2014;20(Suppl. 1):1–9.
   PubMed Web of Science ®Google Scholar
• Klukowska A, Szczepański T, Vdovin V, et al. Novel, human cell line-derived recombinant factor VIII (Human-cl rhFVIII, Nuwiq) in children with severe haemophilia A: efficacy, safety and pharmacokinetics. Haemophilia. 2015 Sep 14. doi:10.1111/hae.12797. [Epub ahead of print].
   Web of Science ®Google Scholar
• European Medicine Agency (EMA). Nuwiq: European Public Assessment Report (EPAR) – product information. Available from: http://www.ema.europa.eu/docs/en_GB/document_ library/EPAR_-_Product_Information/human/002813/WC500179340.pdf
   Google Scholar
• Hironaka T, Furukawa K, Esmon PC, et al. Comparative study of the sugar chains of factor VIII purified from human plasma and from the culture media of recombinant baby hamster kidney cells. J Biol Chem. 1992;267:8012–8020.
   PubMed Web of Science ®Google Scholar
• Tiede A, Oldenburg J, Lissitchkov T, et al. Prophylaxis vs. on-demand treatment with Nuwiq (Human-cl rhFVIII) in adults with severe haemophilia A. Haemophilia. 2016;22:374–380.
   PubMed Web of Science ®Google Scholar
• Lissitchkov T, Hampton K, von Depka M, et al. Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq) in adults with severe haemophilia A: efficacy and safety. Haemophilia. 2016;22:225–231.
   PubMed Web of Science ®Google Scholar
• Food and Drug Administration (FDA). Statistical review – Nuwiq. Available from: http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM464113.pdf
   Google Scholar
• Klamroth R, Lissitchkov T, Rusen L, et al. Personalized prophylaxis with human-cl rhFVIII (Nuwiq) in haemophilia A patients. J Thromb Haemost. 2015;13(Suppl. 2):850-1 (abstract).
   Google Scholar
• Food and Drug Administration (FDA). Summary basis for regulatory action – Nuwiq. Available from: http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM464018.pdf
   Google Scholar
• Medical Dictionary for Regulatory Activities (MedDRA). Available from: www.meddra.org
   Google Scholar
• Klukowska A, Vdovin V, Szczepanski T, et al. Long-term immunogenicity, safety and efficacy of human-cl rhFVIII in previously treated children with severe hemophilia A. Haemophilia. 2016;22(Suppl. 2):96 (abstract).
   PubMed Web of Science ®Google Scholar
• Franchini M. The modern treatment for hemophilia: a narrative review. Blood Transfus. 2013;11:178–182.
   PubMed Web of Science ®Google Scholar
• Franchini M, Lippi G. Recombinant FVIII products. Semin Thromb Hemost. 2010;36:537–549.
   PubMed Web of Science ®Google Scholar
• Carcao M, Re W, Ewenstein B. The role of previously untreated patient studies in understanding the development of FVIII inhibitors. Haemophilia. 2016;22:22–31.
   PubMed Web of Science ®Google Scholar
• Liesner R, Abashidze M, Aleinikova O, et al.Inhibitor development in previously untreated patients with severe hemophilia A treated with Nuwiq, a new generation recombinant FVIII of human origin. Blood. 2016;128:327. (abstract)
   Google Scholar
• Franchini M, Makris M, Santagostino E, et al. Non-thrombotic-, non-inhibitor-associated adverse reactions to coagulation factor concentrates for treatment of patients with hemophilia and von Willebrand’s disease: a systematic review of prospective studies. Haemophilia. 2012;18:e164–72.
   PubMed Web of Science ®Google Scholar
• Coppola A, Franchini M, Makris M, et al. Thrombotic adverse events to coagulation factor concentrates for treatment of patients with haemophilia and von Willebrand disease: a systematic review of prospective studies. Haemophilia. 2012;18:e173–87.
   PubMed Web of Science ®Google Scholar
• Mannucci PM, Garagiola I. Factor VIII products in haemophilia A: one size fits all? Thromb Haemost. 2015;113:911–914.
   PubMed Web of Science ®Google Scholar
• Kruse-Jarres R. Inhibitors: our greatest challenge. Can we minimize the incidence? Haemophilia. 2013;19(Suppl. 1):2–7.
   PubMed Web of Science ®Google Scholar
• Goudemand J, Peyvandi F, Lacroix-Desmazes S. Key insights to understand the immunogenicity of FVIII products. Thromb Haemost. 2016;116(Suppl 1):S2–9.
   PubMed Web of Science ®Google Scholar
• Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia. 2003;9:418–435.
   PubMed Web of Science ®Google Scholar
• Iorio A, Halimeh S, Holzhauer S, et al. Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost. 2010;8:1256–1265.
   PubMed Web of Science ®Google Scholar
• Franchini M, Tagliaferri A, Mengoli C, et al. Cumulative inhibitor incidence in previously untreated patients with severe hemophilia A treated with plasma-derived versus recombinant factor VIII concentrates: a critical systematic review. Crit Rev Oncol Hematol. 2012;81:82–93.
   PubMed Web of Science ®Google Scholar
• Mannucci PM, Shi Q, Bonanad S, et al. Novel investigations on the protective role of the FVIII/VWF complex in inhibitor development. Haemophilia. 2014;20(Suppl 6):2–16.
   PubMed Web of Science ®Google Scholar