Turoctocog alfa (recombinant factor VIII). Manufacturing, characteristics and clinical trial results.
Turoctocog alfa (NovoEight®) is a new recombinant factor VIII (rFVIII) with a truncated B domain and a high degree of tyrosine sulphation, similar to plasma-derived FVIII products. The manufacturing process includes double nanofiltration with a 20-nm pore size and immunoaffinity chromatography with monoclonal F25 anti-FVIII antibodies. Treatment with turoctocog alfa can be monitored with both one-stage and chromogenic substrate assays without a product-specific laboratory standard. In total, 213 previously-treated patients with severe haemophilia A participated in the pivotal part of the clinical trial programme guardianTM. The median annualised bleeding rate during turoctocog alfa prophylaxis was 3.7 and 3.0 in adolescents/adults and children, respectively, with marked differences between participating countries. The success rate for the treatment of breakthrough bleeds was 85% (adults/adolescents) and 94% (children). A total of 41 surgical procedures (15 major, 26 minor) were performed in 33 patients, with a successful haemostatic response reported in all cases. No patient developed confirmed inhibitors in any of the trials.
Prof. Dr. med. Andreas Tiede, Department of Haematology, Haemostasis, Oncology and Stem-Cell Transplantation, Hannover Medical School, Carl-Neuberg-Str. 1, 30625 Hannover, Germany Tel. +49/(0)511/532 84 88, Fax +49/(0)511/532 41 46, tiede.andreas@mh-hannover.de.
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Tiede
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Klamroth
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Oldenburg
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D004305:Dose-Response Relationship, Drug; D004339:Drug Compounding / Q000379:methods*; D019317:Evidence-Based Medicine; D005169:Factor VIII / Q000008:administration & dosage* / Q000138:chemical synthesis* / Q000235:genetics; D005260:Female; D006467:Hemophilia A / Q000175:diagnosis / Q000188:drug therapy* / Q000517:prevention & control*; D006490:Hemostatics / Q000008:administration & dosage / Q000138:chemical synthesis; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D015202:Protein Engineering / Q000379:methods*; D016896:Treatment Outcome
D006490:Hemostatics; C577506:recombinant factor VIII N8; D005169:Factor VIII
Factor VIII; NovoEight™; haemophilia A; turoctocog alfa