Innovative approach for improved rFVIII concentrate.
The development of a new recombinant factor VIII was designed and implemented to answer a number of unmet needs of patients affected by hemophilia A. Turoctocog alfa is bioengineered in a specific Chinese hamster ovary clone to present translational and posttranslational characteristics (sulphation, glycosylation) biosimilar to natural circulating forms of FVIII, with the aim to devoid any minimal change which may impact immunogenicity and antigenicity of recombinant protein. Both producer cell line and media are maintained free of any animal or human plasma derivative. Downstream processes of purification are performed by five steps (immunoaffinity chromatography, ion-exchange chromatography, virus inactivation by means of solvent-detergent treatment and nanofiltration, and to end with gel filtration), to provide the best possible margin of safety from known and unknown infectious agents. Large clinical trials seem to confirm the expectations placed in Turoctocog alfa in terms of high quality and safety of recombinant FVIII toward the goal of overcoming actual and future challenges of hemophilia therapy.
Previous Director of Haemophilia Center, University Hospital of Florence, Via dello Statuto n.1-50129 Florence, Italy.
Massimo
M
Morfini
D000818:Animals; D054316:Biomarkers, Pharmacological; D016466:CHO Cells; D003412:Cricetulus; D004345:Drug Industry; D005169:Factor VIII / Q000096:biosynthesis / Q000235:genetics / Q000302:isolation & purification* / Q000627:therapeutic use; D015870:Gene Expression; D006031:Glycosylation; D006467:Hemophilia A / Q000188:drug therapy; D006801:Humans; D017410:Practice Guidelines as Topic; D046188:Protein Modification, Translational; D011499:Protein Processing, Post-Translational; D011994:Recombinant Proteins / Q000096:biosynthesis / Q000235:genetics / Q000302:isolation & purification / Q000627:therapeutic use; D013431:Sulfates
D054316:Biomarkers, Pharmacological; D011994:Recombinant Proteins; D013431:Sulfates; C577506:recombinant factor VIII N8; D005169:Factor VIII
Turoctocog alfa; haemophilia A; inhibitor; recombinant factor VIII; safety