Preclinical pharmacology of albumin-free B-domain deleted recombinant factor VIII

Semin Thromb Hemost. 2002 Jun;28(3):269-72. doi: 10.1055/s-2002-32661.

Abstract

A second-generation recombinant factor VIII molecule was developed with an albumin-free formulation. In this modified form of factor VIII, the N- and C-terminal sections of the B-domain are retained and fused at serine 743 and glutamine 1638, resulting in a B-domain deleted factor VIII protein known as ReFacto (Genetics Institute, Andover, MA). Preclinical studies of ReFacto have focused on efficacy of the product for the hemophilia A patient population. The efficacy and pharmacokinetic profiles of ReFacto were similar to plasma-derived factor VIII in correcting the hemostatic defect of hemophilia A dogs. Both ReFacto and plasma-derived human factor VIII (Octonativ-M7, Pharmacia, Stockholm, Sweden) were found to associate with von Willebrand factor (vWF) after infusion into hemophilia A dogs as demonstrated by size exclusion chromatography. Infusion of either ReFacto or Octonativ-M7 quickly corrected factor VIII coagulant activity (FVIIIc), whole blood clotting time (WBCT), and activated partial thromboplastin time (aPTT). No obvious differences were seen between ReFacto and Octonativ-M7. Both ReFacto and Octonativ-M7 treatment reduced secondary bleeding time to less than 6 minutes. The clearance was faster and the volume of distribution at steady state was larger for plasma-derived factor VIII compared with ReFacto. The half-life was similar between Octonativ-M7 and ReFacto. These data predict that ReFacto will be effective in correcting human factor VIII deficiency states.

Publication types

  • Review

MeSH terms

  • Animals
  • Dogs
  • Drug Design
  • Drug Evaluation, Preclinical
  • Factor VIII / genetics*
  • Factor VIII / pharmacokinetics*
  • Factor VIII / pharmacology
  • Hemophilia A / drug therapy
  • Protein Engineering
  • Protein Structure, Tertiary
  • Recombinant Proteins / genetics*
  • Recombinant Proteins / pharmacokinetics*
  • Recombinant Proteins / pharmacology
  • Sequence Deletion*

Substances

  • Recombinant Proteins
  • F8 protein, human
  • Factor VIII