Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients

Blood. 2012 Mar 29;119(13):3031-7. doi: 10.1182/blood-2011-09-382846. Epub 2012 Jan 5.

Abstract

Current factor VIII (FVIII) products display a half-life (t(1/2)) of ∼ 8-12 hours, requiring frequent intravenous injections for prophylaxis and treatment of patients with hemophilia A. rFVIIIFc is a recombinant fusion protein composed of a single molecule of FVIII covalently linked to the Fc domain of human IgG(1) to extend circulating rFVIII t(1/2). This first-in-human study in previously treated subjects with severe hemophilia A investigated safety and pharmacokinetics of rFVIIIFc. Sixteen subjects received a single dose of rFVIII at 25 or 65 IU/kg followed by an equal dose of rFVIIIFc. Most adverse events were unrelated to study drug. None of the study subjects developed anti-rFVIIIFc antibodies or inhibitors. Across dose levels, compared with rFVIII, rFVIIIFc showed 1.54- to 1.70-fold longer elimination t(1/2), 1.49- to 1.56-fold lower clearance, and 1.48- to 1.56-fold higher total systemic exposure. rFVIII and rFVIIIFc had comparable dose-dependent peak plasma concentrations and recoveries. Time to 1% FVIII activity above baseline was ∼ 1.53- to 1.68-fold longer than rFVIII across dose levels. Each subject showed prolonged exposure to rFVIIIFc relative to rFVIII. Thus, rFVIIIFc may offer a viable therapeutic approach to achieve prolonged hemostatic protection and less frequent dosing in patients with hemophilia A. This trial was registered at www.clinicaltrials.gov as NCT01027377.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Dose-Response Relationship, Drug
  • Factor VIII / administration & dosage
  • Factor VIII / adverse effects
  • Factor VIII / pharmacokinetics*
  • Factor VIII / therapeutic use*
  • Half-Life
  • Hemophilia A / blood
  • Hemophilia A / drug therapy*
  • Hemophilia A / metabolism
  • Humans
  • Immunoglobulin Fc Fragments / administration & dosage
  • Immunoglobulin Fc Fragments / adverse effects
  • Immunoglobulin Fc Fragments / therapeutic use*
  • Infusion Pumps
  • Male
  • Metabolic Clearance Rate
  • Middle Aged
  • Receptors, Fc / administration & dosage
  • Receptors, Fc / metabolism
  • Receptors, Fc / therapeutic use*
  • Recombinant Fusion Proteins / administration & dosage
  • Recombinant Fusion Proteins / adverse effects
  • Recombinant Fusion Proteins / pharmacokinetics*
  • Recombinant Fusion Proteins / therapeutic use*
  • Time Factors
  • Young Adult
  • von Willebrand Factor / analysis

Substances

  • Immunoglobulin Fc Fragments
  • Receptors, Fc
  • Recombinant Fusion Proteins
  • factor VIII-Fc fusion protein
  • von Willebrand Factor
  • Factor VIII

Associated data

  • ClinicalTrials.gov/NCT01027377