Recombinant factor IX

Thromb Haemost. 1997 Jul;78(1):261-5.

Abstract

Despite the introduction of recombinant preparations of factor VIII and recombinant factor VII and VIIa, patients with other forms of hemophilia, especially hemophilia B, have remained at increased risk for blood borne viruses because of a lack of clinically utilizable preparations of recombinant factor IX. This report describes the state of current tests with a recently licensed preparation of recombinant factor IX, BeneFix, from Genetics Institute. Structurally, functionally, and therapeutically, recombinant factor IX is comparable to monoclonal plasma-derived factor IX. The only observed difference between recombinant and plasma factor IX is the recovery in pharmacokinetic studies where recombinant factor IX recovery was approximately 72% that of a plasma factor IX product. This difference is attributed to be due to minor differences in the post-translational modification of recombinant factor IX compared to plasma. These studies demonstrate that recombinant factor IX is effective in the treatment of hemophilia B and has the safety profile expected from a product prepared by recombinant technology.

Publication types

  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Animals
  • Drug Evaluation, Preclinical
  • Factor IX / biosynthesis
  • Factor IX / isolation & purification
  • Factor IX / therapeutic use*
  • Hemophilia B / drug therapy
  • Hemophilia B / metabolism
  • Humans
  • Protein Processing, Post-Translational
  • Randomized Controlled Trials as Topic
  • Recombinant Proteins / biosynthesis
  • Recombinant Proteins / isolation & purification
  • Recombinant Proteins / therapeutic use

Substances

  • Recombinant Proteins
  • Factor IX