Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: Results of a phase 3 trial

Elena Santagostino, Uri Martinowitz, Toshko Lissitchkov, Brigitte Pan-Petesch, Hideji Hanabusa, Johannes Oldenburg, Lisa Boggio, Claude Negrier, Ingrid Pabinger, Mario Von Depka Prondzinski, Carmen Altisent, Giancarlo Castaman, Koji Yamamoto, Maria Teresa Álvarez-Roman, Christine Voigt, Nicole Blackman, Iris Jacobs

Research output: Contribution to journalArticlepeer-review

Abstract

A global phase 3 study evaluated the pharmacokinetics, efficacy, and safety of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in 63 previously treated male patients (12-61 years) with severe hemophilia B (factor IX [FIX] activity ≤2%). The study included 2 groups: group 1 patients received routine prophylaxis once every 7 days for 26 weeks, followed by either 7-, 10-, or 14-day prophylaxis regimen for a mean of 50, 38, or 51 weeks, respectively; group 2 patients received on-demand treatment of bleeding episodes for 26 weeks and then switched to a 7-day prophylaxis regimen for a mean of 45 weeks. The mean terminal half-life of rIX-FP was 102 hours, 4.3-fold longer than previous FIX treatment. Patients maintained a mean trough of 20 and 12 IU/dL FIX activity on prophylaxis with rIX-FP 40 IU/kg weekly and 75 IU/kg every 2 weeks, respectively. There was 100% reduction in median annualized spontaneous bleeding rate (AsBR) and 100% resolution of target joints when subjects switched from on-demand to prophylaxis treatment with rIX-FP (P

Original languageEnglish
Pages (from-to)1761-1769
Number of pages9
JournalBlood
Volume127
Issue number14
DOIs
Publication statusPublished - Apr 7 2016

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

Fingerprint Dive into the research topics of 'Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: Results of a phase 3 trial'. Together they form a unique fingerprint.

Cite this