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

*Corresponding author for this work

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 <.0001). The median AsBR was 0.00 for all prophylaxis regimens. Overall, 98.6% of bleeding episodes were treated successfully, including 93.6% that were treated with a single injection. No patient developed an inhibitor, and no safety concerns were identified. These results indicate rIX-FP is safe and effective for preventing and treating bleeding episodes in patients with hemophilia B at dosing regimens of 40 IU/kg weekly and 75 IU/kg every 2 weeks. This trial was registered at www.clinicaltrials.gov as #NCT0101496274.

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

Funding

FundersFunder number
Japan Society for the Promotion of Science24591421

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