Clinical Study of Recombinant Factor VIIa (NN-007) in Hemophilia Patients with Inhibitors

Abstract

Phase I study was conducted to evaluate the pharmacokinetics and safety of two dose levels of recombinant factor Vila (rF VIIa, NN-007) in two hemophilia A patients with inhibitor. The mean half-life of rF Vila was 2.5±0.5 hours. rF VIIa shortened the activated partial thromboplastin time (APTT) and the prothrombin time (PT). No adverse events were observed.
Phase III study was conducted to evaluate the safety, efficacy and also pharmacokinetics of rF VIIa in 17 hemophilia A or B patients with inhibitor. The mean half-life of rF VIIa was 2.6±0.5 hours. Both the APTT and the PT were significantly shortened by rF VIIa. In 407 bleeding episodes treated with rF VIIa the efficacy rate of hemostatic effect was 72.7%. No adverse events including laboratory or clinical DIC were observed during the study. There was no indication of new antibody formation against rF VIIa, BHK protein, murine IgG and bovine serum protein.
These results indicate that rF VIIa is safe and effective for the hemostatic management in hemophiliacs with inhibitor.