Japanese clinical study of single/high dose treatment by recombinant activated factor VII for haemophilia patients with inhibitors

Abstract

Recombinant activated factor VII (rFVIIa: NovoSeven^®) is a recombinant bypassing agent developed for the use of bleeding episodes in patients with hemophilia A or B with inhibitors. Some clinical studies of single/high dose with rFVIIa were conducted in foreign courtiers, and a single dose regimen with 270μg/kg was approved in the EU in 2007. We performed an investigator led clinical trial for the single dose regimen of 270μg/kg with rFVIIa. The phase I of this trial evaluated the safety and pharmacological effects of the 270 μg/kg single dose regimen. The phase II of this trial compared the efficacy and safety of the 270μg/kg single dose regimen with a 90μg/kg×3 regimen by a multi-center, randomized, open, cross-over trial. The single dose regimen with 270μg/kg tended to have an equal to or higher efficacy than the conventional regimen of 90μg/kg×3. No safety issues were identified regarding the single dose regimen with 270μg/kg. To ease the burden of haemophilia patients with inhibitor and their families, and to improve their quality of life, we expect that the single dose regimen with 270μg/kg should be widely used in Japan like foreign countries.