2017 Volume 28 Issue 4 Pages 502-509
A recombinant coagulation factor IX albumin fusion protein (rIX-FP, CSL654) was developed with an improved PK profile to support a longer dosing interval for routine prophylaxis treatment. Japanese subjects have participated in a Study CSL654_3001, phase 3 open label, multinational, pivotal study of rIX-FP. Male hemophilia B patients with no inhibitor, 12–65 years of age, at least 150 exposure days with previous FIX products were eligible for enrollment. 10 of the 63 Study 3001 subjects were recruited in Japan. The mean t1/2 of rIX-FP in Japanese and all subjects were 95 h and 102 h respectively. The mean annualized spontaneous bleeding rate in Japanese and all subjects were 12.8 and 14.6 respectively for on-demand treatment, 1.8 and 0.6 respectively for weekly prophylaxis regimen, 0.4 and 1.1 respectively for every 2 weeks prophylaxis regimen. No patient developed an inhibitor and no safety concerns were identified in Study 3001. The results of Japanese subjects were consistent with those observed for the global population. rIX-FP is safe and effective for preventing bleeding episodes at weekly and every 2 weeks dosing regimens in Japanese patients with hemophilia B.
