An ultrapure plasma-derived monoclonal antibody-purified factor IX concentrate (Nonafact®), results of phase III and IV clinical studies

Nonafact®, an ultrapure, monoclonal antibody‐purified factor IX concentrate (FIX) was developed to minimize risk of thrombotic complications and viral transmission. To investigate the pharmacokinetics, efficacy and safety, phase III/IV studies were performed in the Netherlands and Poland from 1996 to 2007. The mean half‐life, in vivo response and recovery of Nonafact® were 18.7 (SD 2.0) h, 1.1 (SD 0.2) IU dL−1 per IU kg−1 b.w. of FIX infused and 49% (SD 10%), respectively. Eleven surgical procedures were performed in eight patients. During two surgeries, both high‐risk, blood loss was observed. No postoperative bleeding occurred. The in vivo recovery of FIX was higher than expected. In the phase III follow‐up study, 26 previously treated patients (PTP) were included with a median follow‐up of 1130 days. From the 1617 minor bleedings, 80.5% was stopped after a single infusion. In the phase IV study thirteen patients were treated for a median study period of 737 days. In the two follow‐up studies the investigators rated the effect of Nonafact® as excellent/good in 95% of major bleedings. Surgeries for which Nonafact® was given prophylactically were without bleeding problems. In total more than 10 million units of Nonafact® were used during almost 120 person‐years. Only one minor adverse event was reported. No inhibitors, viral transmissions and thrombogenic events occurred. In conclusion, Nonafact® is safe and provides excellent haemostasis in haemophilia B patients treated for spontaneous bleeding or undergoing surgical procedures. Due to the excellent in vivo recovery characteristic, treatment with Nonafact® is cost saving compared to other FIX products.