Novel, human cell line-derived recombinant factor VIII (human-cl rhFVIII; Nuwiq®) in adults with severe haemophilia A: efficacy and safety

Introduction: Nuwiq (R) [human cell line-derived recombinant factor VIII (human-cl rhFVIII)] is a new generation rFVIII protein, without chemical modification or fusion to any other protein, produced in a human cell line. Aim/ methods: This prospective, open-label, multinational phase III study assessed the efficacy and safety of human-cl rhFVIII in 32 adult previously treated patients (PTPs) with severe haemophilia A during standard prophylaxis for >= 6 months and >= 50 exposure days. Efficacy in treating bleeds and during surgical prophylaxis was also assessed. Results: Prophylactic efficacy, based on mean monthly bleeding rate, was rated as ` excellent' or ` good' in 97% of patients for all bleeds and in 100% of patients for spontaneous bleeds. Mean (SD) annualized bleeding rate was 2.28 (3.73) [median = 0.9] for all bleeds, 1.16 (2.57) [median = 0] for spontaneous bleeds and 1.00 (1.79) [median = 0] for traumatic bleeds. There were no bleeds in 50% of patients and there were no major, life-threatening bleeds. Efficacy was ` excellent' or ` good' in treating 28 (100%) of 28 bleeds. Overall efficacy was rated as ` excellent' during four surgical procedures (three major, one minor) and 'moderate' during one major surgery. Incremental in vivo recovery (IVR) data were comparable with the one-stage and chromogenic assays. IVR was > 2.0% per IU kg(-1) for all measurements and stable over 6 months. No patients developed FVIII inhibitors and there were no treatment-related serious or severe adverse events. Conclusion: These results in adult PTPs indicate that human-cl rhFVIII is effective for the prevention and treatment of bleeds in adults with severe haemophilia A.