Clinical Trial Information

To add an increased level of safety to antihemophilic factor replacement therapy, Bayer's full-length, recombinant Factor VIII (rFVIII) product has been developed with an additional solvent/detergent virus inactivation step, without using human-derived plasma proteins during purification and formulation stages.

Results from multicenter, international clinical investigations demonstrated that this sucrose formulated recombinant antihemophilic Factor VIII is safe and efficacious for the treatment of hemophilia A in home therapy, demonstrating a pharmacokinetic profile similar to that of rFVIII.^{1, 2}

Further, tolerability studies in previously untreated and minimally treated patients show that inhibitor incidence is in the low end of the range compared to that observed for other recombinant or plasma-derived products.³

Overall, rFVIII-FS provided excellent hemostatic control, was well tolerated, and caused no significant adverse effects, thus demonstrating safety and efficacy for treatment of bleeds in patients with hemophilia A.¹

The following clinical trials were conducted to support the efficacy, safety, and tolerability of Kogenate^® FS.

Sucrose formulated recombinant human antihemophilic Factor VIII is safe and efficacious for treatment of hemophilia A in home therapy¹

Aim: To compare the pharmacokinetic profiles of a sucrose-formulated full-length rFVIII (Kogenate^® FS) to those of an rFVIII product (Kogenate^®) in previously treated patients ( 100 prior exposure days) with severe (<2% FVIII) hemophilia A in North America (NA) and Europe (EU).

Study Design: Three-staged, open-labeled trial. Stage I evaluated bioequivalence of Kogenate^® FS and rFVIII, in a randomized, crossover, pharmacokinetic and safety trial in 35 patients. Stage II/III examined safety and efficacy of Kogenate FS in home therapy for a total of 24 weeks, including an initial mandatory period of prophylaxis. A total of 71 patients (34 Stage I patients and 37 new patients) were enrolled in these two stages. A Stage IIIE monitored Stage III patients for an additional 12 to 18 months of Kogenate^® FS home therapy in an extended analysis of safety and efficacy. The primary efficacy endpoint was number of treatment responses.

Data: Patients on home therapy received a cumulative total of 11,867 exposure days, 12,546 infusions, and 22,443,694 IU of Kogenate^® FS. Of 2,585 bleeds, 93.5% were treated with 1-2 infusions and 80.5% of responses were rated as excellent or good.

Conclusion: The new formulation displayed a pharmacokinetic profile similar to that of rFVIII. Overall, Kogenate^® FS provided excellent hemostatic control, was well tolerated, and caused no significant adverse effects, thus demonstrating safety and efficacy for treatment of bleeds in patients with hemophilia A. No evidence of de novo inhibitor formation was observed.

Safety and Efficacy of a New Recombinant FVIII Formulated With Sucrose (rFVIII-FS) in Patients With Haemophilia A: A Long-Term, Multicentre Clinical Study in Japan²

Aim:To assess the in vivo recovery, haemostatic efficacy, and safety of Kogenate^® FS in previously treated patients.

Study Design: Long-term, multicenter clinical trial conducted in 20 patients with severe or moderate hemophilia A, in Japan.

Data: A total number of 1,115 infusions were included in the analysis, and adequate hemostasis was achieved with one or two infusions in 88.7% of all bleeding episodes.

Conclusion: Kogenate^® FS is safe and effective for long-term treatment of patients with haemophilia A.

Outcome of a prospective, multinational clinical trial in children treated with a full-length, sucrose formulated rFVIII product³

Aim:To assess the safety and clinical efficacy of a second generation formulation of a full-length recombinant Factor VIII (Kogenate^® FS) in previously untreated patients and minimally treated hemophilia A patients.

Study Design: Long-term, prospective clinical trials conducted in 61 patients with severe hemophilia A (<2% FVIII activity), over 45 months in North America (NA: n=31) and Europe (EU: n=30).

Data: Total number of bleeding episodes in NA and EU combined were 1,173. 15% of the patients exhibited de novo inhibitor development.

Conclusion: Kogenate^® FS demonstrated excellent hemostatic response in previously untreated patients and minimally treated patients. Inhibitor incidence was 16% in PUPs.