Sucrose formulated recombinant human antihemophilic factor VIII is safe and efficacious for treatment of hemophilia A in home therapy - Results of a multicenter, international, clinical investigation

To add an increased level of safety to antihemophilic factor replace- ment therapy, a full-length, recombinant Factor VIII (rFVIII) product has been d eveloped without human-derived plasma proteins during purification and form ulation and using an additional solvent/detergent viral inactivation step. This first clinical trial of a sucrose-formulated full-length rFVIII (rFVII I-FS) was conducted in previously treated patients (greater than or equal t o 100 prior exposure-days) with severe (<2% FVIII) hemophilia A in North Am erica (NA) and Europe (EU). Pharmacokinetic profiles for rFVIII-FS were com pared with those of currently licensed rFVIII product (Kogenate((R))) in 35 patients. Safety and efficacy during home therapy were evaluated in 71 pat ients. The new formulation displayed a pharmacokinetic profile similar to t hat of rFVIII. Patients on home therapy received a cumulative total of 11,8 67 exposure days, 12,546 infusions, and 22,443,694 IU of rFVIII-FS. Of 2585 bleeds, 93.5% were treated with 1-2 infusions and 80.5% of responses were rated as excellent or good. No evidence of de novo inhibitor formation was observed. Only 0.27% of infusions were associated wi th any drug-related ad verse event. Except for an episode of intermittent chest pain with palpitat ions which ceased after treatment with analgesics, associated adverse event s were mild or moderate. Overall, rFVIII-FS provided excellent hemostatic c ontrol, was well-tolerated, and caused no significant adverse effects, thus demonstrating safety and efficacy for treatment of bleeds in patients with hemophilia A.