Octapharma initiates Human-cl rhFVIII clinical study for severe hemophilia A

Octapharma, one of the largest human protein products manufacturers in the world, today announced that patients diagnosed with severe hemophilia A have started treatment with the first recombinant Factor VIII derived from a human cell line (Human-cl rhFVIII). Researchers are investigating pharmacokinetics, efficacy, safety and immunogenicity of Human-cl rhFVIII for previously treated patients with severe hemophilia A.

“Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of Human-cl rhFVIII, a Newly Developed Human Cell-Line Derived Recombinant FVIII Concentrate in Previously Treated Patients with Severe Hemophilia A.”

The prospective, randomized, actively controlled, open label, multicenter Phase 2 trial is being conducted at research centers in the US and Germany under the title "Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of Human-cl rhFVIII, a Newly Developed Human Cell-Line Derived Recombinant FVIII Concentrate in Previously Treated Patients with Severe Hemophilia A." The study started in spring 2010 and patient enrollment is well underway. Please visit www.clinicaltrials.gov for details.

Human-cl rhFVIII is a fourth-generation recombinant factor VIII (rFVIII) concentrate developed by Octapharma for the control and prevention of bleeding episodes and for surgical prophylaxis in patients with hemophilia A. Human-cl rhFVIII is produced in genetically modified Human Embryonic Kidney (HEK) 293F cells. Human-cl rhFVIII is currently the only rFVIII, which has a human glycosylation pattern. Today, the development of antibodies against infused FVIII represents the most devastating complication in modern Hemophilia A replacement therapy. The human glycosylation pattern in Human-cl rhFVIII should make it possible to avoid potentially immunogenic epitopes as expressed by hamster cells. This may result in improved safety and long-term reduced immunogenicity of Human-cl rhFVIII. During a previous first single-center clinical study, Human-cl rhFVIII demonstrated its safety and efficacy in 22 PTPs without causing any inhibitors or non-inhibitory antibodies against Human-cl rhFVIII.

Human-cl rhFVIII is concentrated and purified for virus inactivation/removal by solvent detergent and nanofiltration although the cell bank and end of production cells have been extensively tested to ensure they are free of any endogenous or infectious viruses. The process ensures that any theoretical virus contamination is safely inactivated and/or removed. The manufacturing of Human-cl rhFVIII is completely free of animal or human derived added materials. The clinical development plan for Human-cl rhFVIII follows the requirements of the U.S. Food and Drug Administration and European guidelines.