In October, Octapharma announced that previously treated patients with severe hemophilia A had begun to receive the first recombinant factor VIII (rFVIII) product derived from a human cell line (human-cl rhFVIII) at research centers in the U.S. and Germany. The purpose of the phase II clinical trial is to determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
Existing rFVIII products are manufactured using hamster cell lines. It is believed that a recombinant therapy derived from human cell lines, in this case from human embryonic kidney cells, is far less likely to prompt an inhibitor response. Inhibitors result when the immune system produces antibodies against infused clotting factor, neutralizing the protein’s therapeutic effects. They can be costly and difficult to treat.
Investigators are also measuring the pharmacokinetics, safety and immunogenicity of the company’s new therapy. Human-cl rhFVIII has been developed specifically for the control and prevention of bleeding episodes and for surgical prophylaxis in patients with hemophilia A.
Source: Octapharma press release dated October 27, 2010