TUROCTOCOG ALFA FOR THE TREATMENT OF HEMOPHILIA A

Patients with hemophilia A lack functional coagulation factor VIII (FVIII), causing excessive spontaneous bleeding episodes and bleeding during trauma or surgery. Plasma-derived or synthetic recombinant FVIII is used to control bleeding, but currently available treatments are limited because of patient development of FVIII inhibitors and cross-contamination of human and animal-derived infectious agents. Turoctocog alfa is a recombinant FVIII pro-duced in Chinese hamster ovary cells that has demonstrated good efficacy in thrombin generation and clot formation in preclinical evaluations in murine and canine models of hemophilia A and in patient-derived whole blood. Evaluation in clinical trials in pediatric and adult patients with hemophilia A have demonstrated that the drug has good pharmacokinetic parameters, excellent efficacy in improving annualized bleeding rates, few adverse events and no induction of FVIII inhibitors. The drug is FDA approved as a prophylactic agent and for on-demand treatment to control bleeding including during operative procedures.