Abstract
Turoctocog alfa is the first B-domain-truncated third generation recombinant coagulation factor VIII (FVIII) product. Nonclinical in vitro and animal model studies have demonstrated that turoctocog alfa has similar functional potency and hemostatic efficacy as comparator FVIII products. With respect to discrepancies in the level of FVIII concentrate in plasma of current FVIII products on comparing measurement results between one-stage clot and chromogenic assays, there was no difference in the in vitro turoctocog alfa study; however, measured FVIII concentrate in field study was higher with the chromogenic assay (1.08 IU/mL) than with one-stage assay (0.83 IU/mL). Two published clinical studies on previously treated patients (PTPs) and clinical pharmacokinetics have described that the pharmacokinetic parameters are similar, and the safety and efficacy for prevention and treatment for bleeding are also similar to those of standard half-life FVIII products. Three clinical trials are ongoing to assess the long-term safety and efficacy of turoctocog alfa for PTPs and previously untreated patients. Those data will be published in the near future, and it will be possible to use turoctocog alfa for all hemophilia patients. However, studies will be needed to confirm the turoctocog alfa profile, such as the stability of dissolved turoctocog alfa over 24 hours at room temperature and post-marketing clinical research aimed at meeting Europe Medicines Agency post-marketing safety and efficacy requirements in PTPs. It is recommended to wait before using turoctocog alfa for previously untreated patients and major surgery until further data have been collected and published.