Clinical efficacy and safety of moroctocog alfa

Emanuela Marchesini, Domenico Prisco, Alfonso Iorio

The mainstay of treatment for patients with hemophilia A is replacement with either plasma-derived or recombinant (r) coagulation factor (F)VIII. As compared with whole plasma, plasma-derived FVIII concentrates clearly improved the clinical outcome for patients with hemophilia A; however, their use was associated with the transmission of blood-borne viruses such as hepatitis B and C virus and HIV, and prompted the introduction into clinical use of biotechnologically engineered recombinant clotting factor concentrates, with a much lower theoretical risk of transfusion-associated infections. Continued efforts to further improve safety have led to the introduction of newer generations of rFVIII, with each subsequent generation representing an incremental step with regard to pathogen safety. Between 1980 and 1985, research demonstrated that removing the middle portion (the B-domain) of wild-type FVIII protein had no detrimental effect on its procoagulant activity as assessed in  vitro. This finding was the basis for the development of a recombinant B‑domain-deleted FVIII (BDDrFVIII), moroctocog alfa (ReFacto®). Moroctocog alfa albumin-free cell culture (ReFacto AF® in Europe, Xyntha® in the USA) has been developed as a successor to ReFacto. The aim of this article is to describe the clinical evidence about moroctocog alfa and moroctocog alfa albumin-free cell culture.