LAUSR

ABSTRACT Introduction: Hemophilia A is the most frequent inherited bleeding disorder and most challenging coagulation disorder. To combat this, a number of new improved rFVIII/IX concentrates have recently been approved. Some of them are derived from protein fusion biotechnology or pegylation to extend their half-life (HL). However, prophylaxis has become a standard of care to prevent arthropathy in hemophiliacs though the need of frequent venipunctures is a major obstacle to primary prophylaxis. The new Extended Half-Life (EHL) rFIX concentrates allow increased intervals, while the improved HL of new rFVIII was moderate. rFVIII Simoctocog alfa is produced in Human Embryonic Kidney (HEK) cells and the post-translational modifications performed by HEK cells are very similar to those occurring in the native FVIII. Areas covered: Herein, the author provides a review of simoctocog alfa with its contents including information on simoctocog alfa’s manufacturing, clinical trials, safety and tolerability. They also give their expert opinion and future perspectives on this therapy. Expert opinion: An important advantage of simoctocog alfa is the possibility to omit at least 30% of venipunctures with prophylaxis. Consequently, the standard three times weekly bolus administrations may be reduced to twice weekly, meaning approximately 50 fewer venipunctures per year. This may be particularly helpful to children.