rFVIIa, a human recombinant activated
coagulation factor VII, has been used worldwide for more than two decades for the treatment of
bleeding episodes and prevention of
bleeding in patients undergoing surgery/invasive procedures with congenital
haemophilia A or B with inhibitors (CHwI A or B), acquired
haemophilia (AH), congenital
factor VII deficiency and
Glanzmann thrombasthenia (GT), refractory to
platelet transfusion. The approved dosage, administration and indication of
rFVIIa in the US, Europe and Japan differ, depending on the needs of the patient population and regulatory practices. This review presents an overview of the current status and future prospects, including that from a Japanese perspective, of using
rFVIIa in the treatment of approved indications. The efficacy and safety of
rFVIIa in the approved indications has been demonstrated in several randomised and observational studies and data from registries. The overall incidence of
thrombosis across all approved indications in a retrospective safety assessment of clinical trials and registries, prelicensure studies and postmarketing surveillance studies of
rFVIIa use was 0.17%. Specifically, the risk of thrombotic events was 0.11% for CHwI, 1.77% for AH, 0.82% for congenital
factor VII deficiency and 0.19% for GT. Emerging non-factor
therapies such as
emicizumab have changed the treatment landscape of
haemophilia A, including preventing
bleeding in patients with CHwI. However,
rFVIIa will continue to play a significant role in the treatment of such patients, particularly during
breakthrough bleeding or
surgical procedures.