Efficacy and safety of a recombinant factor VIII produced from a human cell line (simoctocog alfa)

ABSTRACT

Introduction: The development of inhibitors against infused factor VIII (FVIII) has a detrimental impact on health and quality of life of patients with hemophilia A. Several observational studies and a recently published randomized trial indicate that the inhibitor risk in previously untreated patients (PUPs) is higher following the use of recombinant FVIII (rFVIII) products compared with plasma-derived FVIII concentrates. There is currently a great interest towards newer rFVIII products that adopt various technological solutions to reduce the inhibitor risk.

Areas covered: This review describes the efficacy and safety of simoctocog alfa (Nuwiq), a human cell-line derived rFVIII developed for the prevention and treatment of bleeding in hemophilia A patients. Particular relevance will be given to the safety aspects of this product, with special emphasis on the rate of inhibitor development, presenting the results of the phase II and III clinical trials on previously treated patients (PTPs) and the interim data from the phase III trial on PUPs.

Expert opinion: The final results from PTP studies and the preliminary data from the PUP study document the high efficacy and safety profile of this rFVIII product, which has the potential to reduce the inhibitor risk in PUPs with severe hemophilia A.

KEYWORDS:

• Hemophilia A
• therapy
• human recombinant FVIII
• safety
• inhibitors

Declaration of interest

PM Mannucci has received lecture fees from Alexion, Baxalta/Shire, Bayer, CSL Behring, Grifols, Kedrion, LFB and Novo Nordisk. He also discloses advisory board membership with Bayer and Kedrion. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Additional information

Funding

This paper was not funded.