title: Efficacy and safety of a recombinant factor VIII produced from a human cell line (simoctocog alfa).
abstract: 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.
authors.affiliation: a Department of Transfusion Medicine and Hematology , Carlo Poma Hospital , Mantova , Italy.
authors.forename: Massimo
authors.initials: M
authors.lastname: Franchini
authors.affiliation: b Angelo Bianchi Bonomi Hemophilia and Thrombosis Center , Fondazione IRCCS Ca' Granda-Ospedale Maggiore Policlinico and University of Milan , Milan , Italy.
authors.forename: Pier Mannuccio
authors.initials: PM
authors.lastname: Mannucci
mesh_terms: D000818:Animals; D002460:Cell Line; D017322:Clinical Trials, Phase II as Topic; D017326:Clinical Trials, Phase III as Topic; D003029:Coagulants / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D005169:Factor VIII / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use; D006467:Hemophilia A / Q000188:drug therapy*; D006801:Humans; D011788:Quality of Life; D016032:Randomized Controlled Trials as Topic; D011994:Recombinant Proteins
chemical_list: D003029:Coagulants; D011994:Recombinant Proteins; D005169:Factor VIII
keywords: Hemophilia A; human recombinant FVIII; inhibitors; safety; therapy