Abstract Number: PB0549
Meeting: ISTH 2021 Congress
Background: Simoctocog alfa (Nuwiq®) is a 4th generation recombinant FVIII (rFVIII) produced in a human cell line. Simoctocog alfa has been evaluated in nine completed prospective clinical studies in adult and paediatric previously treated patients (PTPs) with severe haemophilia A.
Aims: To assess immunogenicity and safety of simoctocog alfa in PTPs enrolled in the GENA clinical trial programme.
Methods: All participants had previously received FVIII for at least 50 exposure days. Pooled immunogenicity and safety data were analysed. In all studies, inhibitory antibodies were measured by the Bethesda assay, with an inhibitor titre ≥0.6 Bethesda units (BU)/mL classified as a positive inhibitor test. All treatment-related adverse events recorded across all studies were identified.
Results: A total of 310 paediatric or adult PTPs were switched to simoctocog alfa and received treatment. Of the 310 individuals, 119 (38.4%) switched from plasma-derived FVIII (pdFVIII) products, 151 (48.7%) from rFVIII, 36 (11.6%) had received both pdFVIII and rFVIII, and 4 (1.3%) switched from an unknown FVIII. Patients received a total of 54,112 infusions during the studies. FVIII inhibitors did not develop in any patient after switching to simoctocog alfa. A total of 15 treatment-related adverse events were recorded in 9 (2.9%) patients. Only one (0.3%) patient had an AE (mild pyrexia that resolved) that was classified as serious because the patient was hospitalised.
Conclusions: None of the 310 PTPs who switched to simoctocog alfa developed an inhibitor and very few treatment-related adverse occurred during extensive exposure.
To cite this abstract in AMA style:Manco-Johnson MJ, Liesner RJ, Tiede A. Immunogenicity and Safety of Simoctocog Alfa in Previously Treated Patients Switching to Simoctocog Alfa in the GENA Clinical Trial Programme [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 1). https://abstracts.isth.org/abstract/immunogenicity-and-safety-of-simoctocog-alfa-in-previously-treated-patients-switching-to-simoctocog-alfa-in-the-gena-clinical-trial-programme/. Accessed September 23, 2021.
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