In previously untreated patients (PUPs) with severe hemophilia A, the use of simoctocog alfa appeared to be efficacious and well tolerated when used as a prophylaxis agent and in treatment of bleeding episodes (BEs), according to a recent study. Study findings were reported in the European Journal of Haematology.
Simoctocog alfa is a recombinant factor VIII (FVIII) agent that, in previously treated patients with severe hemophilia A, has demonstrated efficacy in both preventing and treating BEs. In the phase 3 multicenter, open-label, noncontrolled NuProtect study (ClinicalTrials.gov Identifier: NCT01712438), simoctocog alfa was tested in prophylaxis and treatment of BEs in PUPs with severe hemophilia A.
In this study, patients were given simoctocog alfa for prophylaxis, surgical prophylaxis, and treatment of Bes. Switching was allowed between prophylaxis and on-demand use of the agent. Dosing of simoctocog alfa varied based on the clinical characteristics. Efficacy of prophylaxis was evaluated based on annualized bleeding rates (ABRs) in the absence of inhibitors.
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Efficacy in treatment of BEs was evaluated based on ratings by parents or legal guardians and, in some cases, by the investigator. For surgical prophylaxis, efficacy was rated by the surgeon and hematologist. Safety was assessed in all treated patients.
There were 108 patients treated with simoctocog alfa in this study, of whom 103 were given at least 1 prophylactic dose of this agent. Continuous prophylaxis was given to 50 patients, while 85 patients were treated for BEs, and 21 patients had surgical procedures. Inhibitor development occurred in 28 patients during the study, for whom efficacy data were evaluated during inhibitor-free periods.
The median ABRs for patients receiving continuous prophylaxis were 0.0 for spontaneous BEs and 2.5 for all BEs. Surgical prophylaxis with simoctocog alfa was rated as excellent or good with 94.7% of rated procedures and rated as moderate with 5.3%.
Efficacy in treating BEs with simoctocog alfa was rated as excellent or good in 92.9% of rated BEs. Among all BEs in this study, 65.1% had been considered minor, 34.0% as moderate-to-major, and 0.9% as major to life-threatening.
There were no thromboembolic events, severe allergic reactions, or deaths reported in this study. Serious adverse events deemed possibly or probably related to treatment included inhibitor development in 28 patients, pyrexia in 2 patients, and hypersensitivity in 1 patient.
“Efficacy and safety data from the NuProtect study demonstrate that simoctocog alfa was well tolerated and efficacious for the prevention and treatment of BEs in PUP,” the study investigators wrote in their report.
Disclosures: Some study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of disclosures.
Reference
Mathias M, Abraham A, Belletrutti MJ, et al. Simoctocog alfa (Nuwiq®) in previously untreated patients with severe haemophilia A—Final efficacy and safety results from the NuProtect study. Eur J Haematol. Published online July 13, 2023. doi:10.1111/ejh.14040
