The European Commission has published approval of Octapharma’s Nuwiq ® (simoctocog alfa) for the treatment and prophylaxis of bleeding in all age groups with hemophilia A (congenital factor VIII [FVIII] deficiency). Nuwiq ® is the newest generation of FVIII replacement, produced in a human cell line cultured without additives of human or animal origin. Nuwiq ® is devoid of antigenic non-human protein epitopes and has a high affinity for the von Willebrand coagulation factor, both of which are potentially important properties in reducing FVIII inhibitor formation. The development of Nuwiq ® aimed to address the challenges of inhibitor formation as well as the frequent infusions required for prophylaxis of bleeding.
“Nuwiq ® represents a potential leap forward in dealing with the greatest clinical challenges in hemophilia A – FVIII inhibitors and the frequent infusions needed for bleeding prophylaxis. In clinical trials, Nuwiq ® demonstrated outstanding efficacy in preventing and treating bleeding in patients previously on FVIII (PTPs). No inhibitors in clinical trials in PTPs have been seen”, says Olaf Walter, MD, PhD, MBA, Senior Vice President of International Business Units.
The benefit of Nuwiq ® in terms of prevention and treatment of bleedings and hemostatic efficacy for surgical procedures was studied in three pivotal trials. In a study of 32 adults (GENA-08), overall prophylactic efficacy of Nuwiq ® for spontaneous bleeds was rated as excellent in all (100%) patients. In a study of 59 children (GENA-03), prophylactic efficacy for spontaneous bleeds was rated as excellent or good in 96.6% of patients. Mean annualized bleeding rates for spontaneous bleeds during prophylaxis were approximately 1.5 in children and 1.2 in adults. Treatment of breakthrough bleeds during prophylaxis was rated as excellent or good for 28 of 28 (100%) bleeds in adults and for 89 of 108 (82.4%) bleeds in children. In on-demand treatment with Nuwiq ® in 20 adults and 2 adolescents (GENA-01), efficacy for the treatment of bleeds was excellent or good in 931 of 986 (94.4%) bleeds. Overall efficacy across the GENA program in surgical prophylaxis was rated excellent or good in 32 of 33 (97%) procedures.
The immunogenicity of Nuwiq ® was evaluated in 135 PTPs (74 adults and 61 children). None of the patients developed inhibitors.
The next steps in Nuwiq ® ’s clinical program include continued investigation of previously untreated patients (PUPs), with a target enrolment of 100 patients in the ongoing NuProtect study. PUPs are typically characterized by a higher risk of inhibitors. Octapharma is also committed to the ongoing NuPreviq study in PTPs, which is designed to test a robust program of personalized prophylaxis, with a potential reduction in the number of FVIII infusions.
Regulatory data for Nuwiq ® have been submitted to authorities in Canada, the US, and Australia, with further worldwide submissions planned.