Health Canada and Australia’s Therapeutic Goods Administration (TGA) have both approved a recombinant FVIII product known as simoctocog alfa (Nuwiq).
Health Canada has approved the product to treat and prevent bleeding in hemophilia A patients of all ages.
And the TGA has approved simoctocog alfa for the treatment and prevention of bleeding in previously treated pediatric (≥ 2 years) and adult patients with
hemophilia A.
Simoctocog alfa is a recombinant FVIII product produced in a human cell line cultured without additives of human or animal origin or any exposure to human blood or plasma, making it inherently free from blood-borne pathogens.
Simoctocog alfa is also devoid of antigenic non-human protein epitopes, similar to FVIII produced in healthy humans. It has a high affinity for the von Willebrand coagulation factor.
“The way Nuwiq is produced is exciting, as it allows the molecule to closely resemble the naturally occurring FVIII,” said Anthony Chan, MBBS, Director of the Hemophilia Program at McMaster Children’s Hospital in Hamilton, Ontario.
“Health Canada’s approval of Nuwiq provides patients with hemophilia A a new recombinant product option that will allow further customization of hemophilia treatment on an individual basis.”
Researchers have evaluated the immunogenicity of simoctocog alfa in 135 previously treated patients with hemophilia A (74 adults and 61 children). And none of the patients developed inhibitors.
In the ongoing, phase 3 NuProtect study, researchers are investigating 100 previously untreated patients, a group typically characterized by a higher risk of inhibitor development. The researchers will assess whether the molecular properties of simoctocog alfa will result in lower inhibitor development.
Two additional phase 3 studies in previously treated patients are ongoing. The NuPreviq study and the Canadian Gena-21b study were designed to assess the efficacy and safety of individually tailored prophylaxis.
The goal of these studies is to provide optimal treatment for each patient based on his or her own pharmacokinetic properties, with a potential reduction in the frequency of FVIII infusions.
Simoctocog alfa was approved in the European Union earlier this year and is under review by regulatory authorities in the US. For more details on simoctocog alfa, see the prescribing information.
