Health Canada has approved Esperoct, an engineered formulation of clotting factor VIII, for the treatment of hemophilia A in people of all ages. The approval covers prophylactic, or preventive treatment, on-demand use for controlling bleeding episodes, and use in reducing bleeding during surgery.
Esperoct received similar approval for hemophilia A treatment from the U.S. Food and Drug Administration in early 2019, and recently in the EU. The therapy, an antihemophilic factor VIII (recombinant, B-domain truncated), PEGylated, was developed by the Danish company Novo Nordisk.
People with hemophilia A make abnormally low amounts of a blood clotting factor called factor VIII (FVIII). Esperoct is a recombinant, or lab-made version of FVIII that has been modified to have an extended half life — meaning that it sticks around in the body for a longer period of time — which is crucial for its use as a prophylactic agent.
The Health Canada decision was based on the results of a number of Phase 3 clinical trials (NCT01731600, NCT01480180, NCT01489111, NCT03528551, NCT02137850) that included 270 people — both children and adults — with severe hemophilia A. All of the participants had been previously treated with other FVIII products.
In both adults and adolescents (those over age 12), Esperoct maintained a low median average bleeding rate of 1.18 when given at a dose of 50 IU/kg every 3-4 days. In children under 12, a low median ABR also was maintained, at 1.95, with a dosage of 60 IU/kg (50-75 IU/kg) given twice a week.
“Esperoct has been shown to be effective in reducing the risk of bleeding episodes among patients,” Anthony Chan, MD, a professor of pediatrics at McMaster University in Ontario, said in a press release from Novo Nordisk.
Across the trials and age groups, no safety concerns were identified for Esperoct, and the treatment was well-tolerated. Esperoct’s safety profile reportedly is similar to that of other, like FVIII products.
The approval was welcomed by patient advocacy groups.
“The Canadian Hemophilia Society advocates for access to the widest possible range of coagulation therapies and welcomes the regulatory approval of this additional option for the treatment of hemophilia A,” said Paul Wilton, president of the society’s board of directors.