The U.S. Food and Drug Administration approved turoctocog alfa pegol, an extended half-life factor VIII (FVIII) molecule formerly known as N8-GP, for the treatment of children and adults with hemophilia A.
Turoctocog alfa pegol was approved for the following indications: routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes, and peri-operative management of bleeding.
The agency’s decision to approve the agent was based on the results from five prospective, non–placebo-controlled, multicenter clinical trials that enrolled a total of 270 patients with previously treated, severe hemophilia A and no history of inhibitors. Turoctocog alfa pegol was administered through a fixed-dosing regimen consisting of one injection every four days in adults and adolescents, or twice-weekly injections in children.
Treatment with turoctocog alfa pegol resulted in an annualized bleeding rate of 1.18 in adults and adolescents. The overall safety profile of turoctocog alfa pegol was similar to what has been reported for other long-acting FVIII products, with no safety concerns identified after more than five years of treatment exposure, the researchers reported.
Novo Nordisk, the biologic’s manufacturer, said the treatment will not be available in the U.S. until 2020 due to third-party intellectual property agreements.
Source: Novo Nordisk press release, February 19, 2019.