Danish Novo Nordisk has received approval from the US Food and Drug Administration (FDA) for its drug Esperoct (turoctocog alfa pegol, N8-GP) indicated for haemophilia A treatment in both adults and children.
Esperoct is an extended half-life factor VIII molecule meant for replacement therapy.
It can be used as routine prophylaxis to minimise the frequency of bleeding episodes, on-demand therapy and bleeding episodes control and perioperative management of bleeding.
The drug is said to provide a 1.6-fold half-life prolongation in adults and adolescents and a 1.9-fold prolongation in children, when compared to conventional half-life factor VIII products.
Novo Nordisk executive vice-president and chief science officer Mads Krogsgaard Thomsen said: “We are excited about the approval of Esperoct in the US, and we consider it an important expansion of the treatment options Novo Nordisk can offer people with haemophilia A.
“We are confident that Esperoct will provide people with haemophilia A a less burdensome and simple, fixed dosing regimen for prophylaxis and treatment of bleeding episodes, resulting in improved quality of life.”
The FDA approval is based on data obtained during the pre-registration clinical programme that enrolled 270 previously treated people (PTPs) with severe haemophilia A and more than five years of clinical exposure.
Results showed that the fixed dosing regimen of one Esperoct injection for every four days in adults/adolescents or twice-weekly in children provided effective routine prophylaxis.
In addition, the drug demonstrated efficacy in treatment and control of bleeding episodes and perioperative management.
The drug was also well tolerated across clinical trials and age groups, with no safety concerns identified and its overall safety profile being similar to that of other long-action FVIII products.
Novo Nordisk noted that because of certain intellectual property agreements, Esperoct cannot be commercially launched in the US before 2020.