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Novo Nordisk's Extended Half-Life Factor VIII Product Esperoct Receives EU Approval for Adolescents and Adults with Hemophilia A

Jun 21, 2019 12:19 CST Updated 12:19
Novo Nordisk

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European Commission

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June 21, 2019 /Bio ValleyBIOON/ -- Danish pharmaceutical giant Novo Nordisk recently announced that the European Commission (EC) has approved Esperoct (turoctocog alfa pegol, N8-GP) for routine prophylactic treatment to reduce the frequency of bleeding episodes, on-demand treatment to control bleeding episodes, and perioperative bleeding management in adolescents (aged ≥12 years) and adults with hemophilia A. Novo Nordisk plans to launch Esperoct in the first batch of European countries in the second half of 2019.

In the United States, Esperoct was approved in February 2019FDAApproved for use in adult and pediatric patients with hemophilia A. Due to third-partyIntellectual Property RightsUnder the agreement, Novo Nordisk will not be able to launch Esperoct in the United States before 2020.

Esperoct is an extended half-life coagulation factor VIII product for the treatment of patients with hemophilia A. Compared with standard half-life factor VIII products, Esperoct has a 1.6-fold longer half-life in adult/adolescent patients and a 1.9-fold longer half-life in pediatric patients.

This approval is based on data from the PATHFINDER clinical program, the largest pre-registration clinical program conducted in hemophilia A. It comprised five prospective, multicenter clinical studies, including 270 patients (202 adults/adolescents and 68 children) with severe hemophilia A (endogenous FVIII activity <1%), who were previously treated (PTP), had no inhibitors, and had more than five years of clinical exposure. The total exposure to Esperoct in this program was 80,425 days, equivalent to 889 patient-years of treatment. Esperoct was well tolerated across all age groups and indications, with no safety issues identified during more than five years of clinical exposure.

This project evaluated the efficacy and safety of Esperoct for on-demand treatment, prophylactic treatment, and surgical hemostasis in adult and pediatric patients. The study results showed that Esperoct provides effective routine prophylaxis in patients with severe hemophilia A through a simple, fixed dosing regimen (injections every 4 days for adults and adolescents, and every 3–4 days [twice weekly] for children). In adults and adolescents, Esperoct administered at a dose of 50 IU/kg every 4 days provided effective prophylaxis and maintained a median annualized bleeding rate (ABR) as low as 1.18 events. Furthermore, the results demonstrated that Esperoct was equally effective in treating and controlling bleeding episodes and in perioperative management. Across all studies and age groups, Esperoct was well tolerated, with no safety concerns identified. The overall safety profile of Esperoct was similar to that reported for other long-acting FVIII products.

Mads Krogsgaard Thomsen, Executive Vice President and Chief Scientific Officer at Novo Nordisk, stated, “We are pleased to receive EU approval for Esperoct, which will provide an important expansion to the treatment options for patients with hemophilia A. We believe that Esperoct will offer patients a less burdensome, simple, fixed-dose hemophilia treatment regimen for the prevention and treatment of bleeding episodes, thereby improving quality of life.”

Esperoct (N8-GP) is a glycoPEGylated form of the recombinant coagulation factor VIII product NovoEight (turoctocog alfa), developed for the treatment of patients with hemophilia A. The glycoPEGylation technology extends the circulating half-life of recombinant coagulation factor VIII, thereby reducing the frequency of intravenous administrations and decreasing the frequency of bleeding episodes. The glycoPEGylation site of N8-GP is located within the truncated B domain of turoctocog alfa. As N8-GP is a B-domain-modified turoctocog alfa, the active factor VIII generated upon thrombin activation is identical to activated endogenous FVIII and turoctocog alfa.

NovoEight is the brand name for turoctocog alfa, Novo Nordisk’s third-generation recombinant coagulation factor VIII product, which was approved in 2013FDAApproved by the EU for prophylactic and on-demand treatment of patients with hemophilia A, this product utilizes the latest recombinant gene and protein purification technologies. (Bioon.com)