Novo Nordisk Launches Esperoct (Turoctocog Alfa Pegol) in the U.S. for Hemophilia A with Extended Half-Life and Reduced Dosing Frequency

February 12, 2020 News /Bio ValleyBIOON/ -- Novo Nordisk recently announced that Esperoct (turoctocog alfa pegol, N8-GP) has been launched in the United States for adult and pediatric patients with hemophilia A: (1) routine prophylactic treatment to prevent or reduce the frequency of bleeding episodes; (2) on-demand treatment and control of bleeding episodes; (3) perioperative bleeding management. In the United States, Esperoct was approved in February 2019FDAApproved, but due to third-partyIntellectual PropertyUnder the agreement, Novo Nordisk was unable to launch Esperoct in the U.S. market before 2020.

Hemophilia A is a chronic, hereditary bleeding disorder that affects nearly 20,000 people in the United States. Also known as factor VIII (FVIII) deficiency or classic hemophilia, it is an X-linked condition caused by the absence or defect of clotting factor VIII.HeredityIn this disease, patients experience recurrent persistent or spontaneous bleeding, particularly in the joints, muscles, or internal organs, which can lead to disability over time. It is estimated that approximately 60% of hemophilia A cases are severe. Currently, the standard of care for severe hemophilia A is a prophylactic regimen involving intravenous infusions of coagulation factor VIII two to three times per week.

Esperoct is an extended half-life coagulation factor VIII product. Compared with standard half-life factor VIII products, Esperoct demonstrates a 1.6-fold longer half-life in adult and adolescent patients and a 1.9-fold longer half-life in pediatric patients. Unlike standard half-life factor VIII products that require multiple intravenous infusions, Esperoct helps patients with hemophilia A maintain higher coagulation factor VIII levels for a longer duration, thereby reducing bleeding frequency and the number of administrations.

The approval of Esperoct is based on data from PATHFINDER, the largest and longest clinical program conducted in hemophilia A, comprising five multinational studies involving 270 patients. The program evaluated the efficacy and safety of Esperoct for on-demand treatment, prophylactic treatment, and surgical hemostasis in adult and pediatric patients. Results demonstrated that Esperoct provides effective routine prophylaxis in patients with severe hemophilia A (endogenous FVIII activity <1%) through a simple, fixed-dose regimen (every 4 days in adults and adolescents, and every 3–4 days [twice weekly] in children). In adults and adolescents (≥12 years of age), Esperoct administered at a dose of 50 IU/kg every 4 days provided effective prophylaxis, maintaining a low mean annualized bleeding rate (ABR) of 1.18 events. In pediatric patients under 12 years of age, the median ABR was 1.95 events with Esperoct administered twice weekly at a dose of 60 IU/kg (50–75 IU/kg). Furthermore, study results indicated 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 extended-half-life FVIII products.

Esperoct (N8-GP) is a glycoPEGylated form of the recombinant coagulation factor VIII product NovoEight (turoctocog alpha), 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 alpha. N8-GP is a B-domain-modified turoctocog alpha; therefore, the active factor VIII generated upon thrombin activation is identical to activated endogenous FVIII and turoctocog alpha.

NovoEight is the brand name for turoctocog alpha, 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 leverages the latest recombinant gene and protein purification technologies. (Bioon.com)

Original Source: Novo Nordisk launches ESPEROCT in the U.S. for the treatment of people with hemophilia A