Novo Nordisk's Long-Acting Therapy for Hemophilia A Nominated for Priority Review in China

On September 1, the CDE website showed that Novo Nordisk's injectable Turoctocog Alfa Pegol was proposed for priority review. It is suitable for adult and pediatric patients with Hemophilia A (congenital Factor VIII deficiency): on-demand treatment and control of bleeding episodes; perioperative management; routine prophylaxis to prevent or reduce the frequency of bleeding episodes. On July 25, the marketing application of this product had been accepted by the NMPA.

Turoctocog alfa pegol (brand name: Esperoct) is another long-acting recombinant coagulation factor VIII (FVIII) developed by Novo Nordisk based on Turoctocog alfa (recombinant human coagulation factor VIII for injection, brand name: Novoeight). It was first approved for marketing in the United States in February 2019 and is used for prophylactic treatment and acute treatment in adult and pediatric patients with hemophilia A.

Esperoct modifies a PEG of approximately 40kDa on the B-domain of Turoctocog alfa based on Novoeight, increasing its half-life by nearly two times. Typically, hemophilia A patients require intravenous infusion of coagulation factor three times a week. As a prophylactic treatment administered once every four days, Esperoct can reduce the annual bleeding rate of patients by 96%.

Hemophilia A is a hereditary disease caused by the deficiency of FⅧ, leading to coagulation dysfunction. It is also the most common type of hemophilia, with a higher incidence in males. Clinically, it manifests as bleeding in any part of the body, including joints, muscles, and deep tissues. The severity of bleeding in Hemophilia A patients mainly depends on the FⅧ activity level. Severe Hemophilia A patients (residual FⅧ activity <1%) account for 50% of the entire patient population.

Novo Nordisk has multiple drug layouts in the hemophilia field. In addition to Novoeight and Esperoct mentioned above, its developed human IgG4 monoclonal antibody targeting tissue factor pathway inhibitor (TFPI), Concizumab, has been submitted for marketing authorization to prevent treatment in patients with inhibitors of Hemophilia A and B. However, in April this year, Novo Nordisk received a Complete Response Letter (CRL) from the FDA.

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