FDA Approves Esperoct as Prophylactic Treatment for Hemophilia A, Novo Nordisk Says
Children and adults with hemophilia A in the United States may soon be treated with N8-GP (turoctocog alfa pegol), an engineered formulation of clotting factor VIII developed by Novo Nordisk. The therapy will be available under the brand name Esperoct.
The U.S. Food and Drug Administration (FDA) approved N8-GP, or Esperoct, as a prophylactic (preventive) therapy to reduce frequency of bleeds and for on-demand use to control bleeding episodes in patients with inherited factor VIII deficiency, the company announced in a press release.
Esperoct, however, will not arrive on the U.S. market before 2020 due to third-party intellectual propriety agreements.
“We are excited about the approval of Esperoct in the U.S., and we consider it an important expansion of the treatment options Novo Nordisk can offer people with hemophilia A,” said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk, a Danish company.
“We are confident that Esperoct will provide people with hemophilia A a less burdensome and simple, fixed dosing regimen for prophylaxis and treatment of bleeding episodes, resulting in improved quality of life,” he said.
Esperoct is an engineered factor VIII molecule designed to have enhanced stability and provide better blood clotting ability for hemophilia A patients.
The FDA’s decision to approve Esperoct was based on clinical data collected from 270 patients, children and adults, with severe hemophilia A across who took part in a series of Phase 3 trials (NCT01731600, NCT01480180, NCT01489111, NCT03528551, and NCT02137850).
With data that covered at least five years of clinical exposure to Esperoct, researchers confirmed that the therapy had a 1.6-fold better half-life in adults and adolescents, and a 1.9-times longer half-life in children, compared to standard factor VIII products.
Treatment, given as a single injection of a fixed Esperoct dose of every four days in adults and teenagers, and every 3–4 days in children, was shown to be an effective routine prophylactic to prevent bleeds.
Adult and adolescent patients were able sustain a median annualized bleeding rate of 1.18 by using Esperoct 50 IU/kg every four days. In infants and children up to 12 years old, treatment was found to significantly improve their quality of life and satisfaction.
Esperoct was also found to be an effective in controlling bleeding episodes and in managing bleedings prior to surgery.
In all clinical trials and across all age groups, Esperoct was well-tolerated, with no safety problems being reported. In general, its safety profile was described as similar to that of other long-acting factor VIII replacement therapies.