New Treatment for Hemophilia A Receives FDA Approval
The FDA has approved the Biologics License Application for Novo Nordisk’s long-acting factor VIII replacement treatment for adults and children with hemophilia A.
The FDA has approved the Biologics License Application for Novo Nordisk’s long-acting factor VIII replacement treatment for adults and children with hemophilia A, according to a press release.
Turoctocog alfa pegol, also known as N8-GP (Esperoct), is an extended half-life factor VIII molecule indicated for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment, and control of bleeding episodes and perioperative management of bleeding.
According to the CDC, hemophilia occurs in approximately 1 in 5000 live births and approximately 20,000 individuals in the United States are affected. Hemophilia A is more common than hemophilia B and more than half of individuals with hemophilia A have the severe form.
The approval is based on data from the largest pre-registration clinical program conducted in hemophilia A evaluating 270 previously-treated patients with severe hemophilia A and more than 5 years of clinical exposure. Overall, N8-GP demonstrated effective routine prophylaxis in patients through a simple, fixed dosing regimen of 1 injection every 4 days in adults and adolescents or every 3 to 4 days in children. N8-GP maintained a low median ABR of 1.18 when dosed at 50 IU/kg every 4 days in adults and adolescents.
N8-GP has been evaluated across 5 prospective, multi-center clinical trials in previously-treated patients with severe hemophilia A and no history of inhibitors. The overall safety profile of N8-GP is similar to what has been reported for other long-action FVIII products. No safety concerns were identified after more than 5 years of clinical exposure.
“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 hemophilia A,” Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk, said in a statement. “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.”
Due to third-party IP agreements, Novo Nordisk will not be able to launch the treatment before 2020 in the United States, according to the release.
A version of this article was published by Specialty Pharmacy Times. Visit SpecialtyPharmacyTimes.com to view the article and a video about advances in current and future therapies for hemophilia.
Novo Nordisk recieves US FDA approval of ESPEROCT (turoctocog alfa pegol, N8-GP) [news release]. Novo Nordisk. http://www.globenewswire.com/news-release/2019/02/19/1737267/0/en/Novo-Nordisk-receives-US-FDA-approval-of-ESPEROCT-turoctocog-alfa-pegol-N8-GP.html. Accessed February 19, 2019.