FDA OKs Turoctocog Alfa Pegol (Esperoct ) for Hemophilia A

Megan Brooks

February 21, 2019

The US Food and Drug Administration (FDA) has approved turoctocog alfa pegol (Esperoct, Novo Nordisk) for patients with hemophilia A, a hereditary disorder caused by a lack of the blood clotting factor VIII protein.

Turoctocog alfa pegol (also known as N8-GP) is an extended half-life glycopegylated factor VIII molecule for adults and children with hemophilia A. It is indicated for control and prevention of bleeding, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

Turoctocog alfa pegol has been evaluated in 270 people (202 adults/adolescents and 68 children) in five prospective, multicenter clinical trials involving previously treated people with severe hemophilia A (<1% endogenous FVIII activity) and no history of inhibitors. Total exposure to the drug was 80,425 exposure days, corresponding to 889 patient-years of treatment.

In clinical testing, a fixed dosing regimen consisting of one injection of turoctocog alfa pegol every 4 days in adults and adolescents or every 3 to 4 days (twice weekly) in children provided effective prophylaxis against bleeding. 

Turoctocog alfa pegol maintained a low median annual bleeding rate of 1.18 when dosed at 50 IU/kg every 4 days in adults and adolescents, the company said in a news release.

Turoctocog alfa pegol was well tolerated across all age groups and indications, and no safety concerns were identified after more than 5 years of clinical exposure, the company said.

"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," said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk.

"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," said Thomsen.

Due to third-party intellectual property agreements, Novo Nordisk said it will not be able to launch turoctocog alfa pegol before 2020 in the US.

In the US, hemophilia occurs in about 1 of every 5000 male births, according to the Centers for Disease Control and Prevention, and about 20,000 US boys and men are living with the disorder. Hemophilia A is about four times as common as hemophilia B, and about half of those affected have the severe form.

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