Novo Nordisk has received FDA approval for turoctocog alfa pegol (Esperoct), a new treatment for hemophilia A in children and adults.
Turoctocog alfa pegol (formerly N8-GP) is an extended half-life factor VIII molecule for replacement therapy in people with hemophilia A. It is approved for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes, and perioperative management of bleeding.
In hemophilia A, the failure of the blood to clot can cause frequent and spontaneous bleeding, which can lead to long-term joint damage and disability. For patients who do not have factor VIII inhibitors, standard treatment has required multiple infusions of factor VIII replacements each week. The new treatment offers patients a much less burdensome option.
Turoctocog alfa pegol’s approval was based on results of a clinical trial with 270 previously treated patients who had severe hemophilia A and more than five years of clinical exposure. Patients received the medication through a fixed regimen of one injection every four days for adolescents or adults, or every three to four days for children.
As well as providing “effective routine prophylaxis,” turoctocog alfa pegol enabled patients to maintain a low median annualized bleeding rate of 1.18 when dosing was maintained. The drug was also found to be efficacious in the treatment and control of bleeding episodes and perioperative management. It was well tolerated in all age groups and no safety concerns were identified.
Turoctocog alfa pegol is expected to be available in the U.S. in 2010.
Source: BioSpace, February 20, 2019