A new hemophilia drug! Novo Nordisk long-acting coagulation factor VIII product esperoct (n8-gp) has been approved by CHMP of European Union

April 28, 2019 / BIOON / - Novo Nordisk, a Danish pharmaceutical giant, recently announced that the human pharmaceutical products Committee (CHMP) of the European Drug Administration (EMA) has issued active review opinions and recommended approval of esperoct (turoctocog Alfa Pegol, n8-gp) is used for adolescents and adults with type a hemophilia Routine preventive treatment can reduce the frequency of bleeding attack, control the bleeding attack according to the need, and manage the bleeding during the perioperative period Now, CHMP's comments will be submitted to the European Commission (EC), which will refer to CHMP's comments and make a final review decision in the next 2-3 months In the United States, esperoct was approved by FDA in February 2019 for adult and child patients with hemophilia A type A Due to the third party IP agreement, Novo Nordisk will not be able to launch esperoct in the United States by 2020 Esperoct is an extended half-life coagulation factor VIII product for the treatment of hemophilia A A patients Compared with standard half-life factor VIII products, esperoct has a 1.6-fold longer half-life in adult / adolescent patients and a 1.9-fold longer half-life in children CHMP supports the approval of esperoct, which is based on the data of clinical project Pathfinder, the largest pre registration clinical project in hemophilia A, including 5 prospective multicenter clinical studies 270 patients (202 adults / adolescents, 68 children) with severe hemophilia (endogenous FVII activity < 1%) who have been previously treated (treated, PTP) have been enrolled and have no inhibition The total exposure time of esperoct in this project is 80425 days, which is equivalent to 889 patient years Esperoct was well tolerated in all age groups and indications, and no safety problems were found after more than 5 years of clinical exposure The project evaluated the efficacy and safety of esperoct in adult patients, children patients on-demand treatment, preventive treatment, surgical hemostasis The results show that esperoct can provide effective routine prevention in patients with severe hemophilia through a simple and fixed drug delivery program (injection every 4 days for adults and adolescents, once every 3-4 days [twice a week] for children) In adults and adolescents, esperoct provided effective prophylaxis at a dose of 50 IU / kg every 4 days and maintained a median annual bleeding rate (ABR) as low as 1.18 events In addition, esperoct was shown to be equally effective in the treatment and control of bleeding events and perioperative management Across all studies and age groups, esperoct was well tolerated and no safety issues were identified The overall safety of esperoct is similar to other long-term FVII products reported MADS krogsgaard Thomsen, executive vice president and chief scientific officer of Novo Nordisk, said, "we are very pleased to receive positive comments from the European Union CHMP on esperoct, which will provide an important expansion of the treatment program for patients with hemophilia A a We believe that esperoct will provide patients with a simple and fixed dose hemophilia treatment program to reduce the burden, prevent and treat bleeding events, so as to improve the quality of life " Esperoct (n8-gp) is a glycosylated form of recombinant coagulation factor VIII product novoeight (turocog alpha), which is developed for the treatment of hemophilia A A patients Glycosyl PEGylation can prolong the circulating half-life of recombinant coagulation factor VIII, thus reducing the number of intravenous administration and the frequency of bleeding attack The glycosylated PEGylation site of n8-gp is located in the B domain truncated by turoctocog alfa N8-gp is a B-domain modified turoctocog alfa, so the active factor VIII produced by thrombin activation is the same as the activated endogenous FVII and turoctocog alfa Novoeight is the trade name of the third generation recombinant coagulation factor VIII product turoctocog alpha of Nord Nordisk, which was approved by FDA and EU in 2013 It is used for the preventive treatment and on-demand treatment of hemophilia A A patients The product uses the latest gene recombination and protein purification technology Source of the original text: Novo Nordisk receives positive opinion from the European regulatory authorities for esperoct ® (turotocog Alfa peg, n8-gp) for the treatment of haemophilia A