Novo Nordisk's Long-Acting Hemophilia A Therapy Turoctocog Alfa Pegol Approved in China

Jul 05, 2024 16:11 CST Updated 16:11

Novo Nordisk

Insulin Developer and Manufacturer

▎Edited by the WuXi AppTec content team

Today (July 5), the official website of China's National Medical Products Administration (NMPA) announced the latest information,Novo NordiskInjectable Turoctocog Alfa Pegol (turoctocog alfa pegol, formerly known asPeturro Coagulase α）The marketing application has been approved.According to the priority review announcement by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China, the indication approved this time for the drug is:For on-demand treatment and control of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with Hemophilia A (congenital Factor VIII deficiency).Public information shows that this is a long-acting recombinant coagulation factor VIII (FVIII).

Screenshot source:NMPA Official Website

Hemophilia A is a rare hereditary hemorrhagic disease caused by the deficiency of coagulation factor VIII, with repeated bleeding and related complications as the main clinical manifestations. If repeated bleeding is not treated in time, it can lead to joint deformities or pseudo-tumor formation, and in severe cases, can be life-threatening. Currently, the main treatment for hemophilia A is regular infusion of coagulation factor VIII, but frequent infusions bring great inconvenience to patients' lives. Prolonging the half-life of coagulation factors through chemical modification can help reduce the frequency of infusions for hemophilia patients, alleviating their treatment burden.

Injectable Petrolatum Coagulase α (N8-GP) is aCoagulation factors with significantly prolonged half-life after polyethylene glycol (PEG) modification. It is based on the recombinant FVIII protein turoctocog alfa (trade name: Novoeight) developed by Novo Nordisk, with a PEG of approximately 40 kDa conjugated to the B-domain of turoctocog alfa, thereby extending the half-life.Compared with non-modified FVIII products, the half-life of injectable Pegylated Recombinant Factor VIII Alpha is extended by 1.6 times in adults/adolescents and 1.9 times in children.。

In February 2019, the injectable Petrolatum Coagulation Factor α was approved by the U.S. FDA for market release, intended for prophylactic and acute treatment in adult and pediatric patients with Hemophilia A. The approval was based on clinical trial results involving 270 patients with Hemophilia A. Data shows,Prophylactic injection with Pegloticase Alfa (50 IU/kg) once every 4 days can maintain the annual bleeding rate at a low level of 1.18 times per year.。ResearchersA survey was also conducted on hemophilia A patients using injectable Petrolatum Coagulase α.It was found that, compared with previous treatments, patients were more satisfied after using injectable PEGylated recombinant factor VIII, mainly becauseReduced infusion frequency, easy storage, excellent hemostatic efficacy, improved quality of life.

In July 2021, Novo Nordisk initiated a Phase 3, multi-center, open-label trial (pathfinder10) in China to evaluate the efficacy, safety, and pharmacokinetics of Pegnivacogin α for injection in treating previously treated patients with Hemophilia A. The primary endpoint of the trial is the number of bleeding events, and the principal investigator is from the Hematology Hospital of the Chinese Academy of Medical Sciences.Professor Renchi Yang。

According toClinicalTrialsAccording to the official website, the study has been completed.The research results show,Prophylactic injections of Pegnivacogin α (50 IU/kg) once every 4 days/twice a week can maintain the annual bleeding rate at a low level of 0.00 times per year.。When used to stop bleeding during bleeding episodes, an excellent hemostatic response was observed in 40 out of 52 bleeding episodes.Patients experienced significant pain relief and/or marked improvement in objective signs of bleeding within approximately 8 hours after a single injection of turoctocog alfa.；Good hemostatic response was observed in 9 instances. Approximately 8 hours after a single injection of turoctocog alfa, the patient experienced significant pain relief and/or improvement in objective signs of bleeding, although multiple injections may be required for complete resolution.Three mild hemostatic reactions were observed, with slight improvement in pain and bleeding approximately 8 hours after a single injection of NovoEight (turoctocog alfa), though multiple injections are typically required.

References:

[1][1] Drug Approval Certificate Delivery Information on July 5, 2024 Retrieved July 5, from https://www.nmpa.gov.cn/zwfw/sdxx/sdxxyp/yppjfb/20240705153402194.html

[2]Novo Nordisk receives US FDA approval of ESPEROCT® (turoctocog alfa pegol, N8-GP). Retrieved Feb. 20, 2019, from https://globenewswire.com/news-release/2019/02/19/1737267/0/en/Novo-Nordisk-receives-US-FDA-approval-of-ESPEROCT-turoctocog-alfa-pegol-N8-GP.html

[3]【2023 EAHAD】——The Evolution of Hemophilia Treatment. Retrieved Mar 21, 2023. From https://mp.weixin.qq.com/s/FLC-4qbNzSt8XC3mBPzitw

[4] ClinicalTrials Official Website. Retrieved Jan 18, 2024. From https://classic.clinicaltrials.gov/ct2/show/results/NCT05082116?view=results

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