On July 23, according to latest announcement in the official website of the National Medical Products Administration (NMPA), the recombinant coagulation factor VIII (SCT800) for injection developed by Shenzhou Cell Biotechnology (hereinafter referred to as Shenzhou Cell) has been officially approved. According to public information, this is the first Chinese-made recombinant coagulation factor VIII product. The indications of this approved product are as follows. It is suitable for controlling and preventing bleeding of teenagers and adults with hemophilia A (congenital coagulation factor VIII deficiency).
Hemophilia is a hereditary lifelong disease and its characteristic is blood coagulation cascade is damaged. Most patients are born with the lack of coagulation factors, so the normal touch will make them bleed. The critical patients will also have "spontaneous" bleeding even there is no obvious trauma. Hemophilia is divided into three types: A, B and C with the lack of coagulation factors VIII, IX and IX respectively.
The incidence of patients with hemophilia A is 1/10000, that of hemophilia B is 1/35000, and that of hemophilia C is rare. Almost all patients with hemophilia are male. Generally, women usually do not develop, but they can carry pathogenic genes, which leads to a 50% incidence rate for male offspring. Hemophilia A and B may be atavism, and the most common hemophilia A is caused by coagulation factor VIII deficiency or functional defect.
Blood coagulation cascade signaling pathway (source: References 1)
Although hemophilia is a rare disease, it does not represent that it is an incurable disease. It can reduce bleeding or stop bleeding by exogenously supplementing human-derived or re-giving coagulation factors.
At present, the manufacturers of coagulation factor VIII on the market in China are mainly Shanghai Raas, Hualan Biological Engineering, Inc., Green Cross, Taibang Biologic, Shanghai Xinxing Medicine Co., Ltd., etc., and all their products are the first generation of blood-derived coagulation factor VIII. While the imported products including Baxter's ADVATE, Bayer's Kogenate FS, Pfizer's Xyntha, etc. are mainly the recombinant coagulation factor VIII (second generation). In December 2020, the third generation of recombinant coagulation factor VIII for injection of Novo Nordisk was approved in China. If you are looking for bio pharmaceutical products, then Pharmasources would be your best choice.
The approved SCT800 is the third generation of recombinant coagulation factor VIII product developed by Shenzhou Cell, without albumin in its process and preparation. It is also the first recombinant coagulation factor VIII product in China. New drug application of this product was submitted in November 2019, and was then included in the priority review. According to the information on the official website of Shenzhou Cell, the Phase III on-demand treatment study of SCT800 for patients with hemophilia A and over 12 years old has completed, and Phase III preventive treatment study for patients with hemophilia A and over 12 years old is undergoing. At the same time, the Phase III preventive treatment study for patients with hemophilia A and under 12 years old has also been initiated.
In China, most coagulation factor VIII is extracted from human plasma. However, the productivity of extracting factor VIII from plasma is very limited because of the shortage of human plasma. At the same time, due to the poor thermal stability and low yield of coagulation factor VIII protein, as well as the high technical threshold and difficulty in the production of recombinant coagulation factor VIII protein, there ...
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