Recombinant Human Coagulation Factor VIII for Injection (NL202)

　　 ● Project Overview

　　Registration Category: Biological Products for Therapeutic Use, Category 3

　　Indications: Hemophilia A

　　R&D Progress: Preclinical Studies

　　Market Prospects: China has approximately 100,000 hemophilia patients who require 1.3 billion units of coagulation factor VIII (FVIII) annually. However, the annual supply of FVIII derived from blood sources in China is only 60 to 80 million units, far short of meeting clinical demand. This product can help alleviate the current severe shortage of FVIII and offers both significant economic and social benefits.

　　 ● Mechanism of action

　　FⅧ is a key amplification factor in the intrinsic coagulation cascade. After being activated by thrombin, FⅧ is converted into FⅧa, which serves as a cofactor for FⅨa and, in synergy with FⅨa, accelerates the conversion of FⅩ into FⅩa. FⅩa, in turn, activates prothrombin, leading to the production of more thrombin. Thrombin then converts fibrinogen into fibrin. Fibrin forms an insoluble clot, thereby exerting a hemostatic effect.

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　　 ●Research findings

　　This project uses CHO cells as the expression system to produce BDD-rFVIII. By employing immunoaffinity chromatography and a virus removal process, we can obtain a highly pure target protein while significantly reducing the risk of viral contamination, thereby effectively ensuring product safety.

　　This project has completed preclinical pharmacology and pharmacotoxicology studies. The results indicate that the pharmacokinetic and pharmacodynamic profiles of this product are comparable to those of currently marketed products, and it exhibits a wide safety margin.

　　Neither the production process nor the formulation recipe of this product uses any ingredients derived from humans or animals. Through stepwise scale-up, we have established a 200L-scale manufacturing process and developed stringent preparation procedures and quality standards, ensuring that we can meet the production and supply requirements for clinical trial samples.