The recombinant human coagulation factor VIII-Fc fusion protein for injection (FRSW117), a new generation ultra-long-acting recombinant coagulation factor VIII product developed by Gensciences, has obtained IND approval from drug regulatory authorities in China and the United States. FRSW117 has entered Phase III clinical trial stage in China, the “open-label, multi-center Phase III clinical trial to evaluate the efficacy, safety, immunogenicity and pharmacokinetics of the recombinant human coagulation factor VIII-Fc fusion protein for injection (FRSW117) in patients (adults and adolescents) with treated severe hemophilia A” is being carried out in more than 20 hospitals in China. The kick-off meeting for the first study site was held on December 21, 2023 at the leading unit, the Hematology Hospital of the Chinese Academy of Medical Sciences, which marked the official launch of the clinical trial.

Previously on December 11, the Center for Drug Evaluation (CDE) of the National Medical Products Administration approved the change of the production site of FRSW117 drug substance and drug product to Gensciences (Shanghai) facility. Located in Zhangjiang Science City, the Gensciences (Shanghai) facility is one of the major technological upgrading projects supported by the Shanghai Municipal Government. This GMP-compliant manufacturing facility has a complete quality system and qualified manufacturing capacity for Phase III clinical samples and commercial products. On December 19, the Phase III clinical samples were shipped from Gensciences (Shanghai) facility to clinical study sites, marking the milestone that Gensciences has gained the ability to independently produce innovative drugs.

FRSW117 is an ultra-long-acting recombinant human coagulation factor VIII-Fc fusion protein for injection developed on the basis of the proprietary dual-technology platform of homodimeric Fc fusion protein and PEG modification of Gensciences. It breaks free from the dependence of traditional coagulation factor VIII on the von Willebrand factor (vWF), breaks through the vWF ceiling of the half-life of coagulation factor VIII products, and is expected to reach the gold standard of preventive treatment with “once-weekly dosing regimen” by dramatically increasing the activity of factor VIII at trough concentration. It will provide better bleeding protection and reduce the annual bleeding rate of patients, significantly improve treatment compliance and lower economic burden, thus bringing long-term benefits to hemophilia A patients with a good safety profile. Previous Phase I and Phase II clinical trials in adolescents and adults with hemophilia A have preliminarily confirmed the efficacy and safety of the product.