Hemophilia is a blood clotting disorder caused by a congenital deficiency of blood clotting factors and is the rare disease with the highest incidence (about 1 in 10,000). There are about 140,000 hemophiliacs in China, including those suffering from hemophilia A (coagulation factor VIII deficiency) accounting for 80-85%, and those suffering from hemophilia B (coagulation factor IX deficiency) accounting for 15-20%.
Currently, coagulation factor replacement therapy is the mainstay of treatment for the disease. Patients with severe hemophilia, without prompt and adequate clotting factor therapy, have a high rate of disability or, in some cases, are at risk of life-threatening. However, the high annual cost of treatment for hemophilia coupled with the fact that the supply of drugs in China lags far behind the world’s average has placed a heavy burden on patients’ families and the society. With the introduction of long-acting coagulation factors in the last decade, patients in developed countries abroad have entered the stage of prophylactic treatment, which has greatly reduced the bleeding rate of patients and improved their quality of life. In contrast, patients at home can still only rely on short-acting coagulation factors for on-demand treatment, only satisfying the need for hemostasis and pain relief, and resulting in a quality of their survival far inferior to that of prophylactic patients. Therefore, the gaps in the capacity and efficacy of coagulation factor products in China need to be filled. Domestic long-acting coagulation factor products have been the largest unmet clinical need in China’s hemophilia field.
After years of deep involvement in the field of hemophilia, Gensciences has developed a full series of long-acting coagulation factor products covering the main indications such as hemophilia A (factor VIII), hemophilia B (factor IX), and hemophilia with inhibitors (activated factor VII).