Staidson (Beijing) Pharmaceutical Co., Ltd (SHE: 300204), a biopharmaceutical company based in China, has announced that it has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for its Category 1 biologic bemiltenase alfa (STSP-0601). This designation is for the treatment of hemophilia A or B. The product candidate has successfully completed a Phase I study as an on-demand treatment for bleeding episodes in patients with hemophilia A or B with inhibitors, demonstrating a favorable safety and tolerability profile.
Furthermore, Staidson has completed Phase Ib/II studies of bemiltenase alfa as an on-demand treatment for bleeding episodes in patients with hemophilia A or B with inhibitors, as well as a Phase II study as an on-demand treatment for bleeding episodes in patients with hemophilia A or B without inhibitors, with summary reports available. In September 2022, the company received breakthrough therapy designation (BTD) in China.
Hemophilia is a recessive hereditary bleeding disorder associated with the X chromosome, classified into hemophilia A and hemophilia B. Hemophilia A is marked by a deficiency of coagulation factor VIII (F VIII), while hemophilia B is characterized by a deficiency of coagulation factor IX (F IX). Both conditions result from mutations in the corresponding coagulation factor genes.- Flcube.com
