The Center for Drug Evaluation (CDE) website indicates that Takeda Pharmaceutical Co., Ltd’s Obizur (susoctocog alfa) has been prioritized for review for the on-demand treatment and control of bleeding events in adults with acquired hemophilia A.
Drug Profile and Mechanism
Acquired hemophilia A is an acquired bleeding disorder characterized by reduced FVIII activity due to the presence of anticoagulant factor VIII autoantibodies in the blood. It is marked by spontaneous bleeding episodes in patients with no prior bleeding history or family history of bleeding, or abnormal bleeding during surgery, trauma, or invasive testing. The annual incidence is about 1.5 per 1 million people, with approximately 60.9% of patients experiencing severe bleeding at their first visit.
Obizur, the first recombinant porcine FVIII (rpFVIII) product approved by the US FDA and European Medicine Agency (EMA) for acquired hemophilia A, has a structure and sequence similar to human coagulation factor VIII (FVIII) and is not easily inactivated by anti-human FVIII autoantibodies. It can replace human FVIII to achieve a significant hemostatic effect.
Clinical Study Results
A prospective, multi-center, open Phase II / III study enrolled patients with acquired hemophilia A, with 945 receiving rpFVIII as first-line treatment. 100% of patients experienced a therapeutically evaluable response (cessation or reduction of bleeding) within 24 hours of dosing. Additionally, as a recombinant protein, Obizur reduces the risk of transmission of blood-borne pathogens.-Fineline Info & Tech
