Emicizumab
Emicizumab is a humanized bispecific antibody for the treatment of haemophilia A, developed by Chugai. A Phase I clinical trial found that it was well tolerated by healthy subjects.
In November 2017, it was approved in the United States for treatment of haemophilia A in those who had developed resistance to other treatments. It was subsequently approved by the US FDA in April 2018 under the breakthrough therapy designation for treatment of haemophila A in those who have not developed resistance to other treatments.Mechanism of action
Emicizumab binds to both the activated coagulation factor IX and to factor X, mediating the activation of the latter. This is normally the function of coagulation factor VIII, which is missing in haemophilia A patients.
