Chugai Pharmaceutical Co., Ltd announced that emicizumab received orphan drug designation by the Ministry of Health, Labour and Welfare for the prevention and reduction of bleeding episodes in patients with congenital FVIIl deficiency (hemophilia A) who developed inhibitors to FVIII. Emicizumab is a humanized bispecific antibody for subcutaneous injection under development for hemophilia A.
Hemophilia A is an inherited disorder in which the blood coagulation reaction does not proceed normally due to a congenital lack or impaired function of coagulation FVlll. People with hemophilia A begin to experience severe bleeding repeatedly in various body parts such as joints and muscle in their childhood. Patients with severe hemophilia A currently receive regular FVIII replacement therapy to reduce the frequency of bleeding. For those patients who develop antibodies (inhibitors) against FVlll, bypassing agents are used to promote blood-clotting reaction independently from FVIII.
“With the results from Japanese Phase I/II study (ACE002JP) of emicizumab we have seen so far, I have high hopes that the drug can bring innovation to current treatment for hemophilia A both in terms of efficacy and improvement in quality of life,” said Chugai’s Senior Vice President, Head of Project & Lifecycle Management Unit, Dr. Yasushi Ito. “Particularly, there are high unmet medical needs for patients with inhibitors. We are committed to deliver emicizumab to patients as early as possible to contribute for the realization of better treatments.”
Primary patient enrolment has been completed in the global Phase lll study in adolescents/adults with hemophilia A who acquired FVIII inhibitor, and the study is now under the follow-up phase.
