emicizumab   Click here for help

GtoPdb Ligand ID: 9744

Synonyms: ACE-910 | ACE910 | emicizumab-kxwh | Hemlibra® | RG6013
Approved drug
emicizumab is an approved drug (FDA (2017), EMA (2018))
Compound class: Antibody
Comment: Emicizumab, is a humanised bispecific antibody that binds blood coagulation factors IXa and X and acts as a factor VIII mimetic to bring about activation of factor X, and promote coagulation [2-3,5]. In essence, emicizumab works by bridging between coagulation factors IX and X and is the first-in-class drug for haemophilia A prevention with this mechanism of action.
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Summary of Clinical Use Click here for help
Emicizumab was FDA approved in November 2017 as a routine prophylactic treatment for patients with factor VIII deficiency who have developed factor VIII-neutralising antibodies (inhibitors) in response to replacement factor VIII therapy [1]. See Phase 3 clinical trial NCT02622321 [4]. Emicizumab had EMA orphan medicinal product designation from January 2014-May 2017, for the management of haemophilia A, prior to marketing authorisation being granted in early 2018. In October 2018, FDA approval was expanded to permit treatment of hemophilia A patients regardless of factor VIII inhibitor status.
In Japan, emicizumab is in Phase 3 trial as a potential treatment for the additional indication of acquired (autoimmune) hemophilia A [6].
Mechanism Of Action and Pharmacodynamic Effects Click here for help
Emicizumab mimics the function of factor VIII to promote factor X activation and blood coagulation in patients with haemophilia A.
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