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Emicizumab: A Review in Haemophilia A

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Abstract

Emicizumab (Hemlibra®), a recombinant, humanized, bispecific monoclonal antibody, restores the function of missing activated factor VIII (FVIII) by bridging FIXa and FX to facilitate effective haemostasis in patients with haemophilia A. Subcutaneous emicizumab is approved in several countries, including in the USA and Japan, for the routine prophylaxis of bleeding episodes in patients with haemophilia A with or without FVIII inhibitors. It is also approved in the EU for the routine prophylaxis of bleeding episodes in patients with haemophilia A with inhibitors or severe haemophilia A without inhibitors. In phase III clinical trials, emicizumab prophylaxis significantly reduced annualized bleeding rates compared with no prophylaxis in adolescents and adults with haemophilia A with or without inhibitors, and prevented or substantially reduced bleeding in children with haemophilia A with or without inhibitors. Emicizumab was also associated with beneficial effects on health-related quality of life and health status, and was generally well tolerated. In view of its convenient route of administration and versatile dosage regimens (maintenance dose of once every 1, 2 or 4 weeks), emicizumab provides an effective and generally well-tolerated alternative to conventional FVIII replacement products for the prophylaxis of bleeding episodes in patients with haemophilia A, regardless of the presence or absence of inhibitors.

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Acknowledgements

During the review process, the manufacturer of emicizumab was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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    Hannah A. Blair

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  1. Hannah A. Blair
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Correspondence to Hannah A. Blair.

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Hannah Blair is a salaried employee of Adis International Ltd/Springer Nature, is responsible for the article content and declares no relevant conflicts of interest.

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The preparation of this review was not supported by any external funding.

Additional information

Enhanced material for this Adis Drug Evaluation can be found at https://doi.org/10.6084/m9.figshare.9784490.

The manuscript was reviewed by:J. N. Mahlangu, Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital and Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa; M. E. Mancuso, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy; J. Oldenburg, University Clinic Bonn, Institute for Experimental Haematology and Transfusion Medicine, Bonn, Germany.

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Blair, H.A. Emicizumab: A Review in Haemophilia A. Drugs 79, 1697–1707 (2019). https://doi.org/10.1007/s40265-019-01200-2

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