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Ravulizumab in paroxysmal nocturnal hemoglobinuria: a profile of its use

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Abstract

Ravulizumab (Ultomiris™) is a humanized monoclonal antibody that inhibits terminal complement C5, thereby reducing hemolysis, inflammation, and thrombosis in patients with paroxysmal nocturnal hemoglobinuria (PNH). Ravulizumab was developed by re-engineering eculizumab to create a longer-acting antibody, which allows an 8-weekly administration schedule instead of the 2-weekly schedule required with eculizumab. A single intravenous infusion of ravulizumab provides immediate and complete inhibition of serum free C5. With continued ravulizumab treatment, breakthrough hemolysis rates decrease as a result of eliminating free C5-associated breakthrough hemolysis. The efficacy of ravulizumab is noninferior to that of eculizumab, as determined in complement-inhibitor naive patients and those previously treated with eculizumab. Ravulizumab was generally well tolerated, with a generally comparable tolerability profile to that of eculizumab.

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Acknowledgements

The manuscript was updated from Drugs 2019; 79(3):347–52 [15], and was reviewed by: Richard A. Wells, Department of Hematology/Oncology, Sunnybrook Health Sciences Centre, University of Toronto, ON, Canada; Jong Wook Lee, Department of Haematology, Seoul St. Mary’s Hospital, College of Medicine, The Catholic University of Korea, Seoul, Republic of Korea. During the peer review process, Alexion, the marketing-authorization holder of ravulizumab, was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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  1. Springer Nature, Mairangi Bay, Private Bag 65901, Auckland, 0754, New Zealand

    Kate McKeage & Katherine Ann Lyseng-Williamson

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  1. Kate McKeage
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  2. Katherine Ann Lyseng-Williamson
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Correspondence to Katherine Ann Lyseng-Williamson.

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

Conflict of interest

K. McKeage and K.A. Lyseng-Williamson are employees of Adis International Ltd/Springer Nature and are responsible for the article content. K. McKeage declares no conflicts of interest. K.A. Lyseng-Williamson is the Editor of Drugs & Therapy Perspectives and has no other conflicts of interest to declare. The Editor of D&TP was not involved in any publishing decisions for the manuscript.

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McKeage, K., Lyseng-Williamson, K.A. Ravulizumab in paroxysmal nocturnal hemoglobinuria: a profile of its use. Drugs Ther Perspect 35, 405–412 (2019). https://doi.org/10.1007/s40267-019-00663-3

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  • DOI: https://doi.org/10.1007/s40267-019-00663-3

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