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Efmoroctocog Alfa: A Review in Haemophilia A

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Abstract

Efmoroctocog alfa (Elocta®, Eloctate®, Eloctate™), a first-in-class recombinant factor VIII-Fc fusion protein (rFVIIIFc), has an extended half-life compared with conventional factor VIII (FVIII) preparations, including recombinant FVIII (rFVIII) products. It is approved for the treatment and prophylaxis of bleeding in patients with haemophilia A in multiple countries worldwide. Data accumulated from pivotal phase III studies (A-LONG in adults and adolescents aged ≥12 years; Kids A-LONG in children aged <12 years) and their ongoing extension study (ASPIRE) have demonstrated the long-term effectiveness of efmoroctocog alfa for the treatment of acute bleeding episodes, perioperative management and routine prophylaxis in previously treated males with severe haemophilia A. Among patients on individualized efmoroctocog alfa prophylaxis who had previously received FVIII prophylaxis, all but one of those aged ≥12 years and three-quarters of those aged <12 years reduced their injection frequency compared with their pre-study regimen. FVIII replacement therapy with efmoroctocog alfa was generally well tolerated in previously treated patients, with no evidence of increased immunogenicity. The safety and efficacy of FVIII replacement therapy with efmoroctocog alfa in previously untreated males aged <6 years with severe haemophilia A are currently being evaluated. Although there are no direct, head-to-head studies, the available clinical trial evidence indicates that efmoroctocog alfa provides an effective alternative to conventional FVIII preparations (including rFVIIIs) for the management of haemophilia A. Moreover, by reducing the frequency of injections required, it has the potential to reduce treatment burden, and hence improve adherence to prophylaxis.

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Acknowledgments

During the peer review process, the manufacturer and market authorization holders of efmoroctocog alfa were 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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    James E. Frampton

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  1. James E. Frampton
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Correspondence to James E. Frampton.

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

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James Frampton is a salaried employee of Adis/Springer, is responsible for the article content and declares no relevant conflicts of interest.

Additional information

The manuscript was reviewed by: E. Berntorp, Department of Translational Medicine, Lund University and Centre for Thrombosis and Haemostasis, Skane University Hospital, Malmö, Sweden; J. S. Powell, Division of Hematology and Oncology, University of California Davis Medical Center, Sacramento, CA, USA.

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Frampton, J.E. Efmoroctocog Alfa: A Review in Haemophilia A. Drugs 76, 1281–1291 (2016). https://doi.org/10.1007/s40265-016-0622-z

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