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Efanesoctocog alfa in hemophilia A: a profile of its use

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posted on 2024-02-18, 20:18 authored by Simon Fung, Hannah A. Blair

Declarations

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

Adis medical writers(s) Authorship and conflict of interest S. Fung and H. A. Blair are salaried employees of Adis International Ltd/Springer Nature and declare no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.

Ethics approval, Consent to participate, Consent for publication, Availability of data and material, Code availability Not applicable.

Additional information about this Adis Drug Review can be found here.

Abstract

Efanesoctocog alfa [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl; ALTUVIIIO®], indicated for routine prophylaxis, on-demand treatment of bleeding episodes, and perioperative management of bleeding, is a useful addition to the therapies available for the management of hemophilia A in adults and children. Its half-life is three- to four-fold longer than existing (standard and extended half-life) factor VIII replacement products, which enables once-weekly administration. In a phase 3 trial, pretreated adolescents and adults (aged ≥ 12 years) receiving efanesoctocog alfa prophylaxis had a low annualized rate of treated bleeding episodes, and in a subgroup of these patients, efanesoctocog alfa reduced the annualized bleeding rate compared with the rate in a pre-study, in which the same patients received standard-of-care factor VIII replacement product prophylaxis. A single dose of efanesoctocog alfa was also able to resolve nearly all bleeding episodes, with most responses being good or excellent. Additionally, hemostatic responses in patients receiving efanesoctocog alfa for perioperative management of bleeding were deemed excellent. Efanesoctocog alfa also had efficacy in preventing bleeding as prophylaxis and resolving bleeding episodes as on-demand treatment in pretreated children (aged < 12 years). Efanesoctocog alfa was well tolerated. No factor VIII inhibitors developed in patients receiving efanesoctocog alfa in patients of any age in these trials.