Emicizumab has revolutionized hemophilia A care, yet limitations regarding the "ceiling" of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next-generation FVIIIa mimetics. Mim8 (denecimig) demonstrated superiority over prior prophylaxis in the FRONTIER 2 phase 3 study, offering flexible weekly to monthly dosing options. NXT007, engineered with FAST-Ig technology to incorporate dual light chains, has shown potential to achieve non-hemophilic coagulation ranges in recent Phase 1/2 studies. Regarding access, we evaluate low-dose emicizumab regimens as a strategy for resource-constrained settings. While acknowledging their potential to reduce treated bleeds, we provide a critical appraisal of their limitations, particularly regarding the lack of sensitive joint health monitoring and the risk of subclinical breakthrough bleeding. This review provides a comprehensive roadmap for advancing the standard of care while addressing global equity.
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Matsushita et al. (2026) studied this question.
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