Wolfgang Miesbach, Maria Elisa Mancuso/LinkedIn
Mar 26, 2026, 15:19
Wolfgang Miesbach: A Remarkable Progress With Non-Factor Therapies in Hemophilia Care
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared on LinkedIn:
”Non-factor therapies in haemophilia – a paradigm shift for haemophilia treatment!
Excellent presentation by Maria Elisa Mancuso at EAHAD 2026 in Dublin, covering the remarkable progress with non-factor therapies (NFTs) – and important questions still ahead.
Emicizumab, fitusiran, concizumab, marstacimab, and the emerging FVIIIa mimetics (MIM8/denecimig, NXT007) enable effective prophylaxis across a broad patient spectrum – including those with inhibitors and children with haemophilia A – with dramatically reduced treatment burden:
- Emicizumab: mean ABR 0.2–1.4 across clinical trials and real-world data (66.7% zero bleeds across all studies)
- Fitusiran (ATLAS programme): mean ABR 1.7 in inhibitor patients, 3.1 in non-inhibitor patients
- Concizumab: 86% reduction in ABR in HA without inhibitors vs. no prophylaxis; 79% reduction in HB
- MIM8/denecimig: mean ABR 1.54 (QM) at 52 weeks
- Marstacimab: 87% reduction in ABR vs. on-demand in inhibitor patients
Remaining challenges and unknowns:
- Long-term impact on joint health remains unknown
- FVIIIa mimetics have lower affinity to FIX/FX than FVIII – FVIII-equivalence is hard to establish and animal models have proven unreliable
- Absence of routine lab assays to measure precise hemostatic activity
- Quality of haemostasis: fibrin clot structure under emicizumab or MIM8 differs from normal (as shown elegantly by scanning electron microscopy – Rezigue et al., Thromb Res 2025)
- Thrombotic risk: 5 thrombotic/TMA events with emicizumab plus high-dose aPCC (HAVEN 1); thrombotic events also reported for concizumab, fitusiran (mitigated by AT-based dosing), and marstacimab.”
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