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Wolfgang Miesbach: A Remarkable Progress With Non-Factor Therapies in Hemophilia Care
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.”

Wolfgang Miesbach: A Remarkable Progress With Non-Factor Therapies in Hemophilia Care

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bleeding disorders Concizumab Denecimig EAHAD 2026 Emicizumab Fitusiran Headlines Health Hematology Hemophilia Hemostasis Hemostasis News Hemostasis Today Maria Elisa Mancuso Marstacimab Medicine Mim8 NFTs Wolfgang Miesbach
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