Emicizumab long-term outcomes and coagulation biomarkers in mild or moderate hemophilia A without inhibitors (HAVEN 6)
Abstract
Background Emicizumab, a bispecific monoclonal antibody, substitutes for activated factor (F)VIII, facilitating hemostasis in people with hemophilia A (HA). HAVEN 6 is a multicenter, open-label, single-arm phase 3 study of emicizumab in people with mild or moderate HA. At primary data cut-off, emicizumab demonstrated clinically meaningful bleed control and a favorable safety profile. Objectives To evaluate the long-term efficacy and safety of emicizumab in HAVEN 6. Methods Participants had mild (FVIII >5–<40 IU/dL) or moderate (FVIII ≥1–≤5 IU/dL) HA without FVIII inhibitors, warranting prophylaxis as assessed by the treating physician. Safety endpoints included adverse events (AEs) and AEs of special interest (e.g. thromboembolic events [TEs] and thrombotic microangiopathies). Efficacy endpoints included annualized bleed rates (ABR) for treated bleeds. Pharmacokinetics (PK), pharmacodynamics (PD), safety biomarkers of coagulation, and F8 genotypes were assessed. Further study details are published in the primary analysis (Négrier et al. Lancet Haematol 2023). Results At cut-off (December 11, 2023), median (interquartile range) follow-up was 155.9 (24.1–192.6) weeks (N = 72). In total, 481 AEs were reported; 93.1% of participants experienced ≥1 AE. Two TEs occurred, one since the primary analysis. Both participants continued emicizumab. Model-based ABR for treated bleeds was 1.0 (95% confidence interval: 0.71–1.35). Therapeutic emicizumab exposure levels were maintained throughout the study. FXIa-triggered thrombin generation peak height was higher compared with levels previously observed in people with severe HA. Safety biomarkers of coagulation were unaffected by emicizumab. F8 genotyping of participants found variants consistent with mild or moderate HA. Conclusions These long-term efficacy and safety data confirm emicizumab as a valuable treatment option for people with non-severe HA.
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BibTeX
@article{hermans2026emicizumab,
title = {{Emicizumab long-term outcomes and coagulation biomarkers in mild or moderate hemophilia A without inhibitors (HAVEN 6)}},
author = {Cedric Hermans MD and Víctor Jiménez-Yuste MD, PhD and Giuliana Ventriglia PhD and Anna Kiialainen PhD and Laurent Frenzel MD, PhD and Olivier Catalani MSc and Jerzy Windyga MD and Johannes Oldenburg MD and Christophe Schmitt PharmD and Amy D. Shapiro MD and Johnny Mahlangu MD and Michaela Lehle PhD and Steven W. Pipe MD},
journal = {Therapeutic Advances in Hematology},
year = {2026},
month = oct,
doi = {10.1177/20406207261475838},
url = {https://doi.org/10.1177/20406207261475838},
note = {CC BY},
}
RIS
TY - JOUR
TI - Emicizumab long-term outcomes and coagulation biomarkers in mild or moderate hemophilia A without inhibitors (HAVEN 6)
AU - Cedric Hermans MD
AU - Víctor Jiménez-Yuste MD, PhD
AU - Giuliana Ventriglia PhD
AU - Anna Kiialainen PhD
AU - Laurent Frenzel MD, PhD
AU - Olivier Catalani MSc
AU - Jerzy Windyga MD
AU - Johannes Oldenburg MD
AU - Christophe Schmitt PharmD
AU - Amy D. Shapiro MD
AU - Johnny Mahlangu MD
AU - Michaela Lehle PhD
AU - Steven W. Pipe MD
PY - 2026
DA - 2026/10/24
JO - Therapeutic Advances in Hematology
DO - 10.1177/20406207261475838
UR - https://doi.org/10.1177/20406207261475838
AB - Background Emicizumab, a bispecific monoclonal antibody, substitutes for activated factor (F)VIII, facilitating hemostasis in people with hemophilia A (HA). HAVEN 6 is a multicenter, open-label, single-arm phase 3 study of emicizumab in people with mild or moderate HA. At primary data cut-off, emicizumab demonstrated clinically meaningful bleed control and a favorable safety profile. Objectives To evaluate the long-term efficacy and safety of emicizumab in HAVEN 6. Methods Participants had mild (FVIII >5–<40 IU/dL) or moderate (FVIII ≥1–≤5 IU/dL) HA without FVIII inhibitors, warranting prophylaxis as assessed by the treating physician. Safety endpoints included adverse events (AEs) and AEs of special interest (e.g. thromboembolic events [TEs] and thrombotic microangiopathies). Efficacy endpoints included annualized bleed rates (ABR) for treated bleeds. Pharmacokinetics (PK), pharmacodynamics (PD), safety biomarkers of coagulation, and F8 genotypes were assessed. Further study details are published in the primary analysis (Négrier et al. Lancet Haematol 2023). Results At cut-off (December 11, 2023), median (interquartile range) follow-up was 155.9 (24.1–192.6) weeks (N = 72). In total, 481 AEs were reported; 93.1% of participants experienced ≥1 AE. Two TEs occurred, one since the primary analysis. Both participants continued emicizumab. Model-based ABR for treated bleeds was 1.0 (95% confidence interval: 0.71–1.35). Therapeutic emicizumab exposure levels were maintained throughout the study. FXIa-triggered thrombin generation peak height was higher compared with levels previously observed in people with severe HA. Safety biomarkers of coagulation were unaffected by emicizumab. F8 genotyping of participants found variants consistent with mild or moderate HA. Conclusions These long-term efficacy and safety data confirm emicizumab as a valuable treatment option for people with non-severe HA.
SN - 2040-6215
SN - 2040-6207
ER -