EMICIZUMAB DATA
Clinical Trials
Emicizumab’s effectiveness, safety, and movement through the body (pharmacokinetics) were evaluated in phase 3 studies in people with hemophilia A. Learn more below about the data from these studies.
The Genentech resources below are patient-friendly summaries of data from clinical trials. These patient-friendly summaries include studies with the strongest and most relevant data and are provided only for educational purposes. You are encouraged to discuss any questions you have about your condition and any current or potential treatments with your heathcare provider.
HEMLIBRA Use in Hemophilia A Without Inhibitors
HEMLIBRA Use in Hemophilia A With Inhibitors
HEMLIBRA Use in Children Less Than 12 Years Old With Hemophilia A Without Inhibitors
Select publications and scientific data presentations with the strongest and most relevant data are shown below. Click to view full publications, where available.
Severe Hemophilia A
With Factor VIII Inhibitors (Adults and Young Adults): HAVEN 1 Study
Emicizumab Prophylaxis in Hemophilia A With Inhibitors.
Oldenburg J, Mahlangu JN, Kim B, et al. N Engl J Med. 2017;377:809–818.
With Factor VIII Inhibitors (Children): HAVEN 2 Study
A Multicenter, Open-Label Phase 3 Study of Emicizumab Prophylaxis in Children With Hemophilia A With Inhibitors
Young G, Liesner R, Chang T, et al. Blood. 2019;134:2127–2138.
Without Factor VIII Inhibitors (Adults and Young Adults): HAVEN 3 Study
Emicizumab Prophylaxis in Patients Who Have Hemophilia A Without Inhibitors.
Mahlangu J, Oldenburg J, Pza-Priel I, et al. N Engl J Med. 2018;379:811-822.
With or Without Factor VIII Inhibitors (Adults and Young Adults): HAVEN 4 Study
Efficacy, Safety, and Pharmacokinetics of Emicizumab Prophylaxis Given Every 4 Weeks in People With Haemophilia A (HAVEN 4): A Multicentre, Open-Label, Non-Randomised Phase 3 Study.
Pipe SW, Shima M, Lehle M, et al. Lancet Haematol. 2019;6:e295–e305.
Without Factor VIII Inhibitors (Children): HOHOEMI Study
A Multicentre, Open-Label Study of Emicizumab Given Every 2 or 4 Weeks in Children With Severe Haemophilia A Without Inhibitors.
Shima M, Nogami K, Nagami S, et al. Haemophilia. 2019;25:979–987.
Without Factor VIII Inhibitors (Previously Untreated or Minimally Treated Patients/Infants): HAVEN 7 Study
Emicizumab Prophylaxis in Infants With Hemophilia A (HAVEN 7): Primary Analysis of a Phase 3b, Open-Label Trial
Pipe SW, Collins PW, Dhalluin C, et al. Blood. 2024;143:1355–1364.
Mild or Moderate Hemophilia A
Without Factor VIII Inhibitors (All Ages): HAVEN 6 Study
Emicizumab in People With Moderate or Mild Haemophilia A (HAVEN 6): A Multicentre, Open-Label, Single-Arm, Phase 3 Study.
Négrier C, Mahlangu J, Lehle M, et al. Lancet Haematol. 2023;10(3):e168-e177.