HEMLIBRA® was studied in 2 clinical trials, HAVEN 1 and HAVEN 2 interim, in adults, adolescents, and children with hemophilia A with factor VIII inhibitors. To learn more about the study design, please visit the HEMLIBRA Clinical Trials page.

HAVEN 1: efficacy endpoints

Primary endpoint

  • Adults and adolescents on HEMLIBRA had a median annualized bleed rate of 0 (IQR 0; 3.7) for treated bleeds vs 18.8 (IQR 13.0; 35.1) in patients receiving no prophylaxis
 
To learn more about our safety information, please visit the HEMLIBRA Adverse Reactions page.

Secondary endpoint (intra-patient comparison)

  • Adults and adolescents on HEMLIBRA had a median annualized bleed rate of 0 (IQR 0; 2.2) for treated bleeds vs 12.0 (IQR 5.7; 24.2) in patients treated with previous prophylactic bypassing agents

Annualized bleed rate calculated with a negative binomial regression model, which accounts for different follow-up times.
CI=confidence interval; IQR=interquartile range.
*Defined as bleeds treated with bypassing agents based on an adaptation of standard criteria defined by the International Society on Thrombosis and Haemostasis (ISTH).
A comparison of data from HAVEN 1 vs observations in the non-interventional study prior to enrollment.

 

Primary and secondary endpoints

HEMLIBRA met all primary and secondary bleed endpoints vs no prophylaxis in adults and adolescents

  • Adults and adolescents on HEMLIBRA had a median annualized bleed rate of 0 for all types of treated bleeds

Defined as bleeds treated with bypassing agents.
§Defined as the occurrence of hemorrhage where a reason cannot be identified.
Defined as aura in the joint combined with increasing swelling or warmth of the skin over the joint, increasing pain, and/or progressive loss of range of motion or difficulty in using the limb vs baseline.
Defined as a major joint into which repeated bleeds occur (at least 3 bleeds into the same joint over 24 weeks prior to study entry).
#Defined as bleeds treated and not treated with bypassing agents.

HAVEN 2: efficacy endpoints

Interim analysis after at least 12 weeks of treatment (single-arm trial)

  • At the time of interim analysis, efficacy of HEMLIBRA was evaluated in 23 children
  • Children on HEMLIBRA had a median annualized bleed rate of 0 (IQR 0; 0) for treated bleeds
  • The median observation time for these patients was 38.1 (12.7–41.6) weeks

Interim analysis after at least 12 weeks of treatment (intra-patient comparison)**

Children on HEMLIBRA had a 99% reduction in treated bleeds, with an annualized bleed rate of 0.2 (95% CI: 0.1; 0.8) vs 17.2 (95% CI: 12.4; 23.8) in patients treated with previous bypassing agents (n=13)††

  • 11 out of 13 children (85%) on HEMLIBRA had zero treated bleeds 
  • HAVEN 2 outcomes are from an interim analysis. Statistical significance will be conducted at final analysis

**A comparison of data from HAVEN 2 vs observations in the non-interventional study prior to enrollment.
††12 patients had previous prophylactic treatment and 1 patient had previous on-demand treatment with bypassing agents.

Interim analysis after at least 12 weeks of treatment

  • Children on HEMLIBRA had a median annualized bleed rate of 0 for all types of treated bleeds

Clinical Trials

Learn more about HAVEN 1 and HAVEN 2 clinical trials

Adverse Reactions

Adverse reactions from pooled clinical studies with HEMLIBRA

How HEMLIBRA Is Made

Genentech’s manufacturing process and standards