Demonstrated safety profile across a broad range of patients over the course of 5 years 1-7

HEMLIBRA has been evaluated in the largest clinical trial program of hemophilia A patients with and without FVIII inhibitors. 1,3

Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of aPCC was administered for 24 hours or more to patients receiving HEMLIBRA prophylaxis. 1

The pooled safety data for HEMLIBRA were collected in 391 patients across 5 clinical trials over the course of 5 years, including HAVEN 1, HAVEN 2, HAVEN 3, HAVEN 4, and 1 dose-finding trial. 1

*All ISRs were reported as mild to moderate in intensity; 93% resolved without treatment. 1

As with all therapeutic proteins, there is a potential for immunogenicity 1

Overview of ADAs observed

  • The detection of antibody formation is highly dependent on the sensitivity and specificity of the assay
  • In the dose-finding trial (n=18), 4 patients tested positive for ADAs
  • In pooled clinical trials, 3.5% of patients (14/398) tested positive for ADAs
  • <1% of patients (3/398) developed ADAs with neutralizing potential (based on declining pharmacokinetics) 
    • 1 patient from HAVEN 2, who developed a neutralizing ADA, experienced loss of efficacy after 5 weeks of treatment
  • There was no clinically apparent impact of the presence of ADAs on safety

Surgical experience 2,19

Retrospective review of patients undergoing surgeries and procedures in HAVEN trials.

Analysis of the use of additional BPAs (HAVEN 1 and HAVEN 2) or FVIII (HAVEN 3) for surgeries was not an objective of the HAVEN studies, and there is limited experience from the clinical trials. Click here to see retrospective surgery data.

ADAs=anti-drug antibodies; FVIII=factor VIII; aPCC=activated prothrombin complex concentrate.

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