Safety evaluated in the largest clinical trial program of hemophilia A patients with and without FVIII inhibitors

Pooled safety comparison included 391 patients

  • The pooled safety analysis also included patients with and without inhibitors from the HEMLIBRA dose-finding study (n=18)

Cases of TMA 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

Known safety profile established across multiple clinical trials

  • The most common adverse reactions (≥10%) were injection site reactions, arthralgia, and headache

Pooled safety data
 

Patients with FVIII inhibitors

Patients without FVIII inhibitors

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

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

HAVEN 3 surgical experience

Retrospective review of patients undergoing surgeries and procedures in HAVEN 3

  • Analysis of the use of FVIII for surgeries or procedures was not an objective of the pivotal study, and there is limited experience from the clinical trial 2
  • In the HAVEN 3 trial, 28 patients underwent 46 minor procedures and 4 major procedures. No unexpected bleeding or complication was related to any procedure 2

ADAs=anti-drug antibodies; aPCC=activated prothrombin complex concentrate; FVIII=factor VIII; TMA=thrombotic microangiopathy.
*Overall, TMA and thrombotic events were reported in 0.8% (3/391) and 0.5% (2/391) of patients, respectively.

Efficacy in Patients Without Inhibitors

Learn about the primary and secondary results for patients without FVIII inhibitors

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Laboratory Monitoring Information

Considerations for using laboratory tests to monitor patients taking HEMLIBRA