Emicizumab Evaluated in Children with Hemophilia A

HAVEN 2 is a clinical trial evaluating the efficacy, safety and pharmacokinetics of emicizumab in children with hemophilia A
HAVEN 2 is a clinical trial evaluating the efficacy, safety and pharmacokinetics of emicizumab in children with hemophilia A

Genentech announced preliminary results from the HAVEN 2 study, a second Phase 3 trial evaluating emicizumab for prophylaxis in children less than 12 years old with hemophilia A and inhibitors to factor VIII.

HAVEN 2 is a single-arm, multicenter, open-label clinical trial evaluating the efficacy, safety and pharmacokinetics of emicizumab in children less than 12 years old with hemophilia A and inhibitors to factor VIII who require treatment with bypassing agents. The study's objectives are to evaluate the number of bleeds over time with emicizumab prophylaxis, safety, pharmacokinetics, health-related quality of life (HRQoL) and proxy HRQoL with aspects of caregiver burden. HAVEN 2 intends to enroll a total of 60 children for its final analysis after 52 weeks of treatment. 

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The interim results from HAVEN 2, after a median of 12 weeks of treatment, showed that prophylaxis with emicizumab is associated with clinically meaningful reduction in the number of bleeds over time. These findings are consistent with data from the first Phase 3 trial, HAVEN 1, in which emicizumab prophylaxis showed a statistically significant and clinically meaningful reduction in the number of bleeds over time, in adults and adolescents over 12 years, compared to no prophylaxis or to prior prophylaxis with bypassing agents. Emicizumab was found to be generally well-tolerated, with injection site reactions and nasopharyngitis as the most common adverse events. Data from both HAVEN studies will be presented at an upcoming medical meeting and submitted to regulatory agencies for approval consideration.

Emicizumab, an investigational bispecific monoclonal antibody designed to bring together factors IXa and X, is a ready-to-use solution for once-weekly subcutaneous injection.

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