Genentech recently announced findings from the phase III HAVEN 7 study’s primary analysis at the 2023 American Society of Hematology (ASH) Annual Meeting and Exposition. The data highlighted the efficacy and safety of Hemlibra (emicizumab-kxwh) in previously untreated or minimally treated infants with severe hemophilia A without factor VIII inhibitors. The results also demonstrated that Hemlibra achieved meaningful bleed control in babies aged up to 12 months and was well tolerated.

Steven Pipe, MD, professor of pediatrics and pathology at the University of Michigan, stated, “Hemophilia A can have a devastating impact on any patient, but this is especially true for infants, where the emotional and physical stress due to frequent hospital visits, treatment administration, and other worries can be distressing for babies and their parents and caregivers.” Dr. Pipe also added, “These results reinforce the benefit of starting prophylaxis as soon as possible after birth, as well as for the use of subcutaneous treatments, which are especially valuable in young babies where access to veins can be very difficult.”

The HAVEN 7 study is a phase III, descriptive, single-arm study designed in collaboration with the hemophilia A community, and the goal of the study is to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of SC Hemlibra in infants with severe hemophilia A without factor VIII inhibitors.

The results included data from 55 participants and revealed that at 101.9 weeks median follow-up, 54.5% of participants (n = 30) did not experience any bleeds that warranted treatment, while 16.4% (n = 9) did not have any treated or untreated bleeds. The results also revealed that there were no spontaneous bleeds requiring treatment in any participant, and all treated bleeds were as a result of trauma.

Moreover, in 46 participants (83.6%) 207 bleeds occurred, of which 87.9% were as a result of trauma, and the model-based annualized bleeding rate (95% CI) was 0.4 (0.30-0.63) for treated bleeds.

No new safety signals were documented among participants, and no treatment-related serious adverse events, intracranial hemorrhages, or deaths were reported. Additionally, 3.6% of participants (n = 2) tested positive for factor VIII inhibitors, which may be a result of reduced factor VIII usage in participants treated with Hemlibra, and no participant tested positive for antidrug antibodies. The presenters concluded that the results were consistent with positive results from the interim analysis and from previous phase III HAVEN studies.

“The results of HAVEN 7 provide additional confidence in the efficacy and safety profile of Hemlibra for babies with severe hemophilia A and add to its extensive clinical and real-world evidence across all ages,” stated Levi Garraway, MD, PhD, CMO, head of Global Product Development. “Conducted in collaboration with the hemophilia A community, this trial reflects our ongoing commitment to listen and respond to the needs of those impacted by this condition in hopes of advancing treatment standards even further.”

The results of additional research on biomarkers in the HAVEN 7 study were also presented at the 2023 ASH Meeting and Exposition and confirmed the study’s primary efficacy analysis. The presenters indicated that this added research demonstrated that the pharmacodynamic profiles of Hemlibra in babies were consistent with those previously observed in older children and adults with hemophilia A. The data demonstrated that Hemlibra exhibits the anticipated pharmacodynamic response despite the diminished presence of the clotting factors that Hemlibra binds to in this age group.

The presenters also noted that the results from the phase III HAVEN 7 study added to the data from the larger, pivotal HAVEN clinical program, providing an understanding of the progression of hemophilia A in babies and the impact of introducing preventative treatment from birth. The primary analysis is being tracked by a 7-year extension period, and Hemlibra continues to redefine standards of care in hemophilia A as a flexible treatment option approved across all ages and stages of life irrespective of inhibitor status and at different dosing frequencies.

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