Emicizumab-kxwh (Hemlibra, Genentech) demonstrated a favorable safety profile and effective bleed control in people with mild or moderate hemophilia A without factor VIII inhibitors, according to the results of an interim analysis of the phase 3 HAVEN 6 study. These data were presented at the American Society of Hematology Annual Meeting and Exposition 2021.
“We are pleased to see that Hemlibra continues to show benefit in additional hemophilia A populations, regardless of severity,” said Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development at Genentech, in a press release. “The clinical evidence for Hemlibra derives from one of the largest pivotal clinical trial programs in hemophilia A, with and without factor VIII inhibitors. We remain committed to working together with the hemophilia community as we further explore the efficacy and safety of Hemlibra in broader populations.”
According to the investigators, although the treatment and management of severe hemophilia A are well established, less information and guidance is available for patients with mild or moderate hemophilia A, which can lead to delayed or missed diagnoses of bleeding episodes. As a result, these patients are more likely to have a worsened clinical burden, with less than 30% of individuals with mild or moderate hemophilia A living a bleed-free life, according to the study.
HAVEN 6 is designed to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of Hemlibra in patients with mild or moderate hemophilia A without factor VIII inhibitors. The interim analysis included 71 participants, and was conducted after 50 participants with moderate hemophilia A completed at least 24 weeks in the study or withdrew, with a data cutoff on April 16, 2021.
The study data demonstrated that Hemlibra was associated with effective bleed control and a favorable safety profile, with 80.3% of participants experiencing no bleeding episodes that required treatment and 90.1% experiencing no joint bleeds that required treatment. Consistent with the HAVEN 1 through 4 studies, annualized bleeding rates remained low.
The most common adverse events (AEs), occurring in 10% or more of the study participants, were headache and local injection site reactions. Eleven people (15.5%) reported a Hemlibra-related AE, with injection site reactions being the most common. There were no cases of thrombotic microangiopathy, serious thrombotic events, or deaths in the study.
Interim data from phase III HAVEN 6 study demonstrate favorable safety and efficacy profile of Genentech’s Hemlibra (emicizumab-kxwh) in people with moderate or mild hemophilia A