Roche announced that the primary analysis of the Phase III HAVEN 7 study reinforced the efficacy and safety of Hemlibra® (emicizumab) in previously untreated or minimally treated infants with severe haemophilia A without factor VIII inhibitors.
Results showed that Hemlibra achieved meaningful bleed control in babies up to 12 months of age, and was well tolerated. The new data were presented at the 65th American Society of Hematology Annual Meeting and Exposition taking place 9-12 December 2023, in San Diego, California, and included in the press programme.
“Haemophilia 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,” said Steven Pipe, MD, professor of paediatrics and pathology at the University of Michigan. “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 burden of severe haemophilia A in babies and on their parents and caregivers is significant. The World Federation of Haemophilia treatment guidelines consider the standard of care in haemophilia to be regular prophylaxis initiated at a young age, as studies have shown this improves long-term outcomes, while reducing the risk of intracranial haemorrhage.
However, for many babies with haemophilia A, prophylaxis is not started until after the first year of life because of the high treatment burden. Hemlibra, which is already approved and being used to treat babies with haemophilia A, provides a flexible treatment option that can be administered subcutaneously from birth at different dosing frequencies.
“The results of HAVEN 7 provide additional confidence in the efficacy and safety profile of Hemlibra for babies with severe haemophilia A, and add to its extensive clinical and real-world evidence across all ages,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Conducted in collaboration with the haemophilia 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 HAVEN 7 study is a Phase III, descriptive, single-arm study, set up in collaboration with the haemophilia A community to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of subcutaneous Hemlibra in infants with severe haemophilia A without factor VIII inhibitors.
👉 New data reinforce the benefit of early preventative treatment with Roche’s Hemlibra for babies with severe haemophilia A
