Genetech, a biotechnology company, reported that interim data from a phase 3 study revealed that Hemlibra (emicizumab-kxwh) achieved meaningful bleed control in infants with hemophilia A from birth, according to a news release published in Business Wire. 

The treatment burden of infants with hemophilia A and their parents/caregivers is significant, as prophylactic factor replacement therapy is commonly initiated at a young age. Studies indicate that this approach achieves better long-term results and reduces the risk of intracranial hemorrhage. 

Nevertheless, many infants with hemophilia A do not receive prophylactic treatment until after the first year of life because of the high treatment burden. Genetech has co-developed a novel therapy called Hemlibra, a flexible therapeutic option that can be administered subcutaneously at various dosing frequencies from birth. It is a bispecific factor IXa- and factor X-directed antibody. 

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The interim results from a phase 3, multicenter, open-label study assessing the use of Hemlibra in infants with severe hemophilia A without factor VIII inhibitors (n=54) demonstrated that 77.8% of participants did not experience any bleeding episodes requiring treatment, while 42.5% did not experience any bleeding episodes at all. Meanwhile, in the 57.4% of patients who experienced a bleeding episode, 88.3% of them were traumatic. 

A total of 16.7% of participants reported local injection site reactions, while 14.8% had serious adverse events unrelated to Hemlibra. There were no reports of intracranial hemorrhage, thromboembolic events, or deaths. 

“These initial results support the benefit of starting Hemlibra from birth given that early preventative treatment is essential in infants,” Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development, said. 

Researchers intend to conduct a primary analysis at 52 weeks. In addition, they will continue to follow up on participants for an additional 7 years to ensure that safety and joint health data are collected in a comprehensive manner. 


Interim data from phase III study presented at ASH 2022 show Hemlibra (emicizumab-kxwh) achieved meaningful bleed control in infants from birth. News release. Genetech; December 11, 2022.