Research & Development

Data shared by Sanofi about new drug for treatment of haemophilia A in children

Sanofi has shared new data from the phase 3 XTEND-Kids study, which assessed Altuviiio (Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein) once-weekly prophylaxis in patients under the age of 12 with previously treated severe haemophilia A.
This data was presented at the Annual Meeting of the International Society on Thrombosis and Haemostatis (ISTH) in Montreal, Canada.
The drug is a first-in-class, high-sustained factor VIII replacement therapy indicated for once-weekly treatment, compared to standard of care for paediatric patients who often need injections two to four times per week.
Having been approved by the US Food and Drug Administration (FDA) for routine prophylaxis and on-demand treatment for adults and children with haemophilia A in February 2023, Altuviiio has now met the primary endpoint of the phase 3 trial with no inhibitor development to factor VIII detected, as well as meeting key secondary endpoints.
Lynn Malec MD, medical director of the Comprehensive Center for Bleeding Disorders, associate investigator at The Versiti Blood Research Institute and associate professor of Medicine and Pediatrics at The Medical College of Wisconsin, US, commented: “The results from XTEND-Kids mark an important breakthrough as we strive for optimised bleed protection as the standard of care. Achieving high-sustained factor activity with once-weekly dosing means a freedom from the trade-offs between treatment burden and efficacy we often see in treating severe haemophilia A. The reliable and consistent bleed protection Altuviiio provides offers confidence for children living with haemophilia and their families to manage haemophilia with less worry.”
Karin Knobe MD PhD, therapeutic area head, Rare Diseases and Rare Blood Disorders at Sanofi, added: “In an effort to reduce their risk of bleeding episodes, many children living with haemophilia A are currently limited in their ability to fully participate in daily activities. This burden is compounded by the challenge of administering prophylactic treatments intravenously multiple times a week. Today’s XTEND-Kids results reinforce the ability of Altuviiio to provide effective bleed protection with once-weekly dosing and reinforce our commitment to developing new treatment options designed to redefine the standard of care for people living with rare blood disorders.”