ALTUVIIIO phase 3 data on bleed protection in children with hemophilia A
Lynn Malec, MD, joined us to discuss the phase 3 XTEND-Kids study of ALTUVIIIO in pediatric patients with hemophilia A.
On May 10, 2024, the FDA approved an updated label for ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] (Sanofi), with data demonstrating that once-weekly dosing provides bleed protection in children with hemophilia A.1
Lynn Malec, MD, medical director, Comprehensive Center for Bleeding Disorder; associate investigator, The Versiti Blood Research Institute; and associate professor of Medicine and Pediatrics, The Medical College of Wisconsin, joined Contemporary Pediatrics to discuss the data from the phase 3 XTEND-Kids study (NCT04759131).1
"This was a medicine that was approved for prevention of bleeds in hemophilia A [patients] using just once-weekly dosing, which is quite a change for the way patients are able to be treated in the hemophilia community," Malec said. "The XTEND-Kids study was a pediatric study alone. Patients [who] were eligible for this study are patients with severe hemophilia A that had previously been treated with a factor 8 product."
ALTUVIIIO provided high-sustained factor levels with once-weekly dosing in children under 12 with hemophilia A, with XTEND-Kids results demonstrating highly effective bleed protection in hemophilia A with no inhibitor development to factor VIII, according to Sanofi.2
"What we saw in the XTEND-Kids study was that thankfully none of the patients developed inhibitors, so none of them developed an immune reaction to the factor 8 medicine that would make it ineffective," Malec said. "More importantly for me as a treater, as a safety signal we saw that there was really excellent bleed control for patients in the study."
References:
1. Fitch J. FDA approves ALTUVIIIO updated label for bleed protection in children with hemophilia. Contemporary Pediatrics. May 13, 2024. Accessed August 12, 2024. https://www.contemporarypediatrics.com/view/fda-approves-altuviiio-updated-label-for-bleed-protection-in-children-with-hemophilia-a
2. Press Release: NEJM publishes ALTUVIIIO XTEND-Kids phase 3 data supporting its potential to transform the treatment landscape for children with severe hemophilia A. Sanofi. Press release. July 17, 2024. Accessed August 12, 2024.
