FDA Approves Updated Label for Altuviiio to include Full Data on Pediatric Patients With Hemophilia A

Antihemophilic factor (recombinant) Fc-VWF-XTEN fusion protein-ehtl was initally approved in February 2023 for adults and children with hemophilia A for prophylaxis and on-demand treatment to control bleeding.

The FDA has approved an updated label for antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl (Altuviiio; Sanofi), to include the full results form the phase 3 XTEND-Kids study, demonstrating that once-weekly dosing of the drug provides bleed protection in pediatric patients with hemophilia A.

The drug was first approved in February 2023 for both children and adults for routine prophylaxis and on-demand treatment as well as perioperative management, according to a press release. The update includes the full results from the study, which highlights the clinical evidence of the drug as a treatment option for all patients.

“Hemophilia is a lifelong condition, so starting children on an early prophylactic regimen can help them stay ahead of bleeds and reduce other potential complications, such as joint damage and pain. Delivering significant bleed protection with a reduced treatment burden can allow families to spend more time on activities and less time focused on infusion schedules," Mindy Simpson, MD, pediatric hematologist/oncologist and assistant professor of pediatrics at Rush University Medical Center, said in the press release.

May 15, 2024