Sanofi and Sobi’s Altuviiio (antihaemophilic factor [recombinant], Fc-VWF-XTEN fusion protein-ehtl) label has been updated by the US Food and Drug Administration (FDA) to include new paediatric data from a phase 3 study in children with haemophilia A.
Haemophilia A is a rare hereditary bleeding disorder that occurs when there is a lack of sufficient functioning factor VIII protein to help the blood clot, leading to external bleeding, bruising and bleeding into joints.
Designed to extend protection from bleeding, Altuviiio allows for weekly dosing as it has a half-life that is up to four times longer than standard and extended half-life factor VIII products.
Building on previous results from the XTEND-Kids study from 2023, new data demonstrated that once-weekly dosing with Altuviiio delivered highly effective bleeding protection in children younger than 12 years living with the inherited condition.
Altuviiio met the primary endpoint of safety with no factor VIII inhibitor development detected, as well as key secondary endpoints, including a mean annualised bleeding rate (ABR) of 0.6 and a median ABR of 0.0 in 72 patients.
Additionally, the treatment demonstrated a similar safety profile to that shown in the XTEND-1 trial, which evaluated Altuviiio in people aged 12 years and older, with no new serious allergic reactions, anaphylaxis, embolic, thrombotic, or adverse events reported.
Mindy Simpson, paediatric haematologist/oncologist and assistant professor of paediatrics, Rush University Medical Center, commented: “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.”
Originally granted Breakthrough Therapy Designation by the FDA in May 2022, the US regulator approved Altuviiio in February 2023 to treat adults and children with haemophilia A for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as for perioperative management.
Marked as the first regulatory approval of Altuviiio, the FDA’s decision was based on results from the phase 3 XTEND-1 study, which demonstrated that the therapy showed superiority to prior factor prophylaxis treatment.
Paul Hudson, chief executive officer, Sanofi, said that the approval “allows patients and physicians to reimagine living with haemophilia”, which makes “powerful bleeding protection… a reality for patients”.