The U.S. Food and Drug Administration (FDA) has approved updated prescribing information for ALTUVIIIO™ (Sanofi) to include the full results of the phase 3 XTEND-Kids clinical trial. 


ALTUVIIIO is a recombinant factor VIII therapy (rFVIII), developed with proprietary Fc fusion technologies to extend the half-life of the rFVIII, allowing for more sustained protection from bleeds per infusion. The product first received FDA approval in February 2023 for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A.


Phase 3 XTEND-Kids is evaluating once weekly prophylaxis with ALTUVIIIO in previously treated patients younger than 12 years of age with severe hemophilia A. 


Children with hemophilia often clear factor replacement therapies for their bloodstreams quicker than adults, necessitating multiple infusions per week. However, according to a new company press release, the updated findings suggest that a once weekly dose of ALTUVIIIO provides “highly effective” protection from bleeds in younger children with hemophilia A. 


Sanofi also reports that ALTUVIIIO met its primary endpoint of safety with no factor VIII inhibitor development detected by study investigators. It also met key secondary endpoints, including a mean annualized bleeding rate (ABR) of 0.6 and a median ABR of 0.0. 


In terms of safety, no serious allergic reactions, anaphylaxis, or embolic or thrombotic events were reported. While no inhibitors were detected in the XTEND-Kids study or reported in post-market surveillance to date, the company notes that the formation of inhibitors and allergic reactions are possible when taking ALTUVIIIO. The most common adverse drug reaction was fever. Lastly, no adverse events led to the discontinuation of treatment.


“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,” said Mindy Simpson, MD in the release. Simpson is a Pediatric Hematologist/Oncologist and Assistant Professor of Pediatrics, at the Rush University Medical Center in Chicago, IL.


Source: Sanofi press release dated May 10, 2024