Sanofi and Sobi celebrate completion of XTEND-Kids phase 3 study

pharmafile | March 2, 2023 | News story | Research and Development  

A collaborative study between Sanofi and Sobi called XTEND-Kids phase 3 has finished, solidifying the first milestone needed for regulatory submission of ALTUVIIIO in the EU.


ALTUVIIIO is a first-in-class, high-sustained FVIII therapy. It received Orphan Drug Designation (ODD) from the European Commission (EC) in June 2019, FDA Fast Track Designation in February 2021 and FDA Breakthrough Therapy designation in May 2022. February 2023 saw it achieve FDA approval for routine prophylaxis, on-demand treatment and control of bleeding episodes and preoperative management of bleeding in adults and children.


XTEND-Kids phase 3 pivotal study evaluated the safety, efficacy and pharmacokinetics of ALTUVIIIO as a once-weekly prophylaxis in previously treated patients less than 12 years old with severe haemophilia A. Patients received a once-weekly ALTUVIIIO propylaxis for 52 weeks, which provided high-sustained FVIII levels throughout the weekly dosing interval with a median annualised bleeding rate (ABR) of 0.00 (0.00, 1.02) and an estimated mean ABR of 0.89.


Its primary endpoint was the occurrence of inhibitor development. This was met, with no FVIII inhibitors detected in 74 children, with more than 50 children experiencing at least 50 exposure days ‒ nearly a full year of treatment.


Karin Knobe MD PhD, therapeutic area head, Rare Diseases and Rare Blood Disorders at Sanofi stated: “At Sanofi, we never settle. We work alongside patients, caregivers and advocacy organisations to understand the needs of the haemophilia community and pursue first-in-class technologies to meet those needs. We strive for a future where every child with haemophilia can play without fear, travel free from a rigid treatment schedule and pursue their dreams unencumbered by worry.”


James Spargo

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