Sanofi has announced that the XTEND-Kids Phase III clinical trial of ALTUVIIIO in previously treated patients younger than 12 years of age with severe haemophilia A met its primary safety endpoint.
The XTEND-Kids pivotal trial has been designed for assessing ALTUVIIIO’s safety, efficacy, and pharmacokinetics when given as a once-weekly prophylaxis in previously treated patients of this age group with severe haemophilia.
No FVIII inhibitors were detected in 74 children in this trial and over 50 children experienced at least 50 exposure days, nearly a complete year of treatment.
The trial completion represents the final milestone that is needed for regulatory submission in the EU.
Sanofi Rare Diseases and Rare Blood Disorders therapeutic area head Karin Knobe said: “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.”
In the open-label, non-randomised, interventional XTEND-Kids study, patients were given ALTUVIIIO prophylaxis (50 IU/kg) once-a-week for 52 weeks.
The occurrence of inhibitor development (baseline to 52 weeks) was the trial’s primary endpoint and no inhibitors were identified.
Last month, the first-in-class, high-sustained FVIII therapy ALUTVIIIO received approval from the US Food and Drug Administration (FDA) for routine prophylaxis, on-demand treatment, and control of bleeding episodes.
It was also approved for the perioperative management of bleeding in adults and children.
In January this year, Sanofi enlisted Thread as a global decentrialised clinical trial provider under a five-year collaboration deal.