Sobi announced the publication of full results from the phase III XTEND-Kids study in children younger than 12 years old with severe haemophilia A in The New England Journal of Medicine

Sobi announced the publication of full results from the Phase III XTEND-Kids study in The New England Journal of Medicine (NEJM) confirming the safety and efficacy profile of Altuvoct (efanesoctocog alfa), in children younger than 12 years old with severe haemophilia A.

The study demonstrated that fixed-dose once-weekly prophylaxis with Altuvoct, a new class of high-sustained factor VIII therapy, provided highly effective bleed protection in children.

“Children represent a population for which it has been historically difficult to achieve effective bleed prevention,” said Lynn Malec, MD, Medical Director of Comprehensive Center for Bleeding Disorders and Associate Investigator at The Versiti Blood Research Institute, and Associate Professor of Medicine and Paediatrics at The Medical College of Wisconsin. “The clearance of administered factor concentrates is greater in children than we see in adults, which leads to burdensome treatment regimens involving multiple injections per week. By providing high-sustained factor VIII activity levels consistently early in life with once-weekly dosing, Altuvoct has the potential to improve reported outcomes for children living with haemophilia.”

Published in NEJM, the pivotal XTEND-Kids study shows Altuvoct met primary and key secondary endpoints, which included occurrence of factor VIII inhibitors and annualised bleed rates (ABRs). Results revealed no development of factor VIII inhibitors and sustained protection from bleeding events with once-weekly dosing. These findings support the value of sustaining high factor levels with Altuvoct in children with haemophilia A.

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