The XTEND-Kids phase 3 pivotal study evaluating the safety, efficacy and pharmacokinetics of ALTUVIIIO as once-weekly prophylaxis in previously treated patients <12 years of age with severe haemophilia A met its primary endpoint of safety, 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. The completion of XTEND-Kids represents the final milestone needed for regulatory submission in the EU.
Haemophilia A is a rare, lifelong condition in which the ability of a person’s blood to clot properly is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain, and potentially impact quality of life. The severity of haemophilia is determined by the level of clotting factor activity in a person’s blood, and there is a negative correlation between risk of bleeding and factor activity levels.
ALUTVIIIO is a first-in-class, high-sustained FVIII therapy approved by the US Food and Drug Administration (FDA) for routine prophylaxis, on-demand treatment and control of bleeding episodes, and perioperative management of bleeding in adults and children in February 2023. Granted Breakthrough Therapy designation by the FDA in May 2022 – the first FVIII therapy to receive this designation—ALTUVIIIO also received Fast Track designation in February 2021 and Orphan Drug designation in 2017. The European Commission granted Orphan Drug designation in June 2019.
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