Primary endpoint was met with no factor VIII inhibitors detected, confirming the safety profile of efanesoctocog alfa in previously treated patients under 12 years of age
The completion of XTEND-Kids represents the final milestone needed for regulatory submission in the EU
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Haemophilia A is a rare, genetic disorder in which the ability of a person's blood to clot is impaired due to a lack of factor VIII. Haemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. People with haemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages.
Despite advancements in treatment made in recent years, a large unmet medical need still exists and requires further improvement in the standard of care. Efanesoctocog alfa is a new class of factor VIII replacement therapy decoupled of von Willebrand factor, providing high sustained factor activity levels with a once-weekly prophylactic treatment regimen.
"These data confirm that efanesoctocog alfa has the potential to become a new standard of care for haemophilia A with higher protection for longer across treatment scenarios for all age groups," said
Combined with the XTEND-1 phase 3 trial, these results will provide the basis for regulatory submission in the EU. Efanesoctocog alfa was granted orphan designation by the
About XTEND-Kids
XTEND-Kids is an open-label, non-randomised interventional, single-arm study. Participants received a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. XTEND-Kids evaluates efficacy, safety and pharmacokinetics in 74 previously treated patients <12 years of age with severe haemophilia A.
About efanesoctocog alfa
Efanesoctocog alfa (formerly BIVV001) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for a significant part of the week, extending bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to potentially extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. It is approved as ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] in the US.
XTEN® is a registered trademark of
About the
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Sobi®
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,600 employees across
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https://news.cision.com/swedish-orphan-biovitrum-ab/r/positive-topline-results-from-pivotal-xtend-kids-phase-3-study-of-efanesoctocog-alfa-in-children-und,c3726289
https://mb.cision.com/Main/14266/3726289/1886349.pdf
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