Sobi® has received approval from the European Commission for ALTUVOCT™ (efanesoctocog alfa), a replacement therapy designed for treating and preventing bleeds, as well as for perioperative prophylaxis in individuals with hemophilia A. This therapy is suitable across all age groups and severity levels of the condition. By providing once-weekly prophylaxis, ALTUVOCT enables children, adolescents, and adults to achieve non-hemophilic factor VIII activity levels for most of the week, with trough levels of 15% in adults and adolescents before the next dose. This enhancement offers significantly better protection against bleeds compared to previous factor VIII therapies.

The European Medicines Agency's (EMA) recommendation to maintain ALTUVOCT's orphan drug designation has been endorsed by the European Commission, securing a 10-year market exclusivity period. This designation is based on the observed significant reduction in annual bleeding rates with once-weekly ALTUVOCT prophylaxis, highlighting its clinical benefits over existing treatments.

"Despite medical advancements, hemophilia still imposes significant limitations on patients' lives, underscoring the ongoing need for enhanced treatments. ALTUVOCT’s sustained factor VIII activity and the convenience of a once-weekly dose have the potential to greatly improve the quality of life for individuals with hemophilia A. Clinical trials have demonstrated substantial improvements in bleed prevention, physical health, pain management, and joint health," said Professor Robert Klamroth, MD, PhD, Head of the Department of Internal Medicine, Vascular Medicine, and Coagulation Disorders at Vivantes Klinikum Friedrichshain, Berlin, Germany.

The approval is supported by results from pivotal phase 3 studies: XTEND-1 in adults and adolescents, and XTEND-Kids in children, which assessed ALTUVOCT's efficacy and safety in severe hemophilia A patients. These studies showed that once-weekly ALTUVOCT (50 IU/kg) significantly protected against bleeds across all age groups (mean ABR <1 and 80-88% of patients experiencing no spontaneous bleeds). Outcomes also demonstrated significant improvements in joint health, physical health, pain levels, and overall quality of life at week 52 compared to baseline, with no factor VIII inhibitors detected in the ALTUVOCT clinical trials.

"Today's approval represents a significant advancement in the treatment of hemophilia, with the potential to improve both outcomes and quality of life. This is the first time that factor VIII activity levels can be sustained throughout much of the week with a straightforward once-weekly dosing regimen. We are excited to collaborate with the hemophilia community to lead this shift towards more effective hemostatic management," stated Lydia Abad-Franch, MBA, Head of Research, Development, and Medical Affairs, and Chief Medical Officer at Sobi.

Hemophilia A is a rare, lifelong genetic disorder characterized by the inadequate or dysfunctional production of factor VIII, a vital protein for blood clotting. It affects approximately one in 5,000 male births annually, and less commonly females. Individuals with hemophilia can experience bleeding episodes that may cause pain, irreversible joint damage, and life-threatening hemorrhages. Although there have been significant improvements in treatment options, substantial unmet clinical and social needs remain.

ALTUVOCT was initially approved in the US in February 2023 by the FDA, which had previously granted Breakthrough Therapy designation to efanesoctocog alfa in May 2022—the first factor VIII therapy to receive this designation—Fast Track designation in February 2021, and Orphan Drug designation in 2017.

XTEND-1 was an open-label, non-randomized interventional study with two groups: one receiving weekly prophylactic doses of efanesoctocog alfa for 52 weeks, and the other initially receiving on-demand doses for 26 weeks before switching to weekly prophylaxis for another 26 weeks. The study assessed the efficacy, safety, and pharmacokinetics in 159 previously treated patients aged 12 years and older with severe hemophilia A.

XTEND-Kids was an open-label, non-randomized single-arm study where participants received weekly prophylactic doses of efanesoctocog alfa for 52 weeks. The study evaluated the efficacy, safety, and pharmacokinetics in 74 previously treated patients under 12 years old with severe hemophilia A.

ALTUVOCT (efanesoctocog alfa) [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is the first high-sustained FVIII replacement therapy capable of achieving near-normal factor activity levels for much of the week, thereby enhancing bleed protection with a weekly dose for individuals with hemophilia A. The therapy leverages established Fc fusion technology by incorporating a region of von Willebrand factor and XTEN® polypeptides to extend its circulation time. It is the only therapy demonstrated to surpass the von Willebrand factor ceiling, which limits the half-life of current factor VIII therapies. The European Commission granted Orphan Drug status in June 2019. ALTUVOCT™ is marketed by Sobi in Europe, and as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the United States, Japan, and Taiwan.

 

Source: prnewswire.com

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