In a significant development for hemophilia A treatment, Sobi has announced that the European Commission has granted Marketing Authorization for ALTUVOCT™ (efanesoctocog alfa). This innovative therapy is designed to manage and prevent bleeding episodes in individuals with hemophilia A, offering a high-sustained factor VIII replacement suitable for patients of all ages and severities. With a once-weekly prophylaxis regimen, patients can maintain non-hemophilia factor VIII activity levels for a substantial portion of the week, thereby enhancing protection against bleeds.
The European Commission's decision is in line with the European Medicines Agency's (EMA) recommendation to uphold ALTUVOCT's orphan drug designation, which ensures a 10-year market exclusivity. The EMA has highlighted that ALTUVOCT's once-weekly prophylaxis significantly reduces annual bleeding rates compared to other factor VIII products, providing a clinically meaningful advantage.
Professor Robert Klamroth, a leading expert in coagulation disorders, has emphasized the transformative potential of ALTUVOCT. He noted its capacity to improve quality of life through enhanced bleed prevention and better physical and joint health outcomes. The therapy was evaluated in pivotal phase 3 studies, XTEND-1 and XTEND-Kids, which confirmed its efficacy and safety across different age groups.
Clinical Trial Insights
The XTEND-1 and XTEND-Kids studies demonstrated that once-weekly ALTUVOCT prophylaxis significantly protects against bleeds, with a mean annual bleeding rate of less than one and 80-88% of patients remaining free from spontaneous bleeds. The trials also reported notable improvements in joint and physical health, pain management, and overall quality of life without the development of factor VIII inhibitors.
This authorization marks a pivotal advancement in hemophilia care, offering a simplified dosing schedule that sustains factor VIII activity levels throughout the week. Lydia Abad-Franch, Head of Research and Medical Affairs at Sobi, expressed pride in collaborating with the hemophilia community to drive this paradigm shift towards normal hemostasis.
Understanding Hemophilia A
Hemophilia A is a rare genetic disorder characterized by insufficient or dysfunctional production of factor VIII, crucial for blood clotting. Predominantly affecting males, it occurs in approximately one in 5,000 male births annually. The condition can lead to severe bleeding episodes, causing pain, joint damage, and potentially life-threatening hemorrhages. Despite advancements in treatment, significant unmet needs remain for those living with hemophilia A.
ALTUVOCT received its initial approval from the US Food and Drug Administration (FDA) in February 2023. It was the first factor VIII therapy to earn Breakthrough Therapy designation in May 2022, following Fast Track and Orphan Drug designations in previous years.
As of March 2025, the approval of ALTUVOCT by the European Commission represents a significant milestone in the treatment of hemophilia A. This development not only underscores the potential of innovative therapies to transform patient care but also highlights the ongoing commitment to addressing the unmet needs of those living with this challenging condition. The once-weekly dosing regimen offers a more convenient and effective approach to managing hemophilia A, potentially setting a new standard in treatment protocols.