European Commission grants Sobi Marketing Authorisation for ALTUVOCT for treatment of haemophilia A

The European Commission has granted Marketing Authorisation for Sobi’s ALTUVOCT (efanesoctocog alfa), for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A.

ALTUVOCT is a high-sustained factor VIII replacement therapy for all ages and any disease severity. Children, adolescents, and adults can experience non-haemophilia factor VIII activity levels (above 40%) for a significant part of the week with once-weekly prophylaxis, reaching trough levels of 15% in adults and adolescents before the next dose. This results in significantly improved protection from bleeds compared to prior factor VIII prophylaxis.

The European Commission also endorsed the European Medicines Agency (EMA) recommendation supporting ALTUVOCT’s retention of orphan drug designation, granting a 10-year market exclusivity period. The EMA recommendation noted that even considering existing treatments, once-weekly ALTUVOCT prophylaxis demonstrated a significantly lowered annual bleeding rate compared to other factor VIII products, and this constitutes a clinically relevant advantage.

“Despite advancements, haemophilia still limits the possibilities of patients’ lives and this means there is still a need for treatments that offer elevated protection. ALTUVOCT’s high-sustained factor VIII activity and once-weekly dosing schedule have the potential to significantly improve quality of life for people with haemophilia A. The trials demonstrated substantial improvements in the prevention and treatment of bleeds along with significant improvements in physical health, pain, and joint health,” said Professor Robert Klamroth, MD, PhD, Head of the Department of Internal Medicine, Vascular Medicine and Coagulation Disorders at the Vivantes Klinikum Friedrichshain, Berlin, Germany.

The granting of Marketing Authorisation is based on the results from the pivotal phase 3 studies: XTEND-1 in adults and adolescents and XTEND-Kids in children, which evaluated the efficacy and safety of ALTUVOCT in people with severe haemophilia A. These studies demonstrated that once-weekly ALTUVOCT prophylaxis (50 IU/kg) provided significant bleed protection for any age (mean ABR <1 and 80-88% of patients free from spontaneous bleeds). The outcomes also showed substantial improvement in joint health, physical health, pain and overall quality of life when comparing week 52 and baseline assessments. No factor VIII inhibitors were observed in the ALTUVOCT clinical program.

“Today’s announcement marks a major step forward in haemophilia care, offering the potential to significantly improve treatment outcomes and quality of life. For the first time, factor VIII activity levels can be sustained for a significant part of the week with simplified once-weekly dosing. We are proud to work alongside the haemophilia community, as we lead the paradigm shift towards normal haemostasis and create new possibilities together,” said Lydia Abad-Franch, MBA, Head of Research, Development, and Medical Affairs, and Chief Medical Officer at Sobi.

Haemophilia A is a rare, lifelong genetic condition in which the body does not produce enough, or makes dysfunctional, factor VIII – a protein that is essential for blood clotting. It 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. Clinical outcomes have improved over time thanks to significant advances in the treatment options available, however important unmet clinical and social needs still exist for those living with the condition.

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

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