Policy & Regulation
European Commission grants marketing authorisation for Sobi's ALTUVOCT in haemophilia A treatment
19 June 2024 -

Biopharmaceutical company Swedish Orphan Biovitrum AB (Sobi) (STO: SOBI) announced on Wednesday that the European Commission has granted Marketing Authorisation for ALTUVOCT (efanesoctocog alfa) for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A. This high-sustained factor VIII replacement therapy is suitable for all ages and disease severities, providing non-haemophilia factor VIII activity levels for significant periods with once-weekly prophylaxis.

The European Commission endorsed the European Medicines Agency's recommendation to retain ALTUVOCT's orphan drug designation, granting a 10-year market exclusivity period. The EMA highlighted the drug's significant reduction in annual bleeding rates compared to other factor VIII products as a clinically relevant advantage.

Approval is based on pivotal phase 3 studies, XTEND-1 and XTEND-Kids, which demonstrated that once-weekly ALTUVOCT prophylaxis offers substantial bleed protection across all age groups, with a mean annual bleeding rate (ABR) of less than 1 and 80-88% of patients being free from spontaneous bleeds. The studies also showed improvements in joint health, physical health, pain, and overall quality of life. No factor VIII inhibitors were observed during the trials.

Haemophilia A, a rare genetic condition affecting blood clotting, occurs in about one in 5,000 male births annually. Current treatments have improved clinical outcomes, but significant unmet needs remain, particularly in second-line treatments where there are no approved options post-first-line standard of care.

ALTUVOCT was first approved in the US in February 2023 by the FDA, which previously granted it Breakthrough Therapy designation in May 2022, Fast Track designation in February 2021 and Orphan Drug designation in 2017. The XTEND-1 study involved 159 previously treated patients aged 12 and above, while the XTEND-Kids study included 74 patients under 12 years of age, both demonstrating the drug's efficacy, safety, and pharmacokinetics.

An Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein, ALTUVOCT builds on established Fc fusion technology and includes innovative von Willebrand factor and XTEN polypeptides to extend its circulation time. This allows it to break through the von Willebrand factor ceiling, which limits the half-life of current factor VIII therapies.

The company collaborates with Sanofi (EURONEXT: SAN) (NASDAQ: SNY) on the development and commercialisation of ALTUVOCT, with Sobi holding rights in Europe, North Africa, Russia and the Middle East, while Sanofi holds rights in North America and other regions.

Sobi focuses on transforming the lives of people with rare diseases, with a significant presence in haematology, immunology and specialty care.