Revolutionary Drug for Hemophilia A Gets Regulatory Approval!

Sobi has announced exciting news – the European Medicines Agency (EMA) has officially accepted and validated their application for the revolutionary treatment efanesoctocog alfa for people with haemophilia A, of all ages! This milestone follows the successful completion of phase 3 XTEND-1 and XTEND-Kids studies in adults and adolescents, as well as paediatrics under the age of 12.

Efanesoctocog alfa was approved by the US Food and Drug Administration (FDA) as ALTUVIIIO earlier this year. This revolutionary, high-sustained FVIII treatment provides hope for those battling haemophilia A.

Today, Sobi™ takes a groundbreaking step towards improving the standard of care for the millions of people around the world suffering from rare diseases. Tony Hoos, MD, PhD, Head of Research & Development and Chief Medical Officer, is confident this announcement will result in a potential new treatment option for those living with haemophilia A. Having embarked on the journey with the European Medicines Agency, Sobi is determined to ensure the haemophilia community has timely access to this improved care.

Haemophilia A is a rare hereditary disorder far more common in males than females, with around 1 in 5,000 male births affected worldwide. But despite its rarity, it can have serious consequences – from life-threatening haemorrhages to permanent joint damage and disability. Unchecked, it can drastically reduce quality of life.

Thankfully, thanks to major advancements in care over the last few years, the medical field can now better treat those living with Haemophilia A, but unfortunately the unmet medical needs remain; there is still room for improvement.

About XTEND-1

The XTEND-1 study provided some valuable insights into the therapeutic potential of efanesoctocog alfa in the treatment of severe haemophilia A. The open-label, non-randomized interventional trial was conducted with 159 previously treated participants aged 12 and above.

The participants were assigned to either weekly prophylactic dose or a 26-week on-demand mode of administration, followed by a switch to prophylactic treatment for the next 26 weeks. The goal of the trial was to evaluate the efficacy, safety and pharmacokinetics of efanesoctocog alfa in this widespread condition.

About XTEND-Kids

XTEND-Kids is an innovative open-label, non-randomised interventional study where 74 children with severe haemophilia A, aged <12 years, received efanesoctocog alfa as a prophylactic weekly dose for a period of 52 weeks. The clinical trial seeks to assess the efficacy, safety, and pharmacokinetics of the drug in children.

About efanesoctocog alfa

Efanesoctocog alfa, the first of its kind, holds the promise of providing near-normal factor activity for people with haemophilia A. Its unique Fc fusion technology, combined with a region of von Willebrand factor and XTEN® polypeptides, extends its time in circulation, allowing for a once-weekly dose for improved bleed protection.

This breakthrough was made possible by surpassing the existing von Willebrand factor ceiling. After successfully receiving approval from the US in February 2023, Efanesoctocog alfa is ready to revolutionize the treatment for haemophilia A – a sign of hope for those with the condition.

About the Sanofi and Sobi collaboration

In an unprecedented move, Sobi and Sanofi have joined forces to work on the development and commercialisation of Alprolix® and Elocta®/Eloctate®, as well as efanesoctocog alfa or ALTUVIIIO in the US.

Sobi holds the final rights to development and commercialisation in its territory, which includes Europe, North Africa, Russia, and most Middle Eastern markets, whilst Sanofi has the same rights in North America and all other regions of the world, excluding the Sobi’s territory. This remarkable agreement is a remarkable move that has the potential to change the face of healthcare.


Sanofi is an innovative healthcare company committed to transforming the practice of medicine worldwide. Our dedicated team of over 100 countries is on a mission to make the impossible possible, striving to unlock and deliver life-changing treatments and life-saving vaccine protection to millions of people across the globe.

We believe firmly in sustainable and socially responsible practices, and participate in exchanges on the EURONEXT: SAN and NASDAQ: SNY to make this all happen. Join us in pursuing our powerful mission to extend and improve the lives of patients everywhere.


At Sobi, we are dedicated to helping transform the lives of those living with rare and debilitating diseases. Our mission is to make sure everyone has access to innovative and reliable medicines.

As a biopharmaceutical company with a global presence in Europe, North America, the Middle East, Asia and Australia, we employ over 1,600 people and in 2022, our revenue exceeded 18.8 billion SEK. We specialise in haematology, immunology and specialty care to make sure our patients benefit from the best possible treatments.

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