EMA authorises marketing application for efanesoctocog alfa for treatment of haemophilia A

The European Medicines Agency (EMA) has accepted and validated a marketing authorisation application from pharma company Sobi for efanesoctocog alfa, a new class of high-sustained FVIII developed for the treatment of people with haemophilia A of all age groups. The application is based on data from the pivotal XTEND-1 phase 3 study in adults and adolescents and the XTEND-Kids paediatric study in patients <12 years of age. Efanesoctocog alfa was approved by the US FDA as ALTUVIIIO earlier this year.

“Sobi aims to raise the standard of care for rare disease patients around the globe,” said Tony Hoos, MD, PhD, Head of Research & Development and Chief Medical Officer. This “announcement may represent a crucial step towards improving the lives of people with haemophilia A through a potential new treatment option. We look forward to working closely with EMA during their review of our dossier to allow timely access for the haemophilia community in Europe.”

Haemophilia A is a rare, genetic disorder in which the ability of a person’s blood to clot is impaired due to a lack of factor VIII. Haemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. People with haemophilia can experience bleeding episodes including life-threatening haemorrhages, acute and chronic pain, irreversible joint damage with disability and negative impacts on quality of life. Despite advancements in treatment made in recent years, a large unmet medical need still exists and requires further improvement in the standard of care.

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