The European Commission has granted marketing authorisation to Sobi’s Altuvoct (efanesoctocog alfa) for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A.
The Commission also upheld the European Medicines Agency (EMA) recommendation for Altuvoct to retain its orphan drug designation. It grants a ten-year market exclusivity to the therapy, compared with the eight-year exclusivity that drugs usually receive as part of new drug authorisations.
The EU approval builds on the previous US approval for Altuvoct. In February, the US Food and Drug Administration (FDA) first approved the haemophilia A therapy for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery). The therapy is marketed as Altuviiio in the US.
Haemophilia A is a rare genetic disorder in which a person’s blood clotting ability is impaired because of a lack or total absence of a clotting factor, factor VIII. Altuvoct acts as a high-sustained recombinant factor VIII replacement therapy, which is administered once a week.
Haemophilia A is very rare and affects less than 1 in 10,000 people, as per the Canadian Hemophilia Society. It is also an X-chromosome-linked disorder that is very rare in females and disproportionately almost always affects males.
The European Commission approval was based on the data for adults and adolescents from the Phase III XTEND-1 (NCT04161495) [RM1] and paediatric patient trial data, from patients aged under 12 years, from XTEND-Kids (NCT04759131) trials.
Altuvoct is co-developed and commercialised by Sobi in partnership with Sanofi. Whilst Sobi has final development and commercialisation rights in Europe, North Africa, Russia and most Middle Eastern markets, Sanofi retains these rights in North America and all other regions in the world excluding the Sobi territory. The therapy is expected to generate $2.74bn in sales in 2030, as per GlobalData analysis.
GlobalData is the parent company of Pharmaceutical Technology.
Another high-profile treatment for haemophilia A is gene therapy, but its high cost can be a deterrent to adoption. In June 2023, the US FDA approved BioMarin’s gene therapy Roctavian (valoctocogene roxaparvovec-rvox) as a treatment for adults with haemophilia A. However, its eye-watering $2.9m price tag defied the expectations of the experts in the field, who predicted a price of approximately $1.95m.
