The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Swedish Orphan Biovitrum (Sobi) and Sanofi’s efanesoctocog alfa (BIVV001) to treat haemophilia A.
The new investigational recombinant factor VIII (FVIII) therapy efanesoctocog alfa has been designed to extend protection from bleeds with once-weekly prophylactic dosing for haemophilia A patients.
It is being jointly developed and commercialised by Sobi and Sanofi.
The designation was granted based on the data obtained from the pivotal XTEND-1 Phase III study.
The findings showed that the therapy met the primary endpoint, demonstrating a clinically meaningful prevention of bleeds in severe haemophilia A patients over a period of 52 weeks.
Efanesoctocog alfa also met the key secondary endpoint and showed that it was superior in preventing bleeding events compared to prior prophylactic factor VIII replacement therapy.
Fall, headache, back pain, and arthralgia were the most common treatment-emergent adverse events observed in the XTEND-1 Phase III study.
Sobi chief medical officer and Research & Development head Anders Ullman said: “This designation supports the innovation of efanesoctocog alfa and acknowledges its potential to fulfil an unmet medical need for people living with haemophilia A.
“We are committed to transforming lives for people living with rare diseases, and this is a testament to the medical innovation that science can bring.”
The company holds final development and commercialisation rights for the drug in its territory essentially Russia, Europe, North Africa, and most Middle Eastern markets.
Sanofi holds rights in North America and all other regions in the world excluding the Sobi’s territory.