Sanofi is Granted Breakthrough Therapy Designation for Its Haemophilia A Treatment

Sanofi has recently announced that its landmark efanesoctocog alfa treatment for haemophilia A has been granted breakthrough therapy designation by the USA’s Food and Drug Administration.

Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA and the decision was based on XTEND-1 Phase 3 study data demonstrating a clinically meaningful prevention of bleeds and superiority in prevention of bleeding episodes compared to prior prophylaxis factor treatment.

“Data from the XTEND-1 Phase 3 study are expected to be shared at an upcoming medical meeting, and those data will serve as the basis for submission to FDA mid-year 2022,” said Sanofi in a press release. “The FDA granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021. The European Commission also granted efanesoctocog alfa Orphan Drug designation in June 2019. Regulatory submission in the EU will follow availability of data from the ongoing XTEND-Kids paediatric study, expected in 2023.”

Haemophilia is a serious and debilitating condition and the acceleration of this treatment to orphan drug and now breakthrough therapy designation should bring comfort to many sufferers around the world. Making sure these treatments are as widely available as possible will no doubt be Sanofi’s next priority.