French drug major Sanofi (SNYNF,SNY) announced Wednesday that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A.
Efanesoctocog alfa is the first factor VIII therapy to be awarded Breakthrough Therapy designation by the FDA.
Hemophilia A is a rare and life-threatening bleeding disorder.
Efanesoctocog alfa is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen.
The FDA decision is based on data from the pivotal XTEND-1 Phase 3 study. Topline results from the trial demonstrate efanesoctocog alfa met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A over a 52-week period.
Breakthrough Therapy designation is designed to expedite the development and review of drugs in the US that target serious or life-threatening conditions.
Sanofi and Swedish biopharmaceutical company Sobi collaborate on the development and commercialization of efanesoctocog alfa, which is currently under clinical investigation. Its safety and efficacy have not been evaluated by any regulatory authority.
Sanofi has final development and commercialization rights in North America and all other regions in the world excluding the Sobi territory, essentially Europe, North Africa, Russia and most Middle Eastern markets.
John Reed, Global Head of Research and Development at Sanofi, said, “The Breakthrough Therapy designation highlights efanesoctocog alfa’s potential to transform treatment for people with hemophilia A by providing higher protection for longer duration. This potential new class of factor VIII therapy represents how we are boldlyadvancing science to address unmet needs for the hemophilia community.”
The company expects to share data from the XTEND-1 Phase 3 study at an upcoming medical meeting, and those data will serve as the basis for submission to FDA mid-year 2022.
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 pediatric study, expected in 2023.
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