logo
  

Sanofi: FDA Grants Efanesoctocog Alfa Breakthrough Therapy Designation For Hemophilia A

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.

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
Hospitality company Hilton has teamed with connected fitness platform Peloton Interactive, Inc. to feature Peloton Bikes across its entire U.S. hotel portfolio. In an industry-first partnership, Hilton's nearly all 5,400 U.S. Hilton-branded hotels will feature at least one Peloton Bike in every fitness center by the end of the year. Australia's central bank raised its benchmark rate by a smaller-than-expected quarter point on Tuesday, after lifting rates by 50 basis points in each of the last four meetings. The policy board of the RBA, headed by Governor Philip Lowe, decided to lift the cash rate target by 25 basis points to 2.60 percent from 2.35 percent. This was the highest rate since July 2013. Unemployment data from Spain and producer prices from the euro area are due on Tuesday, headlining a light day for the European economic news. At 2.00 am ET, the National Institute of Statistics is scheduled to issue Romania's producer prices for August.
Follow RTT