Sanofi gets USFDA nod for hemophilia A treatment ALTUVIIIO

Hemophilia A is a rare, lifelong condition in which the ability of a person’s blood to clot properly is impaired, leading to excessive bleeds and spontaneous bleeds into joints that can result in joint damage and chronic pain, and potentially impact quality of life. The severity of hemophilia is determined by the level of clotting factor activity in a person’s blood, and there is a negative correlation between risk of bleeding and factor activity levels.

Lynn Malec, MD, Medical Director of Comprehensive Center for Bleeding Disorders and Associate Investigator at The Versiti Blood Research Institute, and Associate Professor of Medicine and Pediatrics at The Medical College of Wisconsin said, “This approval marks an important clinical advancement for the hemophilia community because we have an option that can achieve higher levels of factor activity with a single simplified weekly dose. By maintaining high levels of factor activity throughout the week, patients can be confident in the bleed protection ALTUVIIIO offers.”

This is the first approval of ALTUVIIIO. The FDA evaluated the application under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. The FDA previously granted ALTUVIIIO Breakthrough Therapy designation in May 2022 – the first factor VIII therapy to receive this recognition – Fast Track designation in February 2021, and Orphan Drug designation in August 2017.

Regulatory submission in the EU is anticipated in the second half of 2023. The European Commission granted Orphan Drug designation in June 2019.