FDA Approves Sanofi’s Once-Weekly Factor VIII Therapy for Hemophilia A

Rare Daily Staff

The U.S. Food and Drug Administration has approved Sanofi’s Altuviiio, a once-weekly factor VIII replacement therapy for people with the bleeding disorder hemophilia A.

Altuviiio is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as the management of surgery for adults and children with hemophilia A.

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.

Altuviiio is a novel von Willebrand factor (VWF) independent recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for adults and children with hemophilia A. Altuviiio has a 3 to 4 fold longer half-life relative to standard and extended half-life factor VIII products. It is the first factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on earlier generation factor VIII therapies. Altuviiio builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to extend its time in circulation.

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, Fast Track, and Orphan Drug designations.

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

“The high sustained factor activity levels that can be achieved with Altuviiio have the potential to change the hemophilia landscape,” Paul Hudson, CEO of Sanofi. “For the first time, with a once-weekly dose, powerful bleed protection is a reality for patients.”

The FDA approval is based on data from the pivotal XTEND-1 phase 3 study recently published in The New England Journal of Medicine. Once-weekly Altuviiio prophylaxis met the primary endpoint, providing significant bleed protection for people with severe hemophilia A with a mean annualized bleeding rate (ABR) of 0.70 and a median ABR of 0.0. Altuviiio met the key secondary endpoint with a significant reduction of 77 percent in ABR versus prior factor prophylaxis based on an intra-patient comparison.

To ensure that patients have access to the improved bleed protection provided by Altuviiio, Sanofi will price Altuviiio at parity to the annual cost of treating a prophylaxis patient on Eloctate. Sanofi will also provide comprehensive patient support services and resources online. In the United States, Altuviiio is expected to be commercially available in April.

Sobi and Sanofi collaborate on the development and commercialization of Alprolix and Elocta/Eloctate. The companies also collaborate on the development and commercialization of efanesoctocog alfa or Altuviiio in the United States. Sobi has final development and commercialization rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialization rights in North America and all other regions in the world excluding the Sobi territory.

Photo: Paul Hudson, CEO of Sanofi