FDA grants priority review to efanesoctocog alfa for people with hemophilia A
- The FDA decision date for efanesoctocog alfa, an investigational factor VIII therapy, is set for February 28, 2023
- Priority review based on pivotal data from the XTEND-1 Phase 3 study
- Efanesoctocog alfa delivers high sustained factor activity levels in the normal to near-normal range for the majority of the week with once weekly prophylaxis dosing, providing higher protection for longer
TheU.S. Food and Drug Administration( FDA) has accepted for precedence review the Biologics License operation( BLA) for efanesoctocog alfa( BIVV001) for the treatment of hemophilia A, a rare and life- hanging bleeding complaint. The target action date for the FDA decision is February 28, 2023. Sanofi and Sobi ® unite on the development and commercialization of efanesoctocog alfa.
Steve Pipe, MD
Professor and Director of Pediatric Hemophilia and Coagulation diseases Program, University of Michigan
“ Factor remedy remains a foundation of hemophilia treatment, but invention has been demanded in this area to address challenges related to bleed protection and clumsy treatmentregimens.However, efanesoctocog alfa can deliver near to normal factor exertion situations for the maturity of the week, potentially offering a new league of protection, If approved. similar remedial benefits would represent important advances in unmet medical requirements for people with hemophilia A and may transfigure the precautionary treatment geography. ”
The BLA is supported by data from the vital XTEND- 1 Phase 3 study. Results were lately presented at the 30th International Society of Thrombosis and Haemostasis Congress. The data demonstrate a clinically meaningful forestallment of bleeds and superiority to previous factor prophylaxis grounded on anintra-patient comparison. Efanesoctocog alfa was well- permitted, and asset development to factor VIII wasn’t detected. The most common treatment- imperative adverse events(> 5 of actors overall) were headache, arthralgia, fall, and back pain.
Dietmar Berger, MD, PhD
Global Head of Development and Chief Medical Officer at Sanofi
“ The results from the vital XTEND- 1 Phase 3 study demonstrate efanesoctocog alfa’s capability to reduce annualized bleeding rates, which supports its eventuality as a remedy with stylish- in- complaint efficacity. We look forward to working nearly with the FDA during the review process as we aim to bring this novel remedy to the hemophilia A community. ”
The FDA subventions precedence review to curatives that have the eventuality to give significant advancements in the treatment, opinion, or forestallment of serious conditions. Efanesoctocog alfa entered Advance remedy designation from the FDA in May 2022 and it’s the first factor VIII remedy to admit this recognition. The FDA also granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.
Regulatory submission in the EU will follow vacuity of data from the ongoing XTEND- Kids pediatric study, with both events anticipated in 2023. The European Commission granted efanesoctocog alfa Orphan Drug designation in June 2019.
About Phase 3 XTEND- 1 Study( NCT04161495)
The Phase 3 XTEND- 1 study( NCT04161495) was an open- marker,non-randomized interventional study assessing the safety, efficacity and pharmacokinetics of formerly-daily efanesoctocog alfa in people 12 times of age or aged( n = 159) with severe hemophilia A who were preliminarily treated with factor VIII relief remedy. The study comported of two resemblant treatment arms the prophylaxis Arm A( n = 133), in which cases who had entered previous factor VIII prophylaxis were treated with formerly-daily intravenous efanesoctocog alfa prophylaxis( 50 IU/ kg) for 52 weeks, and the on- demand Arm B( n = 26), in which cases who had entered previous on- demand factor VIII remedy began 26 weeks of on- demand efanesoctocog alfa( 50 IU/ kg), also switched to formerly-daily prophylaxis( 50 IU/ kg) for an fresh 26 weeks.
About hemophilia A
Hemophilia A is a rare, inheritable complaint in which the capability of a person’s blood to clot is bloodied due to a lack of factor VIII. Hemophilia A occurs in about one in,000 manly births annually, and more infrequently in ladies. People with hemophilia can witness bleeding occurrences that can beget pain, unrecoverable common damage and life- hanging hemorrhages. Factor relief remedy remains a foundation of care and can be used across multiple treatment scripts.
About efanesoctocog alfa( BIVV001)
Efanesoctocog alfa is a new and investigational recombinant factor VIII remedy that’s designed to extend protection from bleeds with formerly-daily precautionary dosing for people with hemophilia A. It builds on the innovative Fc emulsion technology by adding a region of von Willebrand factor and XTEN ® polypeptides to extend its time in rotation. It’s the first investigational factor VIII remedy that has been shown to break through the von Willebrand factor ceiling, which imposes a half- life limitation on current factor VIII curatives. Efanesoctocog alfa is presently under clinical disquisition and its safety and efficacity haven’t been estimated by any nonsupervisory authority.
About the Sanofi and Sobi collaboration
Sobi and Sanofi unite on the development and commercialization of Alprolix ® and Elocta ®/ Eloctate ®. The companies also unite on the development and commercialization of efanesoctocog alfa. Sobi has final development and commercialization rights in the Sobi home( basically Europe, North Africa, Russia and most Middle Eastern requests). Sanofi has final development and commercialization rights in North America and all other regions in the world banning the Sobi home.
About Sobi ®
Sobi is a technical transnational biopharmaceutical company transubstantiating the lives of people with rare conditions. furnishing sustainable access to innovative drugs in the areas of haematology, immunology and specialty care, Sobi has roughly,600 workers across Europe, North America, the Middle East and Asia. In 2021, profit amounted to SEK15.5 billion. Sobi’s share( STOSOBI) is listed on Nasdaq Stockholm.
We’re an innovative global healthcare company, driven by one purpose we chase the cautions of wisdom to ameliorate people’s lives. Our platoon, across some 100 countries, is devoted to transubstantiating the practice of drug by working to turn the insolvable into the possible. We give potentially life- changing treatment options and life- saving vaccine protection to millions of people encyclopedically, while putting sustainability and social responsibility at the center of our intentions.