Full Press Release Details
FDA grants priority review to efanesoctocog alfa for people with hemophilia A
August 30, 2022 The U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of hemophilia A, a rare and
life-threatening bleeding disorder. The target action date for the FDA decision is February 28, 2023. Sanofi and Sobi collaborate on the development and commercialization of
Professor and Director of Pediatric Hemophilia and Coagulation Disorders Program, University of Michigan
Factor therapy remains a cornerstone of hemophilia treatment, but innovation has been needed in this area to address
challenges related to bleed protection and cumbersome treatment regimens. If approved, efanesoctocog alfa can deliver close to normal factor activity levels for the majority of the week, potentially offering a new tier of protection. Such
therapeutic benefits would represent important advances in unmet medical needs for people with hemophilia A and may transform the prophylactic treatment landscape.
The BLA is supported by data from the pivotal XTEND-1 Phase 3 study. Results were recently presented at the 30th International Society of Thrombosis and Haemostasis Congress. The data
demonstrate a clinically meaningful prevention of bleeds and superiority to prior factor prophylaxis based on an intra-patient comparison. Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected. The most
common treatment-emergent adverse events (>5% of participants 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 pivotal XTEND-1 Phase 3 study demonstrate efanesoctocog
alfa s ability to reduce annualized bleeding rates, which supports its potential as a therapy with best-in-disease efficacy. We look forward to working closely with
the FDA during the review process as we aim to bring this novel therapy to the hemophilia A community.
The FDA grants priority review to
therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. Efanesoctocog alfa received Breakthrough
Therapy designation from the FDA in May 2022 and it is the first factor VIII therapy to receive 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 availability of data from the ongoing XTEND-Kids pediatric study, with both events expected 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-label, non-randomized interventional study assessing the safety, efficacy and pharmacokinetics of once-weekly efanesoctocog alfa in people 12 years of age or older
(n=159) with severe hemophilia A who were previously treated with factor VIII replacement therapy. The study consisted of two parallel treatment arms the
prophylaxis Arm A (n=133), in which patients who had received prior factor VIII prophylaxis were treated with once-weekly intravenous efanesoctocog alfa prophylaxis (50 IU/kg) for 52
weeks, and the on-demand Arm B (n=26), in which patients who had received prior on-demand factor VIII therapy began 26 weeks of
on-demand efanesoctocog alfa (50 IU/kg), then switched to once-weekly prophylaxis (50 IU/kg) for an additional 26 weeks.
The primary efficacy endpoint was the annualized bleeding rate (ABR) in Arm A, and the key secondary endpoint was an intra-patient comparison
of ABR during the efanesoctocog alfa weekly prophylaxis treatment period versus the prior factor VIII prophylaxis ABR for participants in Arm A who had participated in a previous observational study (Study
Hemophilia A is a rare, genetic disorder in which the ability of a person s blood to clot is impaired due to a lack of factor VIII. Hemophilia A
occurs in about one in 5,000 male births annually, and more rarely in females. People with hemophilia can experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. Factor replacement therapy
remains a cornerstone of care and can be used across multiple treatment scenarios.
About efanesoctocog alfa (BIVV001)
Efanesoctocog alfa is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with
once-weekly prophylactic dosing for people with hemophilia A. It builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to extend its
time in circulation. It is the first investigational factor VIII therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. Efanesoctocog alfa is currently
under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
and Sobi collaboration
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. 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.
Sobi is a specialized international biopharmaceutical company transforming the lives of people with
rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,600 employees across Europe, North America, the Middle East and Asia. In 2021, revenue amounted
to SEK 15.5 billion. Sobi s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and
an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the
impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY.
Sandrine Guendoul | + 33 6 25 09 14 25
Bain | + 1 617 834 6026 | sally.bain@sanofi.com
Kate Conway | + 1 508 364 4931 | kate.conway@sanofi.com
Eva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.com
Arnaud Del pine | + 33 6 73 69 36 93 |
Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.com
Felix Lauscher | + 1 908 612 7239 |
Nanduri | + 1 617 764 6418 | priya.nanduri@sanofi.com
Nathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com
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