Sanofi’s ALTUVIIIO Granted Marketing Authorization in Japan for Hemophilia A Treatment

(IN BRIEF) The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO, a novel factor VIII replacement therapy used to control bleeding in patients with hemophilia A. ALTUVIIIO, also known as efanesoctocog alfa, stands out as the first and only hemophilia A treatment that provides near-normal factor activity levels with once-weekly dosing in adults and adolescents. This high-sustained factor activity significantly reduces bleeds and can be used for various clinical scenarios, including routine prophylaxis, on-demand treatment, control of bleeding episodes, and perioperative management. The approval is based on positive data from clinical trials, and ALTUVIIIO aims to improve the quality of life for individuals living with hemophilia A. It was previously approved in the United States and Taiwan.

(PRESS RELEASE) PARIS, 26-Sep-2023 — /EuropaWire/ — The Japanese Ministry of Health, Labor, and Welfare (MHLW) has granted marketing authorization for ALTUVIIIO®[Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein], a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for control of bleeding tendency in patients with hemophilia A (factor VIII deficiency). ALTUVIIIO was also recently approved by the Taiwan Food and Drug Administration for treatment of adults and children with hemophilia A on August 31, 2023.

Also referred to as efanesoctocog alfa, ALTUVIIIO is the first and only hemophilia A treatment that delivers normal to near-normal factor activity levels (over 40%) for most of the week with once-weekly dosing in adults and adolescents, and significantly reduces bleeds compared to prior factor VIII prophylaxis in adults and adolescents with severe hemophilia A. ALTUVIIIO can be used for routine prophylaxis, on-demand treatment and control of bleeding episodes, and perioperative management of bleeding. The simple recommended dose of 50 IU/kg is intended for all patients and for different clinical scenarios.

Brian Foard, Executive Vice President, Global Head of Specialty Care ad interim, Sanofi “The approval of ALTUVIIIO in Japan and Taiwan represents a major step forward for people living with hemophilia A in those countries. The high-sustained factor activity levels will enable patients and physicians to reimagine living with hemophilia. ALTUVIIIO is a testament to Sanofi’s promise to deliver first-in-class best-in-class therapies that can redefine the treatment paradigm and transform the standard of care for people around the world living with hemophilia.”

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.

The MHLW approval is based on positive data from patients with severe hemophilia A, including the pivotal XTEND-1 trial in adults and adolescents and data from the XTEND-Kids trial in children under 12 years of age. In the XTEND-1 study, once-weekly ALTUVIIIO prophylaxis (50 IU/kg) met the primary endpoint, providing significant bleed protection for people with severe hemophilia A with a mean annualized bleeding rate (ABR) of 0.71 (95% CI: 0.52 – 0.97) and a median ABR of 0.00 (Q1, Q3: 0.00, 1.04). ALTUVIIIO met the key secondary endpoint with a significant reduction of 77% in ABR versus prior factor VIII prophylaxis based on an intra-patient comparison (95% CI: 58%, 87%).

Data from XTEND-Kids, showed that children younger than 12 years of age receiving once-weekly ALTUVIIIO (50 IU/kg) for 52 weeks (n=73) experienced a mean ABR of 0.6 (95% CI: 0.4 – 0.9) and a median ABR of 0 (Q1, Q3: 0.0 – 1.0). Safety results were consistent with data from the XTEND-1 trial.

Across these studies, ALTUVIIIO has an established safety profile and there were no reports of factor VIII inhibitor development, although inhibitor formation is possible following administration of ALTUVIIIO. The most common side effects (>10%) of ALTUVIIIO are headache and arthralgia.

ALTUVIIIO was first approved in February 2023 by the US Food and Drug Administration. The FDA previously granted Breakthrough Therapy designation in May 2022 — the first factor VIII therapy to receive this designation — Fast Track designation in February 2021, and Orphan Drug designation in 2017. The European Commission granted Orphan Drug designation in June 2019, and the European Medicines Agency accepted the Marketing Authorization Application (MAA) for efanesoctocog alfa in May 2023.

About ALTUVIIIO®
ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] is a first-in-class high-sustained factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for adults and children with hemophilia A. In adults and adolescents, ALTUVIIIO has a 3- to 4-fold longer half-life relative to standard and extended half-life factor VIII products, providing high-sustained factor activity levels within normal to near-normal range for most of the week, allowing for once-weekly administration. ALTUVIIIO 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.

About the Sanofi 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, or ALTUVIIIO in the US. 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.

About Sobi®
Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable 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, Asia and Australia. In 2022, revenue amounted to SEK 18.8 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube.

About Sanofi
We are 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

Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that pandemics or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2022. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements.

Sanofi’s ALTUVIIIO Granted Marketing Authorization in Japan for Hemophilia A Treatment

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