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FDA approves once-weekly ALTUVIIIO™, a new class of factor VIII therapy for hemophilia A that offers

FDA approves once-weekly ALTUVIIIO™, a new class of factor VIII therapy for hemophilia A that offers significant bleed protection

DA approvesonce-weekly ALTUVIIIO™, anew classof factor VIII therapyforhemophilia A that offers significant bleed protection

Paris and Stockholm – February 23, 2023 – The U.S. Food and Drug Administration (FDA) has approved ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia A. 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, and significantly reduces bleeds compared to prior factor VIII prophylaxis.

Paul Hudson CEO, Sanofi "Today’s approval of ALTUVIIIO allows patients and physicians to reimagine living with hemophilia. The high sustained factor activity levels that can be achieved with ALTUVIIIO have the potential to change the hemophilia landscape. For the first time, with a once-weekly dose, powerful bleed protection is a reality for patients. Significant shifts in treatment paradigms that improve people’s lives, like ALTUVIIIO, are what we have committed to delivering at Sanofi.”

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

“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.

ALTUVIIIO helps elevate expectations for hemophilia A by providing protection for longer

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 (95% CI: 0.5-1.0) and a median ABR of 0.0 (Q1, Q3: 0.0, 1.0). ALTUVIIIO met the key secondary endpoint with a significant reduction of 77% in ABR versus prior factor prophylaxis based on an intra-patient comparison (95% CI:58%-87%).

Additional data showed prevention of joint bleeds with a median annualized joint bleeding rate of 0 (Q1, Q3: 0.0, 1.0). Treatment with ALTUVIIIO provided 100% resolution of target joints, which are joints that have recurrent bleeds (e.g., knee, ankle, or elbow). ALTUVIIIO provided mean factor VIII activity greater than 40% for most of the week and greater than 10% at Day 7; these levels were associated with a low bleed risk. In the study, ALTUVIIIO was well-tolerated and inhibitor development to factor VIII was not detected, although is possible following administration of ALTUVIIIO.

Additionally, interim data from XTEND-Kids showed that children younger than 12 years of age receiving once-weekly ALTUVIIIO for 26 weeks (n=23) experienced a mean ABR of 0.5 (95% CI: 0.2-1.3) and a median ABR of 0 (Q1, Q3: 0.0, 1.3). Safety results were consistent with data from the XTEND-1 trial. The full results from XTEND-Kids will be presented at a future medical meeting.

Across the 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 is indicated 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.

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®[Antihemophilic Factor (Recombinant), Fc Fusion Protein]. Sanofi will also provide comprehensive patient support services and resources online and at 1.855.

MyALTUVIIIO (855.692.5888). In the U.S., ALTUVIIIO is expected to be commercially available in April.

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