Sanofi Strengthens Hemophilia Leadership with New Data on ALTUVIIIO and Fitusiran

Sanofi is set to present significant new data from its hemophilia treatment portfolio at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH), scheduled for June 22-26, 2024, in Bangkok, Thailand. This initiative highlights the company's dedication to developing innovative treatments for rare blood disorders and underscores their leadership in the hemophilia arena.

One of the key highlights is the interim results from the XTEND-ed phase 3 study for ALTUVIIIO. These results indicate that once-weekly dosing of ALTUVIIIO continues to provide effective bleed protection in both adults and children with severe hemophilia A over a two-year period. This study reinforces ALTUVIIIO’s safety and efficacy, showing improvements or maintenance in joint health with no detection of factor VIII inhibitors. Important presentations include data on the annualized bleed rate (ABR) and joint health outcomes, demonstrating significant benefits for patients with severe hemophilia A.

Moreover, ALTUVIIIO has shown promising results in perioperative management for hemophilia A patients. Data from various clinical studies, including XTEND-1, XTEND-Kids, and XTEND-ed, reveal that ALTUVIIIO ensured successful hemostasis during major surgeries for both adults and children, further highlighting its robustness as a treatment option.

In addition to ALTUVIIIO, Sanofi will showcase new data on fitusiran from the ATLAS phase 3 clinical development program. Fitusiran, an antithrombin-lowering therapy, aims to offer consistent bleed protection for individuals with hemophilia A or B, with or without inhibitors. Clinical outcomes from the ATLAS program suggest that fitusiran provides effective prophylaxis, enabling patients to manage bleeds during major surgeries safely. The perioperative data indicate that fitusiran can effectively manage bleeds irrespective of inhibitor status.

The favorable safety profile of fitusiran was also underlined, showcasing a reduced incidence of thrombotic events and liver enzyme elevations. The revised antithrombin-based dosing regimen (AT-DR) was crucial in achieving these outcomes, ensuring the mitigation of thrombotic risks and liver-related complications.

Regulatory submissions for fitusiran have reached significant milestones, with the US Food and Drug Administration (FDA) set to review the New Drug Application by March 28, 2025. This follows an earlier Breakthrough Therapy Designation granted by the FDA for hemophilia B with inhibitors.

Sanofi’s Chief Medical Officer, Dietmar Berger, emphasized the company’s ongoing commitment to the hemophilia community, highlighting the importance of providing versatile treatment options like ALTUVIIIO and fitusiran that can cater to diverse clinical needs throughout a patient’s life.

ALTUVIIIO is a first-in-class factor VIII therapy designed for extended bleed protection with once-weekly prophylaxis, distinguishing itself with a longer half-life and the ability to bypass the von Willebrand factor ceiling. It is currently approved in the US, Taiwan, Japan, and recently by the European Commission under the name Altuvoct.

The XTEND-ed study, involving previously treated severe hemophilia A patients, continues to explore the long-term efficacy and safety of ALTUVIIIO. Meanwhile, the collaboration between Sanofi and Sobi focuses on the development and commercialization of hemophilia treatments, strengthening the reach and impact of these innovative therapies globally.

Fitusiran, still under clinical investigation, holds promise as a first-in-class treatment for hemophilia A or B. Utilizing advanced siRNA technology, it aims to reduce the treatment burden with minimal injections per year, offering a substantial benefit to the global hemophilia community.

Sanofi remains at the forefront of hemophilia treatment advancements, driven by a mission to improve patient outcomes and innovate within the realm of rare blood disorders.