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Sanofi presented new clinical trial results at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress, demonstrating its dedication to developing therapies for uncommon blood disorders. The presentations reaffirmed the effectiveness of Sanofi's hemophilia treatments, ALTUVIIIO and Qfitlia, and emphasized the potential of rilzabrutinib, a reversible oral BTK inhibitor, in treating immune thrombocytopenia (ITP). Advancements in Immune Thrombocytopenia (ITP) Sanofi reported data from the Phase 3 LUNA 3 trial, highlighting rilzabrutinib's capacity to treat the underlying immune dysregulation in ITP. A podium presentation reported platelet response rates by International Working Group (IWG) criteria—a principal clinical endpoint used for informed decision-making about treatment. Two poster presentations reported the first efficacy and safety data during the LUNA 3 open-label extension and investigated platelet count fluctuation in rilzabrutinib-treated patients. Rilzabrutinib is also under review by regulatory authorities in the U.S., EU, and China for ITP and is expected to receive a decision from the U.S. Food and Drug Administration by August 29, 2025. The FDA has bestowed fast track designation on the therapy for its ability to fill unmet medical needs. Our commitment to rare blood diseases drives us to find innovative solutions for the persistent unmet needs of patients and caregivers. Our data highlight the benefits of our hemophilia treatments and the potential of our reversible oral BTK inhibitor, rilzabrutinib, which could be transformative in rare diseases marked by complex immune dysregulation and inflammation. Christopher Corsico Enhancing Hemophilia Treatment Options Additionally, Sanofi provided evidence demonstrating leadership in the treatment of hemophilia. The benefits of ALTUVIIIO and the recently launched Qfitlia, which are both intended to provide more treatments and improve patient outcomes, were emphasized in the presentations. By offering cutting-edge hemophilia treatment alternatives, these therapies aim to address the persistent unmet needs of patients and caregivers. Source: Sanofi Last Modified:

Tag: Rare Diseases

Sanofi Showcases Breakthrough Data in Rare Blood Disorders at ISTH 2025

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