Sanofi Presents Rare Disease Data at Thrombosis and Hemostasis Congress
Sanofi will present new data on rare disease treatments, including immune thrombocytopenia and hemophilia, at the International Society on Thrombosis and Haemostasis (ISTH) congress in Washington D.C.
Pharmaceutical company Sanofi will present 18 new research findings, including five oral presentations, at the 33rd congress of the International Society on Thrombosis and Haemostasis (ISTH) in Washington D.C., June 21-25, 2025. The data focus on rare blood diseases, particularly immune thrombocytopenia (ITP) and hemophilia.
Sanofi will showcase data on the safety and efficacy of rilzabrutinib, an oral BTK inhibitor, for ITP. New findings from the LUNA 3 Phase 3 study will highlight the drug's potential to improve platelet counts. Rilzabrutinib is currently under regulatory review in the US, EU, and China for ITP, with a decision expected from the US Food and Drug Administration (FDA) in August 2025.
Additionally, data on Sanofi's hemophilia treatments, ALTUVIIIO and Qfitlia, will be presented. Modeling data for Qfitlia predict a correlation between lowering antithrombin (AT) levels and bleed protection. Long-term data for ALTUVIIIO reinforce its efficacy and safety in adults and children with severe hemophilia A.
Sanofi states its commitment to addressing unmet needs in rare blood diseases. The company sees potential in rilzabrutinib to target underlying immune dysregulation and inflammation in rare conditions.
These presentations underscore Sanofi's position in hemophilia treatment and its efforts to expand treatment options for patients with rare blood disorders.