Thanks to Ali Sadeghi-Khomami, PhD, PBI research scientist, for the following heads-up:
On Friday, March 28, 2025, Sanofi Inc. announced the much-awaited approval of fitusiran (Qfitlia®), a “rebalancing” therapeutic that dramatically reduces the ABR in hemophilia A and hemophilia B patients ≥12 YOA with or without inhibitors. Fitusiran is a messenger RNA inhibitor (RNAi) that targets antithrombin production. Antithrombin is a coagulation control factor that slows the activation of prothrombin (FII) and FX. By reducing antithrombin production, fitusiran enables the hemophilic patient to regain near-normal clot formation, relying on the “Goldilocks Principle.” Fitusiran is administered SC as infrequently as once every two months. Sanofi provides the Siemens Healthineers’ INNOVANCE Antithrombin® assay, a chromogenic substrate method, as a companion diagnostic tool to support fitusiran dosing. Fitusiran joins recently approved rebalancing therapeutics concizumab (Alhemo®) and marstacimab (HYMPAVZI®), both anti-TFPI SC therapeutics.
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