Sanofi's Qfitlia (Fitusiran), the First siRNA Therapy for Hemophilia, Receives FDA Approval
Sanofi
Pharmaceutical R&D Developer
Local time on March 28, Sanofi announcedsiRNA TherapyQfitlia (Fitusiran) Receives FDA Approval in the United StatesUsed forPrevention or reduction in adult and pediatric patients with hemophilia A or B(With or without factor VIII or IX inhibitors)Frequency of bleeding episodes。
Screenshot source: Sanofi official website
Hemophilia A and B are rare congenital lifelong bleeding disorders, characterized by coagulation dysfunction, which can cause excessive bleeding in joints. Fitusiran can reduce antithrombin.(A protein that inhibits blood coagulation)Level, thereby promoting thrombin generation, rebalancing hemostatic function, and preventing bleeding.
This approval is based on the data from the Phase III study ATLAS. In this study,QfitliaShowed a lower bleeding rate in subgroups, requiring only six injections per year.. The main results include:
Compared to on-demand use of clotting factor concentrates, prophylactic treatment with QfitliaABR bleeding significantly reduced by 71% in patients without inhibitors(Estimated mean: ABR 9.0 vs. 31.4; p<0.0001), compared with on-demand use of bypass agents,ABR bleeding reduced by 73% in patients with inhibitors(Estimated mean: ABR 5.1 vs. 19.1; p=0.0006)。
The median ABR observed during the open-label extension study was 3.8 in patients without inhibitors.（IQR：0.0–11.2）, 1.9 in patients with inhibitors（IQR：0.0–5.6）。
The median annual spontaneous bleeding rate observed during the open-label extension study was 1.9 in patients without inhibitors.(Interquartile Range (IQR): 0.0-7.5), 1.9 in patients with inhibitors（IQR： 0.0-3.7)。
In the open-label extension study,Nearly half of the patients experienced one or fewer bleeding episodes.(31% with 0 bleeding episodes and 47% with 0-1 bleeding episodes)。
In terms of safety, serious adverse reactions may occur, including thrombotic events, acute and recurrent gallbladder disease, and hepatotoxicity. The most common adverse reactions(Incidence rate >10%)It is viral infection, nasopharyngitis, and bacterial infection.
Combined with the approval of Qfitlia, the FDA also approved Siemens Healthineers' INNOVANCE® Antithrombin assay as a companion diagnostic for Qfitlia to measure AT levels. Through the Qfitlia Testing Program in collaboration with Labcorp, the FDA-approved companion diagnostic will be provided free of charge to patients receiving a Qfitlia prescription to measure AT levels.
In China, the CDE accepted the new drug marketing application for Fitusiran Injection in May 2024. The product has also been CDE Included in the "Patient-Centered Rare Disease Drug Development Pilot Program ('Care Program')" pilot project, intended for use as a routine preventive treatment,For adult and adolescent patients ≥12 years old with Hemophilia A or B, with or without inhibitors to coagulation Factor VIII or IX, to prevent or reduce the frequency of bleeding episodes.。
