Qfitlia FDA Approval History
Last updated by Judith Stewart, BPharm on March 29, 2025.
FDA Approved: Yes (First approved March 28, 2025)
Brand name: Qfitlia
Generic name: fitusiran
Dosage form: Injection
Company: Sanofi
Treatment for: Hemophilia A, Hemophilia A with Inhibitors, Hemophilia B, Hemophilia B with Inhibitors
Qfitlia (fitusiran) is an antithrombin-directed small interfering ribonucleic acid for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.
- Qfitlia is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B with or without factor VIII or IX inhibitors.
- Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency (factor VIII in hemophilia A and factor IX in hemophilia B) which prevents normal blood clotting, resulting in episodes of bleeding.
- Qfitlia is a small interfering RNA (siRNA) therapy and the first antithrombin-lowering therapy for use in hemophilia. It is designed to lower antithrombin (a protein that inhibits blood clotting) to promote thrombin generation and prevent bleeds. Qfitlia utilizes Alnylam Pharmaceutical Inc.’s ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability, offering consistent protection with as few as six injections a year.
- FDA approval was is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors. Key results include:
- significant bleed reduction by 71% in ABR for patients without inhibitors treated with Qfitlia prophylaxis compared to clotting factor concentrate on-demand and by 73% in ABR compared to bypassing agent on-demand for patients with inhibitors.
- median observed ABR during the open-label extension study was 3.8 in patients without inhibitors and 1.9 in patients with inhibitors.
- median observed annualized spontaneous bleeding rate during the open-label extension study was 1.9 in patients without inhibitors and 1.9 in patients with inhibitors.
- nearly half of patients in the open-label extension study experienced one or fewer bleeds (31% 0 bleeds and 47% 0-1 bleeds). - Qfitlia is administered by subcutaneous injection, starting at once every 2 months. The dose or frequency of administration can be adjusted to maintain antithrombin activity between 15-35%.
- Qfitlia comes with a Boxed Warning for adverse reactions, including thrombotic events, and acute and recurrent gallbladder disease. Warnings and precautions associated with Qfitlia include hepatotoxicity, and liver tests should be obtained at baseline and then monthly for at least 6 months after initiating treatment, and after dose increases.
- Common adverse reactions (incidence >10%) include viral infection, nasopharyngitis, and bacterial infection.
Development timeline for Qfitlia
Further information
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