The U.S. Food and Drug Administration (FDA) has approved Qfitlia (fitusiran) as a new therapy to prevent bleeding episodes in individuals with hemophilia A and B, including children aged 12 and older.
Qfitlia received FDA approval today, offering benefits to patients with hemophilia by requiring fewer medication administrations as compared to currently available drugs,” stated Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research. The approval reflects our ongoing commitment to improving the quality of life for individuals with hemophilia.
People with the inherited blood disorders hemophilia A and B experience impaired blood clotting due to a deficiency in clotting factors VIII (FVIII) or IX (FIX). Insufficient clotting factor in individuals with hemophilia A or B results in uncontrolled bleeding episodes that occur through spontaneous body bleeding and injuries. Medical professionals provide patients with two treatment options, including on-demand care and prophylactic dose regimens that utilize clotting factor products.
Qfitlia works through a mechanism different from conventional treatments. In contrast to other treatments, it does not substitute for the deficient clotting factor. Instead, it lowers the level of the protein antithrombin, which results in a rise in thrombin, an enzyme essential for blood clotting.
The main advantage of using Qfitlia is its bi-monthly dosing schedule because it needs treatment only once every two months, unlike standard options. The INNOVANCE Antithrombin companion diagnostic test helps health providers determine the correct dose and injection schedule for each patient. Patients benefit from the INNOVANCE Antithrombin companion diagnostic test to regulate their treatment and maintain optimal antithrombin levels that minimize the risks of both bleeding and clotting.
The efficacy and safety of Qfitlia were examined in two multicenter, randomized clinical trials with 177 patients with hemophilia A or B. Patients in one study had antibodies that prevented their body from reacting to FVIII or FIX therapy. Patients in the other study had no such antibodies.
In both trials, the patients either received Qfitlia or their usual on-demand treatments for nine months. The research revealed that Qfitlia minimized bleeding episodes by 73% annually among patients who made antibodies (inhibitor patients) and by 71% among other patient groups compared to their prior treatment methods.
The doses were adjusted throughout a long-term study according to antithrombin level test results from the patients. Medical professionals discovered that patient-specific treatment methods reduced bleeding occurrences progressively.
Qfitlia carries important safety considerations. It includes warning about risk of blood clots and gallbladder disease, requiring some patients to receive gallbladder removal surgery. The medication can lead to liver toxicity, so healthcare providers must perform liver examination tests before initiation and during the first half year after increasing treatment doses.
Qfitlia treatment is associated with an increased risk of viral infections like the common cold and bacterial infections. Despite these risks, Qfitlia represents a new treatment opportunity for people with hemophilia, although it also carries safety risks regarding infections. The approval demonstrates continued attempts to provide hemophilia patients with superior treatment methods that also bring increased accessibility and improve patients’ quality of life.
References: Food and Drug Administration. FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors Published March 28, 2025, Accessed April 03, 2025 FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors | FDA
