The U.S. Food and Drug Administration (FDA) has approved Qfitlia (fitusiran), the first-ever antithrombin-lowering therapy for hemophilia A and B. This groundbreaking treatment is designed to reduce bleeding episodes in both adult and pediatric patients (ages 12 and older), regardless of the presence of factor VIII or IX inhibitors.
Qfitlia works by lowering the levels of antithrombin, a protein that inhibits blood clotting, thereby enhancing thrombin generation and improving blood clotting. It utilizes small-interfering RNA technology, allowing for fewer injections—only six per year—via prefilled pens or syringes. The therapy is associated with lower bleeding rates and offers a convenient treatment regimen compared to traditional therapies.
The approval is based on results from the ATLAS Phase 3 studies, which showed that Qfitlia significantly reduced annualized bleeding rates (ABR) in patients with and without inhibitors. For example, patients without inhibitors experienced a 71% reduction in ABR, while those with inhibitors saw a 73% reduction when compared to on-demand treatments.
Experts highlight Qfitlia’s potential to transform hemophilia care by reducing treatment frequency, offering simplified administration, and providing effective protection against bleeding. The therapy has received Orphan Drug Designation, Fast Track Designation, and Breakthrough Therapy Designation from the FDA, reflecting its significance for the hemophilia community.
In addition to Qfitlia’s approval, Siemens Healthineers’ INNOVANCE® Antithrombin assay has been cleared as a companion diagnostic to measure antithrombin levels in patients prescribed Qfitlia. The therapy will be available at a comparable price to other hemophilia treatments, and Sanofi has launched HemAssist, a patient support program, to offer financial assistance and educational resources.
Qfitlia is also under review in Brazil, with a regulatory decision expected in China later in 2025.
