US FDA approves Sanofi’s bleeding disorder therapy

 ​On March 28, 2025, the U.S. Food and Drug Administration (FDA) approved Sanofi's Qfitlia (fitusiran) for routine prophylaxis to prevent or reduce bleeding episodes in individuals aged 12 and older with hemophilia A or B, with or without factor VIII or IX inhibitors. ​

Qfitlia is a subcutaneous therapy administered every two months, offering a less frequent dosing schedule compared to existing treatments like Pfizer's weekly injection Hympavzi and Novo Nordisk's daily Alhemo. It functions by lowering antithrombin, a protein that inhibits blood clotting, thereby enhancing the body's ability to form clots and prevent bleeding. ​

Clinical trials demonstrated that Qfitlia reduced annualized bleeding rates by 90% compared to control groups, indicating a significant improvement over on-demand treatments. The therapy is scheduled to be available starting in April 2025, with an annual list price of $642,000 for most patients. ​

Hemophilia is a rare genetic disorder affecting approximately 33,000 males in the United States, characterized by a deficiency in clotting factors leading to severe bleeding episodes. The approval of Qfitlia introduces a novel treatment option that may significantly enhance the quality of life for individuals managing this condition.