The U.S. Food and Drug Administration (FDA) recently approved Qfitlia™ (fitusiran) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors (neutralizing antibodies). The new therapy is manufactured by Sanofi.
Qfitlia is a subcutaneous therapy that uses small interfering RNA (siRNA) technology to target antithrombin (AT), a liver-generated clotting protein that plays a key role in the regulation of blood clots. siRNA molecules are segments of RNA (ribonucleic acid) that block or “silence” the activity of certain genes through RNA interference, a natural biological process common in plants and mammals. It works by silencing the gene responsible for AT, which inhibits the protein’s anticoagulant function. This then compensates for the imbalance caused by deficiencies in other clotting proteins, such as factor VIII (hemophilia A) or factor IX (hemophilia B).
“Today’s approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,” said Tanya Wroblewski, MD, deputy director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research. “This new treatment option highlights our continued efforts to improve the lives of patients with hemophilia.”
Read the full FDA release to learn more, including the clinical trial results that supported the agency's approval of Qfitlia.
Source: FDA news release dated March 28, 2025
