Fitusiran

WEDNESDAY, April 2, 2025 -- The U.S. Food and Drug Administration has 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). Subcutaneous Qfitlia does not replace the missing coagulation factor VIII or IX, but reduces the amount of the protein antithrombin, leading to an increase in thrombin, an enzyme critical for blood clotting. Dosing starts at once every two months and is based on the FDA-cleared INNOVANCE Antithrombin companion diagnostic test (Siemens). Fixed dosing of Qfitlia is not approved due to excessive clotting risk. Qfitlia has a boxed warning for thrombotic events and gallbladder disease. The most common side effects reported were viral infection, nasopharyngitis, and bacterial infection. The approval is based on two randomized clinical trials that enrolled 177 adult and pediatric male patients with either hemophilia A or hemophilia B. Among participants with inhibitors who received the antithrombin-based dosing regimen of Qfitlia, there was a 73 percent reduction in the estimated annualized bleeding rate compared with that seen in those who received on-demand treatment with bypassing agents. Among participants without inhibitors receiving the antithrombin-based dosing regimen of Qfitlia, there was a 71 percent reduction in the estimated annualized bleeding rate versus that seen in those who received on-demand treatment with clotting factor concentrates. "Today's approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options," Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA Center for Drug Evaluation and Research, said in a statement. The approval of Qfitlia was granted to Sanofi. More Information Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Sanofi’s Qfitlia (fitusiran) has been approved by the US Food and Drug Administration (FDA) as the first therapy to treat haemophilia A or B regardless of inhibitor status. The drug has been authorised to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients aged 12 and older with or without factor VIII or IX inhibitors, antibodies that prevent factor replacement treatments from working. Haemophilia A and B are rare genetic bleeding disorders caused by a dysfunction or deficiency of clotting factor VIII or IX, respectively. Patients may bleed for a longer time than normal after injury or surgery, and can also experience spontaneous bleeding into muscles, joints and organs. Utilising Alnylam Pharmaceuticals’ ESC-GalNAc conjugate technology, Qfitlia prevents bleeds and helps rebalance haemostasis by lowering antithrombin, a protein that inhibits blood clotting. The therapy is given as a subcutaneous injection starting once every two months, with the dose and frequency adjusted using Siemens Healthineers’ Innovance Antithrombin assay, a companion diagnostic test to measure antithrombin levels. Phil Gattone, president and chief executive officer, National Bleeding Disorders Foundation, said: “Current treatment options can make people with haemophilia feel they need to choose between effective bleed control and convenient dosing schedules, leading to trade-offs when it comes to disease management. “Qfitlia takes a novel approach to providing protection for people living with haemophilia while reducing the frequency of dosing for patients and their families.” The FDA’s decision was supported by positive results from the late-stage ATLAS studies, in Qfitlia which demonstrated low bleed rates across patient subgroups. Prophylaxis treatment with Qfitlia reduced annualised bleeding rate (ABR) by 71% compared to on-demand clotting factor concentrate in patients without inhibitors. Among those with inhibitors, Sanofi’s drug was shown to reduce ABR by 73% compared to on-demand treatment with bypassing agents. Brian Foard, executive vice president, head of specialty care, Sanofi, said: “Qfitlia has the potential to meaningfully change the haemophilia landscape through effective bleed protection, infrequent dosing and simplified administration. “Our robust portfolio of haemophilia treatment options continues to grow as we focus on offering protection with reduced treatment burden that best fits an individual’s needs.”