Related introduction of Qfitlia (Fitusiran): What kind of medicine is it used to treat?

Qfitlia (Fitusiran) is an antithrombin-directed small interfering RNA indicated for the prophylactic treatment of patients with hemophilia A or B, with or without inhibitors. Qfitlia is also indicated for routine prophylaxis in adults and children 12 years of age and older with hemophilia A or B, to prevent or reduce the frequency of bleeding events, with or without factor VIII or IX inhibitors.

Hemophilia is a family of rare inherited blood disorders caused by a deficiency in clotting factors (factor VIII in hemophilia A and factor IX in hemophilia B) that prevents normal blood clotting, leading to bleeding episodes. Qfitlia, a small interfering RNA (siRNA) therapy, is the first antithrombin-lowering therapy for hemophilia. It is designed to lower antithrombin, a protein that inhibits blood clotting, to promote thrombin generation and prevent bleeding.

Qfitlia utilizes Alnylam Pharmaceutical Inc.'sESC-GalNAc conjugate technology, which enables subcutaneous delivery with greater potency and durability, providing consistent protection with just six injections per year. The U.S. Food and Drug Administration (FDA) approval is based on data from the phase ATLAS study, which demonstrated clinically meaningful protection against bleeding, as measured by the annual bleeding rate (ABR) in patients with hemophilia with and without inhibitors. Qfitlia is administered by subcutaneous injection, starting every 2 months. The dose or frequency of administration can be adjusted to maintain 15-35% antithrombin activity.

Qfitlia comes with a boxed warning for adverse reactions, including thrombotic events and acute and recurrent gallbladder disease. Common adverse reactions (incidence >10%) include viral infections, nasopharyngitis, and bacterial infections. Warnings and precautions related to Qfitlia include hepatotoxicity, liver tests should be performed at baseline and then monthly for at least 6 months after starting treatment and after dose increases.

Reference materials:https://www.drugs.com/history/qfitlia.html