Lee Arnold
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Drugs. 2025 Aug;85(8):1073-1077. doi: 10.1007/s40265-025-02203-y. Epub 2025 Jun 28.
Haemophilia A and B are inherited bleeding disorders caused by mutations in clotting factors. Small interfering RNA therapies may be utilised to restore coagulation by preventing the synthesis of antithrombin. Fitusiran (QFITLIA™) is a small interfering RNA therapy that targets antithrombin, which is being developed by Sanofi for the prophylactic management of haemophilia A and B with or without inhibitors. Fitusiran has the potential to be administered less frequently than other prophylactic treatments for haemophilia. This article summarizes the milestones in the development of fitusiran leading to this first approval for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients aged 12 years and older with haemophilia A or B with or without factor VIII or IX inhibitors.
甲型和乙型血友病是由凝血因子突变引起的遗传性出血性疾病。小干扰RNA疗法可通过阻止抗凝血酶的合成来恢复凝血功能。fitusiran（QFITLIA™）是一种靶向抗凝血酶的小干扰RNA疗法，赛诺菲正在开发该疗法用于有或无抑制剂的甲型和乙型血友病的预防性治疗。与其他血友病预防性治疗相比，fitusiran有可能减少给药频率。本文总结了fitusiran的研发历程中的重要节点，该药物首次获批用于常规预防，以预防或减少12岁及以上患有甲型或乙型血友病且有或无VIII或IX因子抑制剂的成人和儿科患者的出血发作频率。
