FDA approves siRNA drug for haemophilia prophylaxis
Briefly

Sanofi and Alnylam's fitusiran, branded as Qfitlia, has been approved by the FDA for routine prophylaxis in patients with hemophilia A or B, applicable for those 12 years and older. This innovative treatment targets antithrombin using siRNA and offers a dosing flexibility of once every two months, which is a more convenient option than traditional factor VIII and IX therapies that require more frequent administration. This represents a major step forward in managing hemophilia, improving patient adherence to treatment regimens and potentially enhancing their quality of life.
Sanofi and Alnylam's fitusiran (Qfitlia) is a groundbreaking treatment approved by the FDA for routine prophylaxis in patients with hemophilia A or B, distinguishing itself with a dosing schedule of once every two months.
The approval of fitusiran marks a significant advancement in hemophilia treatment, offering an innovative solution for patients aged 12 and older, whether or not they have factor VIII or IX inhibitors.
Read at Nature
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