Qfitlia scores a first with FDA approval

The US Food and Drug Administration (FDA) has approved Qfitlia (fitusiran), the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors.

The approval is based on data from the ATLAS Phase III studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR) across hemophilia patients with or without inhibitors, said the drug’s developer, French pharma major Sanofi (Euronext: SAN).

“Current treatment options can make people with hemophilia 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 hemophilia while reducing the frequency of dosing for patients and their families,” commented Phil Gattone, president and chief executive of the National Bleeding Disorders Foundation.