FDA Approves Sanofi's Qfitlia for Haemophilia A and B Treatment

Sanofi's new medication, Qfitlia (fitusiran), recently received approval from the US Food and Drug Administration (FDA), marking it as the first therapy to address both haemophilia A and B irrespective of inhibitor presence. This advancement aims to prevent or decrease the frequency of bleeding episodes in individuals aged 12 and older, regardless of the presence of factor VIII or IX inhibitors. These inhibitors are antibodies that interfere with the effectiveness of factor replacement therapies.

Haemophilia A and B are uncommon genetic disorders characterized by a deficiency or dysfunction in clotting factors VIII and IX, respectively. This condition results in prolonged bleeding post-injury or surgery and can also lead to spontaneous bleeding within muscles, joints, and organs.

Qfitlia works through Alnylam Pharmaceuticals’ ESC-GalNAc conjugate technology. It helps prevent bleeding by reducing antithrombin, a protein that hinders blood clotting, thereby assisting in rebalancing haemostasis. The medication is administered via subcutaneous injection, initially every two months. The dosing schedule is tailored using Siemens Healthineers’ Innovance Antithrombin assay, a diagnostic test designed to measure antithrombin levels.

Phil Gattone, president and CEO of the National Bleeding Disorders Foundation, emphasized the significance of this new treatment option. He noted that existing therapies often force patients to choose between effective control of bleeding and convenient dosing, leading to compromises in disease management. Qfitlia, according to Gattone, offers a novel approach by ensuring protection for those living with haemophilia while reducing the frequency of doses, thus easing the burden on patients and their families.

The FDA's approval of Qfitlia was backed by positive outcomes from the late-stage ATLAS studies. These studies demonstrated low bleeding rates across different patient groups. Prophylactic treatment with Qfitlia lowered the annualized bleeding rate (ABR) by 71% compared to on-demand clotting factor concentrate among patients without inhibitors. For those with inhibitors, the drug showed a 73% reduction in ABR compared to on-demand treatment with bypassing agents.

Brian Foard, executive vice president and head of specialty care at Sanofi, spoke of Qfitlia's potential to significantly alter the treatment landscape for haemophilia. He highlighted its benefits in providing effective bleed protection while offering infrequent dosing and simplified administration. Foard also mentioned that Sanofi's comprehensive range of haemophilia treatment options continues to expand as the company remains focused on delivering solutions that combine optimal protection with a reduced treatment burden tailored to individual needs.