FDA Approves Sanofi's Qfitlia as First Universal Hemophilia Treatment

Sanofi has achieved a significant milestone with the approval of its new haemophilia treatment, Qfitlia (fitusiran), by the US Food and Drug Administration (FDA). As the company navigates a competitive market, it hopes Qfitlia's unique attributes will distinguish it from existing therapies.

Qfitlia has been approved for use in both adults and adolescents who suffer from haemophilia A or B, irrespective of the presence of factor VIII or IX inhibitors. This flexibility is a significant advantage over other treatments that may not accommodate patients with inhibitors. Inhibitors are antibodies that can develop in some haemophilia patients, rendering factor replacement therapies ineffective.

Sanofi's Qfitlia represents a new class of treatment as it is the first antithrombin-lowering therapy approved for routine prophylaxis. Its primary function is to prevent or decrease the frequency of bleeding episodes commonly experienced by patients with haemophilia. Unlike traditional treatments that replace missing clotting factors, Qfitlia operates by reducing levels of antithrombin, thereby enhancing thrombin production. Thrombin is an enzyme crucial for blood clotting. The drug utilizes small-interfering RNA technology, which facilitates less frequent treatment, subcutaneous administration, and low-volume injections.

The efficacy of Qfitlia has been demonstrated in three completed Phase III clinical trials: ATLAS-INH, ATLAS-A/B, and ATLAS-PPX. These studies have shown that Sanofi’s monthly subcutaneous therapy can reduce annualized bleeding rates by 90% compared to on-demand clotting factor concentrates and bypassing agents. However, patients using Qfitlia may experience common adverse events such as viral and bacterial infections. The drug carries a boxed warning for potential thrombotic events and gallbladder disease, as well as liver toxicity risks. Sanofi had previously faced clinical holds due to safety concerns, including a fatal blood clot in 2017 and a non-fatal thrombotic event in 2020, but these risks were mitigated after identifying a revised treatment protocol.

Currently, an ongoing Phase III open-label extension study is examining a new dosing regimen, aiming to reduce the frequency of injections to as few as six per year.

In the competitive landscape of haemophilia treatments, Qfitlia's monthly administration schedule already gives it an edge over Novo Nordisk's daily Alhemo (concizumab) and Pfizer's weekly Hympavzi (marstacimab), both recently approved by the FDA. Qfitlia’s ability to treat patients regardless of inhibitor presence further enhances its appeal, especially when compared to Alhemo, which is limited to patients with inhibitors, and Hympavzi, which is used for those without. Pfizer has also recently discontinued its other haemophilia medication, Beqvez (fidanacogene elaparvovec), due to a lack of interest in the market.

Phil Gattone, president and CEO of the National Bleeding Disorders Foundation, emphasized the significance of such advancements, noting that current treatment options often force patients to choose between effective control of bleeding and convenient dosing schedules. Qfitlia offers a novel approach by providing effective protection while reducing the treatment burden for patients and their families.

The haemophilia drug market in the United States is projected to reach $7.2 billion by 2030. Currently, Roche’s Hemlibra (emicizumab) stands as the top-selling drug in the field, particularly for haemophilia A patients, generating significant revenue globally. As Sanofi enters this lucrative market with Qfitlia, it aims to redefine treatment possibilities for haemophilia patients, offering them enhanced management options and improved quality of life.