FDA approves Sanofi’s Qfitlia for Hemophilia A and B

USA—The U.S. Food and Drug Administration (FDA) has approved Qfitlia (fitusiran), an innovative treatment for patients with hemophilia A and B.

This approval signifies a significant advancement in the management of genetic bleeding disorders characterized by deficiencies or dysfunctions in clotting factors VIII and IX, respectively.

Patients with hemophilia often experience prolonged bleeding following injuries or surgeries and may suffer from spontaneous bleeding in muscles, joints, and organs, which can be life-threatening.

Qfitlia is intended to decrease the frequency of bleeding episodes in adult and pediatric patients aged 12 and older, regardless of the presence of inhibitors—antibodies that may reduce the effectiveness of traditional treatments.

Unlike traditional treatments that replace missing clotting factors, Qfitlia functions by reducing levels of antithrombin, a protein that hinders blood clotting.

This action enhances thrombin production, an enzyme crucial for blood clotting, thereby restoring balance to the body’s clotting system.

Tanya Wroblewski, M.D., Deputy Director of the Division of Non-Malignant Hematology at the FDA, highlighted the significance of this approval.

She noted that Qfitlia can be administered less frequently than existing treatments, which is a major advantage for patients.

This new treatment option underscores the FDA’s ongoing efforts to improve the lives of those living with hemophilia.

Qfitlia is administered via subcutaneous injections, initially given once every two months.

The dosage and frequency of injections are adjusted based on the Innovance Antithrombin Assay, a companion diagnostic test that ensures antithrombin levels remain within a target range.

 This approach helps minimize the risk of both excessive bleeding and clotting.

The efficacy and safety of Qfitlia were evaluated in clinical trials involving adult and pediatric patients with hemophilia A or B.

These studies demonstrated significant reductions in bleeding rates compared to traditional on-demand treatments.

For patients with inhibitors, Qfitlia reduced bleeding rates by 73%, while those without inhibitors saw a 71% reduction.

However, Qfitlia carries significant warnings. It includes a boxed warning concerning thrombotic events (blood clots) and gallbladder disease, which may require surgical removal in some cases.

Additionally, there’s a warning regarding liver toxicity, necessitating regular monitoring of liver function tests.

Common side effects include viral infections, symptoms of the common cold, and bacterial infections.

Despite these risks, Qfitlia offers a novel approach to managing hemophilia by providing effective bleed protection with less frequent dosing, which can simplify disease management for patients and their families. 

The FDA granted Qfitlia Orphan Drug and Fast Track designations, reflecting its potential to address significant unmet medical needs.

Sanofi, the developer of Qfitlia, has emphasized its commitment to advancing innovation in the treatment of rare blood disorders.

 As the first therapy of its kind to receive FDA approval for both hemophilia A and B, Qfitlia represents a promising step forward in the management of these conditions.

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