FDA Approves Qfitlia: A New Hemophilia Treatment with Less Frequent Dosing 

United States: The U.S. Food and Drug Administration approved Qfitlia (fitusiran) for prevention of bleeding episodes in patients aged 12 and older with factor VIII or IX deficiency who have or do not have inhibitors. The new drug addition brings an infrequent administration schedule available for patients who have hemophilia. 

How Qfitlia Works 

Qfitlia functions through subcutaneous delivery to decrease antithrombin protein amounts which enables an increase in blood-clotting enzyme thrombin. The procedure generates a beneficial impact on preventing bleeding that exceeds safe levels in patients affected by hemophilia. 

Unlike fixed-dose treatments, Qfitlia requires personalized dosing, determined by the FDA-certified INNOVANCE Antithrombin companion diagnostic test (Siemens). The standard regimen involves injections every two months based on test results. 

However, due to high clotting risks, the FDA has not approved fixed dosing. Additionally, Qfitlia carries a boxed warning for blood clotting events and gallbladder disease. Commonly reported side effects include viral infections, nasopharyngitis, and bacterial infections, according to HealthDay. 

Key Clinical Trial Findings 

The FDA granted its approval after professionals conducted two randomized clinical trials with 177 male patients who had hemophilia A or B. The results demonstrated: 

The yearly bleeding rates dropped by 73% for patients with inhibitors who used these proteins versus those who relied on on-demand bypassing agents. 

A 71% reduction in estimated annual bleeding rates compared to those relying on clotting factor concentrates as needed. 

These findings confirm Qfitlia’s effectiveness in significantly reducing bleeding episodes in patients with hemophilia. 

A Milestone for Hemophilia Treatment 

During a recent interview Dr. Tanya Wroblewski underlined the significance of this regulatory approval for the FDA Center for Drug Evaluation and Research. 

“Today’s approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,” she stated in a report by HealthDay. 

With fewer injections and strong efficacy in preventing bleeds, Qfitlia provides a new and promising treatment alternative for individuals managing hemophilia A or B.