WASHINGTON, D.C. — The U.S. Food and Drug Administration (FDA) has announced the approval of Qfitlia (fitusiran), a treatment designed to prevent or reduce the frequency of bleeding episodes in individuals with hemophilia A or hemophilia B. This approval applies to both adults and pediatric patients aged 12 years and older, including those with or without factor VIII or IX inhibitors.
“Today’s approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,” said Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research. “This new treatment option highlights our continued efforts to improve the lives of patients with hemophilia.”
A Novel Approach to Treating Hemophilia
Hemophilia A and B are genetic disorders that impair the body’s ability to form blood clots due to a deficiency of coagulation factors VIII (FVIII) or IX (FIX). This leads to excessive and prolonged bleeding following injuries, surgeries, or even spontaneous bleeding episodes in joints, muscles, or organs. Current management options include clotting factor replacement or treatments that mimic clotting mechanisms.
Qfitlia offers a different approach by targeting antithrombin, a protein that inhibits clot formation. By reducing antithrombin levels, Qfitlia increases levels of thrombin, a critical enzyme in the blood clotting process. Unlike traditional treatments, Qfitlia does not directly replace the missing clotting factors.
The treatment is delivered through subcutaneous injections, starting once every two months. The dosage and frequency are customized for each patient using data from the FDA-approved INNOVANCE Antithrombin companion diagnostic test. This test measures antithrombin activity and ensures the appropriate level is maintained to prevent excessive bleeding or clotting. The INNOVANCE Antithrombin test, developed by Siemens Healthcare Diagnostics GmbH, was cleared in conjunction with Qfitlia’s approval.
Clinical Trial Results Support Efficacy
The FDA’s approval of Qfitlia was based on findings from two large multicenter, randomized clinical trials involving 177 male patients with hemophilia A or B, some with inhibitory antibodies to FVIII or FIX. One trial studied patients using Qfitlia compared to on-demand treatment with bypassing agents, while the other compared Qfitlia against clotting factor concentrates.
Results showed that participants using the antithrombin-based dosing regimen of Qfitlia experienced a substantial reduction in bleeding episodes. For those with inhibitors, bleeding rates dropped by 73% compared to on-demand treatments. Among participants without inhibitors, rates decreased by 71%. These findings were further corroborated in a long-term extension study in which participants received personalized, adjustable doses based on periodic antithrombin measurements.
The fixed-dose regimen originally used in clinical trials was not approved due to reports of excessive clotting in some patients. The approved adjustable dosing regimen minimizes this risk while maintaining efficacy.
Safety and Warnings
Qfitlia includes a boxed warning for potential thrombotic events, gallbladder disease, and liver toxicity. Patients are advised to undergo regular liver function monitoring for at least six months after initiating treatment or following dose adjustments. Common side effects of Qfitlia include viral and bacterial infections as well as symptoms of the common cold, such as nasopharyngitis.
Regulatory Pathway and Designations
The FDA granted Qfitlia Orphan Drug and Fast Track designations, underscoring its importance for addressing the unmet medical needs of the hemophilia community. The approval was granted to Sanofi, reaffirming their role in advancing innovative treatments for rare bleeding disorders.
Transformative Impact on Hemophilia Care
This approval marks a notable advancement in the management of hemophilia, providing patients with a treatment that aligns with modern demands for convenience and efficacy. By reducing the frequency of administration while achieving significant reductions in bleeding episodes, Qfitlia has the potential to improve quality of life for individuals managing hemophilia A or B.
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