For most patients suffering from a chronic illness symptoms is of course their main struggling area, but what sometimes goes un noticed is the frequency & commitment to take their medications each day for the rest of their life.
Luckly with growing strides to find more patient compliant solutions FDA has just recently approved the use of Qfitlia (fitusiran) for routine prophylaxis aimed at preventing or minimizing the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B, including those with or without factor VIII or IX inhibitors (neutralizing antibodies).
Hemophilia A and B are genetic bleeding disorders resulting from a deficiency or dysfunction of coagulation factors VIII (FVIII) or IX (FIX). Patients with these conditions struggle to clot effectively, leading to prolonged bleeding after injuries or surgeries and potential spontaneous bleeding in muscles, joints, and organs, which can be life-threatening. Bleeding episodes are usually managed through on-demand treatment or prophylaxis with FVIII or FIX products, or alternatives that mimic these factors.
What differentiates Qfitlia (fitusiran) among already existing treatment options is the dosing frequency, as it is taken once every two-month making it easier for patients to adhere to. It works by decreasing the levels of antithrombin protein which in turn increases thrombin that is responsible for providing the clotting effect.
One group included patients with inhibitors to FVIII or FIX who had been treated with bypassing agents, while the other group included those without inhibitors who had used clotting factor concentrates. Participants were randomized to receive either a fixed monthly dose of Qfitlia or their usual on-demand treatment for nine months. However, the fixed-dose regimen of Qfitlia was not approved due to the risk of excessive clotting observed in some patients.
The approval was based on results from two randomized trials and a long-term extension study, which demonstrated a 71–73% reduction in annualized bleeding rates compared to on-demand treatment in both inhibitor and non-inhibitor patient populations.
While Qfitlia presents a significant step forward, it carries a boxed warning for potential thrombotic events and gallbladder disease, along with cautions regarding liver toxicity. FDA granted Qfitlia Orphan Drug and Fast Track designations.
Learn more: FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors | FDA
