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Investing.com -- The US Food and Drug Administration (FDA) has approved Sanofi (NASDAQ:SNY)’s treatment for hemophilia, Qfitlia (fitusiran), which can be administered as infrequently as once every other month. This new approval provides patients with more choices as the market for therapies continues to grow.
Qfitlia is designed for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or B, with or without factor VIII or IX inhibitors.
Tanya Wroblewski, M.D., deputy director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research, highlighted the significance of this approval, stating that Qfitlia can be administered less frequently than other existing options, reflecting the FDA’s ongoing efforts to improve the lives of patients with hemophilia.
Hemophilia A and B are genetic bleeding disorders caused by a dysfunction or deficiency of coagulation factor VIII (FVIII) or IX (FIX), respectively. Qfitlia does not replace the missing clotting factor; instead, it reduces the amount of a protein called antithrombin, leading to an increase in thrombin, an enzyme critical for blood clotting.
Qfitlia is administered under the skin (subcutaneously) starting once every two months. The dose and frequency of injections are adjusted using the FDA-cleared INNOVANCE Antithrombin companion diagnostic test, which is intended to monitor and achieve antithrombin activity in the target range to reduce the risk of bleeding and excessive blood clotting. The INNOVANCE Antithrombin test was granted clearance by the FDA to Siemens (ETR:SIEGn) Healthcare Diagnostics GmbH.
The efficacy and safety of Qfitlia were assessed in two multicenter, randomized clinical trials, involving a total of 177 adult and pediatric male patients with either hemophilia A or B. Participants subsequently entered a long-term extension study in which they received an adjustable dose of Qfitlia based on periodic measurements of antithrombin activity. This antithrombin-based dosing regimen is the approved dosage regimen.
The primary measure of efficacy of Qfitlia was the estimated annualized bleeding rate of treated bleeds. In participants who received the antithrombin-based dosing regimen of Qfitlia, there was a 73% reduction in estimated annualized bleeding rate compared to those who received on-demand treatment with bypassing agents. There was a 71% reduction in estimated annualized bleeding rate in participants without inhibitors who received the antithrombin-based dosing regimen of Qfitlia, compared to those who received on-demand treatment with clotting factor concentrates.
Qfitlia comes with a boxed warning for thrombotic events (blood clotting) and gallbladder disease, with some patients requiring gallbladder removal. It also includes a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with Qfitlia or after a dose increase.
Common side effects of Qfitlia include viral infection, common cold symptoms (nasopharyngitis), and bacterial infection. The FDA granted Qfitlia Orphan Drug and Fast Track designations for this application.
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