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Fitusiran, a groundbreaking treatment for hemophilia A and B is approved by the FDA, offering a less frequent dosing schedule of once every 2 months. This approval applies to adults and pediatrics aged 12 years and older.
In a major advancement for hemophilia care on 28 March 2025, the Food and Drug Administration (FDA) has granted approval to fitusiran (small interfering RNA [siRNA] therapy) for routine prophylaxis to prevent or minimize bleeding episodes in hemophilia A or B. This also includes individuals with or without factor inhibitors (neutralizing antibodies).
The New Treatment Option with Less Frequent Dosing
Fitusiran stands out by offering a subcutaneous injection every 2 months, while current treatments often require frequent infusions of clotting factors. Fitusiran doesn't replace the missing clotting factor; instead, it works by lowering levels of a protein called antithrombin. This reduction boosts the production of thrombin—an essential enzyme for blood clotting. This results in a 71-73% reduction in bleeding episodes when compared to existing therapies.
Fitusiran’s efficacy and safety were assessed through two multicenter, randomized clinical trials. The trial included 177 adult and pediatric male patients with hemophilia A or B. Volunteers were randomized to receive either monthly fixed-dose fitusiran or their usual on-demand therapy. After 9 months, patients entered a long-term extension study, receiving individualized, antithrombin-based dosing of fitusiran—the approved regimen.
Important Eligibility Updates:
As hemophilia care continues to evolve, fitusiran’s approval marks a potential turning point for patients seeking a more manageable, less frequent treatment option. With ongoing advancements in therapies like this, hope grows for a future where hemophilia is not just treated but truly controlled — offering patients the freedom to live life without the constant burden of bleeding episodes.
FDA News Release
FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors
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