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FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors
Today, the U.S. Food and Drug Administration approved Qfitlia (fitusiran) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors (neutralizing antibodies).
Hemophilia A and hemophilia B are genetic bleeding disorders caused by a dysfunction or deficiency of coagulation factor VIII (FVIII) or IX (FIX), respectively. Patients with these hemophilias are unable to clot properly and may bleed for a longer time than normal after injury or surgery. They may also have spontaneous bleeding in muscles, joints and organs, which can be life-threatening. These bleeding episodes are typically managed by either on-demand, episodic treatment or prophylaxis using products containing FVIII or FIX, or a product that mimics a factor.
Qfitlia does not replace the missing clotting factor. Rather, it reduces the amount of a protein called antithrombin, leading to an increase in thrombin, an enzyme critical for blood clotting.
Qfitlia has a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some patients requiring gallbladder removal). Qfitlia also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least 6 months after initiating treatment with Qfitlia or after a dose increase of Qfitlia.
The most common side effects of Qfitlia are viral infection, common cold symptoms (nasopharyngitis), and bacterial infection.
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