Hemophilia: Why FDA approval for this novel treatment is significant

The US Food and Drug Administration has on March 28, approved a novel treatment (fitusiran) 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). | Depositphotos hemophilia

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. | Depositphotos hemophilia

This drug approval is significant for patients with hemophilia because it can be administered less frequently than other existing options. | Depositphotos Hemophilia

Hemophilia A and B 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 | Depositphotos Hemophilia

This new drug 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. | Freepik Hemophilia

The World Federation of Hemophilia (WFH) has announced the new theme "Access for All: Women and Girls Bleed Too" as the central focus for the for 2025 World Hemophilia Day observance. This theme represents a significant step forward in addressing the often-overlooked needs of women and girls with bleeding disorders (WGBDs). Despite advancements in hemophilia treatment and care, WGBDs continue to face challenges in diagnosis, treatment, and overall quality of life. | Depositphotos Hemophilia