Fitusiran: First Approval

Declarations

Funding The preparation of this review was not supported by any external funding.

Authorship and Conflict of interest During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Arnold Lee is a contracted employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.

Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability Not applicable.

Additional information about this Adis Drug Review can be found here. 

  

Abstract 

Haemophilia A and B are inherited bleeding disorders caused by mutations in clotting factors. Small interfering RNA therapies may be utilised to restore coagulation by preventing the synthesis of antithrombin. Fitusiran (QFITLIA™) is a small interfering RNA therapy that targets antithrombin, which is being developed by Sanofi for the prophylactic management of haemophilia A and B with or without inhibitors. Fitusiran has the potential to be administered less frequently than other prophylactic treatments for haemophilia. This article summarizes the milestones in the development of fitusiran leading to this first approval for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients aged 12 years and older with haemophilia A or B with or without factor VIII or IX inhibitors.

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