{"title":"Fitusiran: First Approval.","authors":"Arnold Lee","doi":"10.1007/s40265-025-02203-y","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":""},"PeriodicalIF":13.0000,"publicationDate":"2025-06-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Drugs","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40265-025-02203-y","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0
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.
期刊介绍：
Drugs is a journal that aims to enhance pharmacotherapy by publishing review and original research articles on key aspects of clinical pharmacology and therapeutics. The journal includes:
Leading/current opinion articles providing an overview of contentious or emerging issues.
Definitive reviews of drugs and drug classes, and their place in disease management.
Therapy in Practice articles including recommendations for specific clinical situations.
High-quality, well designed, original clinical research.
Adis Drug Evaluations reviewing the properties and place in therapy of both newer and established drugs.
AdisInsight Reports summarising development at first global approval.
Moreover, the journal offers additional digital features such as animated abstracts, video abstracts, instructional videos, and podcasts to increase visibility and educational value. Plain language summaries accompany articles to assist readers with some knowledge of the field in understanding important medical advances.
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