{"id":"fitusiran-sar439774","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Elevated liver enzymes (transaminases)"},{"rate":null,"effect":"Thrombotic events"},{"rate":null,"effect":"Headache"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Fitusiran uses small interfering RNA (siRNA) chemistry to target and degrade antithrombin mRNA in hepatocytes, leading to decreased antithrombin protein levels. By reducing this natural anticoagulant, the drug compensates for deficient clotting factors in hemophilia patients, allowing residual factor activity to generate sufficient thrombin for hemostasis. This mechanism enables prophylaxis against bleeding in both hemophilia A and B, including patients with inhibitors.","oneSentence":"Fitusiran is an RNA interference (RNAi) therapeutic that silences antithrombin (AT) messenger RNA to reduce antithrombin levels and enhance the intrinsic coagulation pathway, thereby increasing thrombin generation in patients with hemophilia.","_ai_confidence":"high"},"_scrapedAt":"2026-03-27T23:46:31.740Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Hemophilia A prophylaxis (including patients with inhibitors)"},{"name":"Hemophilia B prophylaxis (including patients with inhibitors)"}]},"trialDetails":[{"nctId":"NCT07285460","phase":"PHASE3","title":"A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B","status":"RECRUITING","sponsor":"Sanofi","startDate":"2025-12-18","conditions":"Hemophilia","enrollment":85},{"nctId":"NCT06145373","phase":"PHASE4","title":"A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab","status":"RECRUITING","sponsor":"Sanofi","startDate":"2024-03-01","conditions":"Hemophilia A","enrollment":20},{"nctId":"NCT03974113","phase":"PHASE3","title":"Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B","status":"ACTIVE_NOT_RECRUITING","sponsor":"Genzyme, a Sanofi Company","startDate":"2020-01-28","conditions":"Hemophilia","enrollment":32},{"nctId":"NCT02554773","phase":"PHASE1, PHASE2","title":"An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2015-09-18","conditions":"Hemophilia A, Hemophilia B","enrollment":34}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":0,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["SAR439774, Qfitlia®"],"phase":"marketed","status":"active","brandName":"Fitusiran (SAR439774)","genericName":"Fitusiran (SAR439774)","companyName":"Genzyme, a Sanofi Company","companyId":"genzyme-a-sanofi-company","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Fitusiran is an RNA interference (RNAi) therapeutic that silences antithrombin (AT) messenger RNA to reduce antithrombin levels and enhance the intrinsic coagulation pathway, thereby increasing thrombin generation in patients with hemophilia. Used for Hemophilia A prophylaxis (including patients with inhibitors), Hemophilia B prophylaxis (including patients with inhibitors).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}