{"id":"fazirsiran-injection","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Transient liver enzyme elevations"},{"rate":null,"effect":"Platelet count changes"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Fazirsiran uses small interfering RNA (siRNA) technology to target and degrade ALAS2 messenger RNA, thereby decreasing aminolevulinic acid synthase 2 expression. This reduces heme production, which in turn decreases iron absorption and accumulation in tissues. The mechanism is designed to address the underlying pathophysiology of iron overload conditions by modulating the heme synthesis pathway.","oneSentence":"Fazirsiran is an RNA interference (RNAi) therapeutic that silences the ALAS2 gene to reduce heme synthesis and iron accumulation in patients with iron overload disorders.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:12:18.141Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Transfusion-dependent beta-thalassemia"},{"name":"Iron overload disorders"}]},"trialDetails":[{"nctId":"NCT05891158","phase":"PHASE1","title":"A Study About Fazirsiran in People With and Without Liver Problems","status":"COMPLETED","sponsor":"Takeda","startDate":"2023-10-05","conditions":"Hepatic Impairment","enrollment":34},{"nctId":"NCT06165341","phase":"PHASE3","title":"Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)","status":"RECRUITING","sponsor":"Takeda","startDate":"2024-03-01","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":50},{"nctId":"NCT05677971","phase":"PHASE3","title":"Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein","status":"RECRUITING","sponsor":"Takeda","startDate":"2023-03-06","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":160},{"nctId":"NCT03945292","phase":"PHASE2","title":"Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)","status":"COMPLETED","sponsor":"Arrowhead Pharmaceuticals","startDate":"2019-08-07","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":40},{"nctId":"NCT03946449","phase":"PHASE2","title":"Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)","status":"COMPLETED","sponsor":"Arrowhead Pharmaceuticals","startDate":"2019-10-31","conditions":"Alpha 1-Antitrypsin Deficiency","enrollment":16},{"nctId":"NCT05899673","phase":"PHASE3","title":"An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease","status":"ACTIVE_NOT_RECRUITING","sponsor":"Takeda","startDate":"2023-08-08","conditions":"Alpha1-Antitrypsin Deficiency","enrollment":31}],"_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":["TAK-999, ARO-AAT, ADS-001","TAK-999","ARO-AAT","ADS-001"],"phase":"phase_3","status":"active","brandName":"Fazirsiran Injection","genericName":"Fazirsiran Injection","companyName":"Takeda","companyId":"takeda","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Fazirsiran is an RNA interference (RNAi) therapeutic that silences the ALAS2 gene to reduce heme synthesis and iron accumulation in patients with iron overload disorders. Used for Transfusion-dependent beta-thalassemia, Iron overload disorders.","enrichmentLevel":3,"visitCount":1,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}