{"id":"cael-101","safety":{"commonSideEffects":[{"rate":null,"effect":"Peripheral edema"},{"rate":null,"effect":"Dyspnea"},{"rate":null,"effect":"Fatigue"},{"rate":null,"effect":"Diarrhea"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"CAEL-101 works by directly binding to transthyretin tetramers and stabilizing their native conformation, which prevents dissociation into monomers that would otherwise misfold and aggregate into amyloid fibrils. This mechanism halts the progression of transthyretin amyloidosis by reducing the formation of toxic amyloid deposits that damage tissues, particularly the heart and peripheral nerves. By stabilizing the protein rather than clearing existing amyloid, it addresses the root cause of disease progression.","oneSentence":"CAEL-101 is a transthyretin (TTR) stabilizer that binds to and stabilizes the transthyretin protein, preventing its misfolding and amyloid fibril formation.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:42:02.235Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Transthyretin cardiac amyloidosis (ATTR-CM)"},{"name":"Hereditary transthyretin amyloidosis (hATTR)"}]},"trialDetails":[{"nctId":"NCT04512235","phase":"PHASE3","title":"A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Alexion Pharmaceuticals, Inc.","startDate":"2020-11-03","conditions":"AL Amyloidosis","enrollment":281},{"nctId":"NCT04504825","phase":"PHASE3","title":"A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)","status":"ACTIVE_NOT_RECRUITING","sponsor":"Alexion Pharmaceuticals, Inc.","startDate":"2020-08-25","conditions":"AL Amyloidosis","enrollment":125},{"nctId":"NCT04304144","phase":"PHASE2","title":"A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis","status":"COMPLETED","sponsor":"Alexion Pharmaceuticals, Inc.","startDate":"2020-03-18","conditions":"AL Amyloidosis","enrollment":25}],"_emaApprovals":[],"_faersSignals":[{"count":1,"reaction":"ACUTE KIDNEY INJURY"},{"count":1,"reaction":"CHEST DISCOMFORT"},{"count":1,"reaction":"COUGH"},{"count":1,"reaction":"DIARRHOEA"},{"count":1,"reaction":"DIZZINESS"},{"count":1,"reaction":"DYSPNOEA"},{"count":1,"reaction":"HYPERGLYCAEMIA"},{"count":1,"reaction":"LEUKOCYTOSIS"},{"count":1,"reaction":"OEDEMA"}],"_approvalHistory":[],"publicationCount":9,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Anselamimab"],"phase":"phase_3","status":"active","brandName":"CAEL-101","genericName":"CAEL-101","companyName":"Alexion Pharmaceuticals, Inc.","companyId":"alexion-pharmaceuticals-inc","modality":"Small molecule","firstApprovalDate":"","aiSummary":"CAEL-101 is a transthyretin (TTR) stabilizer that binds to and stabilizes the transthyretin protein, preventing its misfolding and amyloid fibril formation. Used for Transthyretin cardiac amyloidosis (ATTR-CM), Hereditary transthyretin amyloidosis (hATTR).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":3,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}