{"id":"eliglustat-gz385660","safety":{"commonSideEffects":[{"rate":null,"effect":"Diarrhea"},{"rate":null,"effect":"Tremor"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Fatigue"}]},"_chembl":{"chemblId":"CHEMBL2110588","moleculeType":"Small molecule","molecularWeight":"404.55"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Eliglustat works by inhibiting glucosylceramide synthase, the enzyme responsible for synthesizing glucosylceramide from glucose and ceramide. By blocking this enzyme, the drug reduces the accumulation of glucosylceramide in macrophages and other cells affected in Gaucher disease, thereby reducing organomegaly and other disease manifestations. This substrate reduction therapy approach addresses the underlying pathophysiology of lysosomal storage disorders.","oneSentence":"Eliglustat is a glucosylceramide synthase (GCS) inhibitor that reduces the production of glucosylceramide, a lipid that accumulates in Gaucher disease.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T00:46:10.716Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Gaucher disease type 1"}]},"trialDetails":[{"nctId":"NCT03485677","phase":"PHASE3","title":"Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3","status":"COMPLETED","sponsor":"Sanofi","startDate":"2018-04-11","conditions":"Gaucher's Disease Type I, Gaucher's Disease Type III","enrollment":57},{"nctId":"NCT06188325","phase":"PHASE1","title":"A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers","status":"COMPLETED","sponsor":"Sanofi","startDate":"2018-01-01","conditions":"Gaucher's Disease","enrollment":18},{"nctId":"NCT06193304","phase":"PHASE1","title":"A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth","status":"COMPLETED","sponsor":"Sanofi","startDate":"2014-08-25","conditions":"Gaucher's Disease","enrollment":6},{"nctId":"NCT06143904","phase":"PHASE1","title":"A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants","status":"COMPLETED","sponsor":"Sanofi","startDate":"2009-06-03","conditions":"Gaucher's Disease Type I","enrollment":10},{"nctId":"NCT02536755","phase":"PHASE3","title":"Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2015-10-27","conditions":"Gaucher Disease","enrollment":31},{"nctId":"NCT02536937","phase":"PHASE1","title":"A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate","status":"COMPLETED","sponsor":"Sanofi","startDate":"2015-09","conditions":"Gaucher Disease","enrollment":32},{"nctId":"NCT02536911","phase":"PHASE1","title":"A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2015-09","conditions":"Gaucher Disease","enrollment":24},{"nctId":"NCT02422654","phase":"PHASE1","title":"Taste Evaluation of Different Liquid Formulations With Eliglustat","status":"COMPLETED","sponsor":"Genzyme, a Sanofi Company","startDate":"2015-04","conditions":"Gaucher Disease","enrollment":8}],"_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":["Cerdelga"],"phase":"phase_3","status":"active","brandName":"Eliglustat GZ385660","genericName":"Eliglustat GZ385660","companyName":"Sanofi","companyId":"sanofi","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Eliglustat is a glucosylceramide synthase (GCS) inhibitor that reduces the production of glucosylceramide, a lipid that accumulates in Gaucher disease. Used for Gaucher disease type 1.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}