{"id":"cysteamine-bitartrate-delayed-release-capsules","safety":{"commonSideEffects":[{"rate":null,"effect":"Gastrointestinal disturbances (nausea, vomiting, diarrhea)"},{"rate":null,"effect":"Abdominal pain"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Rash"},{"rate":null,"effect":"Fever"}]},"_chembl":{"chemblId":"CHEMBL1200577","moleculeType":"Small molecule","molecularWeight":"227.24"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Cystinosis is a lysosomal storage disorder caused by mutations in the CTNS gene, leading to cystine accumulation in lysosomes and cellular damage. Cysteamine acts as a reducing agent that converts the disulfide cystine into two molecules of cysteine and cysteamine, which can then exit the lysosome via alternative transporters. This mechanism reduces toxic cystine levels and slows progression of organ damage, particularly in the kidneys.","oneSentence":"Cysteamine bitartrate reduces cystine accumulation in lysosomes by converting cystine to cysteine and cysteamine, thereby decreasing intracellular cystine levels.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:08:17.375Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Cystinosis (nephropathic cystinosis)"}]},"trialDetails":[{"nctId":"NCT01733316","phase":"PHASE3","title":"Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis","status":"COMPLETED","sponsor":"Amgen","startDate":"2013-01-31","conditions":"Cystinosis","enrollment":41},{"nctId":"NCT02473445","phase":"PHASE2","title":"A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease","status":"TERMINATED","sponsor":"Amgen","startDate":"2015-05-19","conditions":"Mitochondrial Diseases","enrollment":22},{"nctId":"NCT01744782","phase":"PHASE3","title":"Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis","status":"COMPLETED","sponsor":"Amgen","startDate":"2012-12-20","conditions":"Cystinosis","enrollment":17},{"nctId":"NCT01197378","phase":"PHASE3","title":"Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)","status":"COMPLETED","sponsor":"Amgen","startDate":"2010-08-27","conditions":"Cystinosis","enrollment":60},{"nctId":"NCT02023866","phase":"PHASE2","title":"Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease","status":"COMPLETED","sponsor":"Amgen","startDate":"2014-05","conditions":"Inherited Mitochondrial Disease, Including Leigh Syndrome","enrollment":36},{"nctId":"NCT00872729","phase":"PHASE1, PHASE2","title":"Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis","status":"COMPLETED","sponsor":"Amgen","startDate":"2009-05","conditions":"Cystinosis","enrollment":9},{"nctId":"NCT01000961","phase":"PHASE3","title":"Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis","status":"COMPLETED","sponsor":"Amgen","startDate":"2010-06","conditions":"Cystinosis","enrollment":43},{"nctId":"NCT01529268","phase":"PHASE2, PHASE3","title":"Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children","status":"COMPLETED","sponsor":"National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)","startDate":"2012-06","conditions":"Nonalcoholic Fatty Liver Disease (NAFLD)","enrollment":169}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":8,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["RP103","PROCYSBI®"],"phase":"phase_3","status":"active","brandName":"Cysteamine Bitartrate Delayed-release Capsules","genericName":"Cysteamine Bitartrate Delayed-release Capsules","companyName":"Amgen","companyId":"amgen","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Cysteamine Bitartrate Delayed-release Capsules are used to treat Cystinosis, a condition caused by the accumulation of cystine in the body. They work as a cystine reducing agent, specifically targeting cystine to reduce its levels in the body.","enrichmentLevel":3,"visitCount":0,"regulatoryByCountry":[{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"2013-04-30T00:00:00.000Z","mah":"HORIZON","brand_name_local":"PROCYSBI","application_number":"NDA203389"},{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"2020-02-14T00:00:00.000Z","mah":"HORIZON","brand_name_local":"PROCYSBI","application_number":"NDA213491"}],"trialStats":{"total":1,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}