{"id":"ivacaftor-ataluren","safety":{"commonSideEffects":[{"rate":null,"effect":"Respiratory tract infection"},{"rate":null,"effect":"Cough"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Nausea"}]},"_chembl":{"chemblId":"CHEMBL2010601","moleculeType":"Small molecule","molecularWeight":"392.50"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Ivacaftor is a small-molecule potentiator that increases the open probability of CFTR channels at the cell surface, allowing more chloride ion transport. Ataluren is a nonsense suppression agent that enables ribosomes to read through premature stop codons, allowing production of full-length, functional CFTR protein in patients with nonsense mutations. Together, they address two distinct molecular defects in cystic fibrosis.","oneSentence":"This combination therapy enhances defective CFTR protein function through two complementary mechanisms: ivacaftor acts as a CFTR potentiator to increase channel opening, while ataluren promotes read-through of nonsense mutations to restore full-length CFTR protein production.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-27T23:50:37.781Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Cystic fibrosis with nonsense mutations (G542X and other nonsense CFTR mutations)"}]},"trialDetails":[{"nctId":"NCT03256968","phase":"PHASE4","title":"PTC Study to Evaluate Ataluren in Combination With Ivacaftor","status":"COMPLETED","sponsor":"University of Alabama at Birmingham","startDate":"2017-01-27","conditions":"Cystic Fibrosis","enrollment":1},{"nctId":"NCT03256799","phase":"PHASE4","title":"Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations","status":"COMPLETED","sponsor":"University of Alabama at Birmingham","startDate":"2017-03-17","conditions":"Cystic Fibrosis","enrollment":1}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":13,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"marketed","status":"active","brandName":"Ivacaftor/Ataluren","genericName":"Ivacaftor/Ataluren","companyName":"University of Alabama at Birmingham","companyId":"university-of-alabama-at-birmingham","modality":"Small molecule","firstApprovalDate":"","aiSummary":"This combination therapy enhances defective CFTR protein function through two complementary mechanisms: ivacaftor acts as a CFTR potentiator to increase channel opening, while ataluren promotes read-through of nonsense mutations to restore full-length CFTR protein production. Used for Cystic fibrosis with nonsense mutations (G542X and other nonsense CFTR mutations).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}