{"id":"ivacaftor-and-symdeko","safety":{"commonSideEffects":[{"rate":"5-10","effect":"Elevated transaminases (ALT/AST)"},{"rate":"10-15","effect":"Headache"},{"rate":"5-10","effect":"Nausea"},{"rate":"5-10","effect":"Diarrhea"},{"rate":"5-10","effect":"Rash"}]},"_chembl":{"chemblId":"CHEMBL2010601","moleculeType":"Small molecule","molecularWeight":"392.50"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Ivacaftor works by binding to and stabilizing the open state of CFTR channels, allowing increased chloride ion transport across cell membranes. Lumacaftor acts as a corrector by assisting in proper folding and trafficking of CFTR protein to the cell membrane. Together, these agents restore CFTR function in cystic fibrosis patients, improving mucociliary clearance and reducing lung inflammation.","oneSentence":"Ivacaftor is a CFTR potentiator that increases the opening probability of the cystic fibrosis transmembrane conductance regulator (CFTR) channel, while Symdeko combines ivacaftor with lumacaftor, a CFTR corrector that helps misfolded CFTR protein reach the cell surface.","_ai_confidence":"high"},"_scrapedAt":"2026-03-27T23:50:37.335Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Cystic fibrosis in patients with specific CFTR mutations (F508del and other gating mutations for ivacaftor; F508del-mediated CF for Symdeko)"}]},"trialDetails":[{"nctId":"NCT03624101","phase":"PHASE4","title":"Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations","status":"TERMINATED","sponsor":"University of Alabama at Birmingham","startDate":"2018-12-01","conditions":"Cystic Fibrosis","enrollment":1},{"nctId":"NCT04215796","phase":"NA","title":"Evaluation of the ReX-C System, in Patients Receiving CFTR Modulators for the Treatment of Cystic Fibrosis (CF).","status":"UNKNOWN","sponsor":"Dosentrx Ltd.","startDate":"2020-05-12","conditions":"Cystic Fibrosis, Adherence, Medication","enrollment":10}],"_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":[],"phase":"marketed","status":"active","brandName":"Ivacaftor and Symdeko","genericName":"Ivacaftor and Symdeko","companyName":"University of Alabama at Birmingham","companyId":"university-of-alabama-at-birmingham","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Ivacaftor is a CFTR potentiator that increases the opening probability of the cystic fibrosis transmembrane conductance regulator (CFTR) channel, while Symdeko combines ivacaftor with lumacaftor, a CFTR corrector that helps misfolded CFTR protein reach the cell surface. Used for Cystic fibrosis in patients with specific CFTR mutations (F508del and other gating mutations for ivacaftor; F508del-mediated CF for Symdeko).","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}