{"id":"ataluren-ptc124","safety":{"commonSideEffects":[{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Diarrhea"},{"rate":null,"effect":"Nausea"},{"rate":null,"effect":"Upper respiratory tract infection"}]},"_chembl":{"chemblId":"CHEMBL256997","moleculeType":"Small molecule","molecularWeight":"284.25"},"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Ataluren is a small-molecule nonsense suppression agent that binds to the ribosome and enables translational read-through of premature termination codons (PTCs) caused by nonsense mutations. By allowing the ribosome to insert an amino acid at the stop codon position instead of terminating translation, the drug permits synthesis of full-length, partially functional protein. This mechanism is particularly relevant for genetic diseases caused by nonsense mutations, where the underlying defect is a single point mutation creating a premature stop signal.","oneSentence":"Ataluren allows ribosomes to read through premature stop codons (nonsense mutations) and produce full-length, functional proteins.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T01:02:45.216Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Duchenne muscular dystrophy (DMD) with nonsense mutations"},{"name":"Cystic fibrosis with nonsense mutations"},{"name":"Hemophilia B Leyden"}]},"trialDetails":[{"nctId":"NCT01557400","phase":"PHASE3","title":"Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2012-05-20","conditions":"Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy","enrollment":94},{"nctId":"NCT01140451","phase":"PHASE3","title":"Extension Study of Ataluren (PTC124) in Cystic Fibrosis","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2010-08-12","conditions":"Cystic Fibrosis","enrollment":191},{"nctId":"NCT02819557","phase":"PHASE2","title":"Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2016-06-09","conditions":"Duchenne Muscular Dystrophy","enrollment":14},{"nctId":"NCT01141075","phase":"PHASE2","title":"Ataluren for Nonsense Mutation Methylmalonic Acidemia","status":"TERMINATED","sponsor":"PTC Therapeutics","startDate":"2010-07-19","conditions":"Amino Acid Metabolism, Inborn Errors","enrollment":11},{"nctId":"NCT02139306","phase":"PHASE3","title":"Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2014-08","conditions":"Cystic Fibrosis","enrollment":279},{"nctId":"NCT02409004","phase":"PHASE1","title":"Effects of Rifampin on the Pharmacokinetics of Ataluren","status":"COMPLETED","sponsor":"PTC Therapeutics","startDate":"2015-02","conditions":"Healthy","enrollment":15}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":2,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":[],"phase":"phase_3","status":"active","brandName":"Ataluren (PTC124®)","genericName":"Ataluren (PTC124®)","companyName":"PTC Therapeutics","companyId":"ptc-therapeutics","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Ataluren allows ribosomes to read through premature stop codons (nonsense mutations) and produce full-length, functional proteins. Used for Duchenne muscular dystrophy (DMD) with nonsense mutations, Cystic fibrosis with nonsense mutations, Hemophilia B Leyden.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}