{"id":"napba","safety":{"commonSideEffects":[{"rate":null,"effect":"Nausea"},{"rate":null,"effect":"Vomiting"},{"rate":null,"effect":"Abdominal pain"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Syncope"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"NaPBA is a prodrug that is metabolized to phenylacetate, which conjugates with glutamine via the enzyme phenylacetyl-CoA:glutamine-N-acetyltransferase to form phenylacetylglutamine. This conjugate is readily excreted in the urine, effectively removing excess nitrogen and reducing hyperammonemia. This mechanism bypasses the defective urea cycle, making it useful in patients with urea cycle disorders.","oneSentence":"NaPBA (sodium phenylbutyrate) reduces ammonia levels by providing an alternative pathway for nitrogen excretion through conjugation with glutamine.","_ai_confidence":"high"},"_scrapedAt":"2026-03-27T23:56:36.871Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Urea cycle disorders (hyperammonemia management)"},{"name":"Acute hyperammonemic encephalopathy"}]},"trialDetails":[{"nctId":"NCT01257737","phase":"PHASE4","title":"To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)","status":"COMPLETED","sponsor":"Amgen","startDate":"2010-10-04","conditions":"Urea Cycle Disorders","enrollment":88},{"nctId":"NCT01347073","phase":"PHASE3","title":"Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)","status":"COMPLETED","sponsor":"Amgen","startDate":"2011-07","conditions":"Urea Cycle Disorders","enrollment":23},{"nctId":"NCT00947544","phase":"PHASE2","title":"Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders","status":"COMPLETED","sponsor":"Amgen","startDate":"2010-03","conditions":"Urea Cycle Disorders","enrollment":17},{"nctId":"NCT00551200","phase":"PHASE2","title":"Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders","status":"COMPLETED","sponsor":"Amgen","startDate":"2007-10","conditions":"Urea Cycle Disorders","enrollment":14},{"nctId":"NCT00947297","phase":"PHASE3","title":"Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)","status":"COMPLETED","sponsor":"Amgen","startDate":"2009-11","conditions":"Urea Cycle Disorders","enrollment":60},{"nctId":"NCT00992459","phase":"PHASE3","title":"Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders","status":"COMPLETED","sponsor":"Amgen","startDate":"2009-10","conditions":"Urea Cycle Disorders","enrollment":46},{"nctId":"NCT03335488","phase":"PHASE4","title":"Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)","status":"COMPLETED","sponsor":"Amgen","startDate":"2018-02-20","conditions":"Urea Cycle Disorder","enrollment":16},{"nctId":"NCT00986895","phase":"PHASE1","title":"A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group","status":"COMPLETED","sponsor":"Amgen","startDate":"2006-09","conditions":"Hepatic Encephalopathy, Urea Cycle Disorders","enrollment":32},{"nctId":"NCT00977600","phase":"PHASE1","title":"A Study of Glyceryl Tri-(4-phenylbutyrate) (GT4P)","status":"COMPLETED","sponsor":"Amgen","startDate":"2005-03","conditions":"Healthy","enrollment":24},{"nctId":"NCT00345605","phase":"PHASE2","title":"Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder","status":"COMPLETED","sponsor":"Brendan Lee","startDate":"2008-02","conditions":"Argininosuccinic Aciduria, Amino Acid Metabolism, Inborn Errors, Urea Cycle Disorders","enrollment":12}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":19,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["GT4P, Glyceryl tri-(4-phenylbutyrate)","Sodium phenylbutyrate"],"phase":"marketed","status":"active","brandName":"NaPBA","genericName":"NaPBA","companyName":"Amgen","companyId":"amgen","modality":"Small molecule","firstApprovalDate":"","aiSummary":"NaPBA (sodium phenylbutyrate) reduces ammonia levels by providing an alternative pathway for nitrogen excretion through conjugation with glutamine. Used for Urea cycle disorders (hyperammonemia management), Acute hyperammonemic encephalopathy.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}