{"id":"buphenyl-napba","safety":{"commonSideEffects":[{"rate":null,"effect":"Nausea"},{"rate":null,"effect":"Vomiting"},{"rate":null,"effect":"Abdominal pain"},{"rate":null,"effect":"Taste aversion"},{"rate":null,"effect":"Hyperchloremic metabolic acidosis"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"In urea cycle disorders, ammonia accumulates due to enzymatic defects in the urea cycle. Buphenyl bypasses the defective urea cycle by providing a nitrogen-scavenging mechanism: phenylbutyrate is metabolized to phenylacetate, which conjugates with glutamine via phenylacetyl-CoA:glutamine-N-acetyltransferase to form phenylacetylglutamine. This water-soluble conjugate is readily excreted in urine, effectively removing two nitrogen atoms per molecule of phenylacetylglutamine and reducing ammonia levels.","oneSentence":"Buphenyl (sodium phenylbutyrate) provides an alternative pathway for nitrogen excretion by conjugating with glutamine to form phenylacetylglutamine, which is then renally eliminated.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T01:41:57.236Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Urea cycle disorders (hyperammonemia management)"}]},"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":18,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["sodium phenylbutyrate"],"phase":"phase_3","status":"active","brandName":"Buphenyl (NaPBA)","genericName":"Buphenyl (NaPBA)","companyName":"Amgen","companyId":"amgen","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Buphenyl (NaPBA) is a small molecule used to treat various conditions, including Urea Cycle Disorders, Hepatic Encephalopathy, and Argininosuccinic Aciduria, as well as other inborn errors of amino acid metabolism. It is administered as an intervention in clinical trials, often in combination with other treatments.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}