{"id":"saproterin-dihydrochloride","safety":{"commonSideEffects":[{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Rhinitis"},{"rate":null,"effect":"Diarrhea"},{"rate":null,"effect":"Pharyngitis"}]},"_chembl":null,"_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Saproterin (also known as BH4 or tetrahydrobiopterin) is an essential cofactor for the enzyme phenylalanine ammonia-lyase, which catalyzes the conversion of phenylalanine to tyrosine. In phenylketonuria (PKU), patients have deficient or dysfunctional PAL enzyme; saproterin supplementation can restore or enhance residual enzyme activity, thereby lowering toxic phenylalanine accumulation. This mechanism is distinct from dietary phenylalanine restriction and allows some PKU patients to achieve better metabolic control and dietary flexibility.","oneSentence":"Saproterin dihydrochloride is a synthetic form of tetrahydrofolate that acts as a cofactor to enhance phenylalanine ammonia-lyase (PAL) enzyme activity, reducing blood phenylalanine levels in phenylketonuria.","_ai_confidence":"high"},"_scrapedAt":"2026-03-28T00:06:56.874Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Phenylketonuria (PKU) in patients with residual phenylalanine ammonia-lyase activity"}]},"trialDetails":[{"nctId":"NCT02677870","phase":"PHASE4","title":"The Effectiveness of Kuvan in Amish PKU Patients","status":"COMPLETED","sponsor":"University Hospitals Cleveland Medical Center","startDate":"2018-01-22","conditions":"Phenylketonuria","enrollment":7}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":4,"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Kuvan treatment"],"phase":"marketed","status":"active","brandName":"saproterin dihydrochloride","genericName":"saproterin dihydrochloride","companyName":"University Hospitals Cleveland Medical Center","companyId":"university-hospitals-cleveland-medical-center","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Saproterin dihydrochloride is a synthetic form of tetrahydrofolate that acts as a cofactor to enhance phenylalanine ammonia-lyase (PAL) enzyme activity, reducing blood phenylalanine levels in phenylketonuria. Used for Phenylketonuria (PKU) in patients with residual phenylalanine ammonia-lyase activity.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":1,"withResults":1},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}