{"id":"pegvaliase-pqpz","safety":{"commonSideEffects":[{"rate":null,"effect":"Injection site reactions"},{"rate":null,"effect":"Anti-drug antibodies/immunogenicity"},{"rate":null,"effect":"Arthralgia"},{"rate":null,"effect":"Headache"}]},"_chembl":null,"_dailymed":{"setId":"6dba844a-db02-44f8-8593-ce497ed9406c","title":"PALYNZIQ (PEGVALIASE-PQPZ) INJECTION, SOLUTION [BIOMARIN PHARMACEUTICAL INC.]"},"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"The enzyme phenylalanine ammonia lyase (PAL) converts the amino acid phenylalanine into trans-cinnamic acid and ammonia, effectively lowering circulating phenylalanine concentrations. Pegylation extends the drug's half-life and reduces immunogenicity. This mechanism addresses the underlying metabolic defect in phenylketonuria by providing an alternative pathway for phenylalanine catabolism.","oneSentence":"Pegvaliase-pqpz is a pegylated phenylalanine ammonia lyase that catalytically degrades phenylalanine to reduce blood phenylalanine levels in patients with phenylketonuria.","_ai_confidence":"medium"},"_scrapedAt":"2026-03-28T04:01:51.739Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L"}]},"trialDetails":[{"nctId":"NCT05813678","phase":"","title":"A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)","status":"RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2022-06-06","conditions":"Phenylketonuria (PKU)","enrollment":450},{"nctId":"NCT05579548","phase":"","title":"A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding","status":"RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2022-11-22","conditions":"Phenylketonuria, Maternal","enrollment":50},{"nctId":"NCT06305234","phase":"","title":"A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)","status":"RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2024-01-20","conditions":"Phenylketonuria (PKU)","enrollment":200},{"nctId":"NCT07477691","phase":"PHASE4","title":"Immune Modulation During Palynziq® Treatment in Adults (IMPALA)","status":"NOT_YET_RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2026-03-31","conditions":"Phenylketonuria","enrollment":12},{"nctId":"NCT04404530","phase":"","title":"Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)","status":"RECRUITING","sponsor":"Emory University","startDate":"2019-10-08","conditions":"Phenylketonurias","enrollment":45},{"nctId":"NCT05356377","phase":"","title":"Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU","status":"COMPLETED","sponsor":"University of Missouri-Columbia","startDate":"2022-05-25","conditions":"Phenylketonurias","enrollment":6},{"nctId":"NCT06780332","phase":"PHASE4","title":"Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq","status":"RECRUITING","sponsor":"BioMarin Pharmaceutical","startDate":"2025-01","conditions":"Phenylketonuria, PKU","enrollment":10},{"nctId":"NCT03792451","phase":"","title":"Nutrition Status of Adults Treated With Pegvaliase","status":"COMPLETED","sponsor":"Boston Children's Hospital","startDate":"2019-01-17","conditions":"Phenylketonurias","enrollment":18}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":5,"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":"Pegvaliase-Pqpz","genericName":"Pegvaliase-Pqpz","companyName":"University of Missouri-Columbia","companyId":"university-of-missouri-columbia","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Pegvaliase-pqpz is a pegylated phenylalanine ammonia lyase that catalytically degrades phenylalanine to reduce blood phenylalanine levels in patients with phenylketonuria. Used for Phenylketonuria (PKU) in patients with blood phenylalanine levels ≥600 μmol/L.","enrichmentLevel":3,"visitCount":0,"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}