{"disease":{"id":"phenylketonuria-pku","name":"phenylketonuria pku"},"drugs":{"marketed":[{"drug_id":"pegvaliase","indication_name":"Phenylketonuria (PKU) Management","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":null,"generic_name":"PEGVALIASE","company_name":"","drug_phase":"marketed","molecular_target":"phenylalanine","drug_class":"Phenylalanine Metabolizing Enzyme [EPC]","quality_score":10,"revenue":null,"mechanism":"Pegvaliase-pqpz converts phenylalanine to ammonia and trans-cinnamic acid, reducing blood phenylalanine levels in PKU patients."},{"drug_id":"sepiapterin","indication_name":"sepiapterin-responsive phenylketonuria (PKU)","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Sephience","generic_name":"SEPIAPTERIN","company_name":"Ptc Therap","drug_phase":"marketed","molecular_target":"","drug_class":"","quality_score":46,"revenue":null,"mechanism":"Sephience works by replenishing tetrahydrobiopterin (BH4), a critical cofactor for amino acid metabolism."}],"pipeline":[],"offLabel":[],"totalMarketed":2,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT05813678","title":"A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)","phase":"","overall_status":"RECRUITING","enrollment_count":450,"lead_sponsor_name":"BioMarin Pharmaceutical","has_results":false},{"nct_id":"NCT01889862","title":"Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":215,"lead_sponsor_name":"BioMarin Pharmaceutical","has_results":true},{"nct_id":"NCT06305234","title":"A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)","phase":"","overall_status":"RECRUITING","enrollment_count":200,"lead_sponsor_name":"BioMarin Pharmaceutical","has_results":false},{"nct_id":"NCT06969209","title":"Brain Aging in Phenylketonuria","phase":"","overall_status":"RECRUITING","enrollment_count":90,"lead_sponsor_name":"Insel Gruppe AG, University Hospital Bern","has_results":false},{"nct_id":"NCT06792240","title":"Iron Supplements Effect on b-Phe Levels in PKU Patients During 3 Years Study","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":84,"lead_sponsor_name":"ASST Santi Paolo e Carlo","has_results":false},{"nct_id":"NCT03505125","title":"A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults","phase":"","overall_status":"COMPLETED","enrollment_count":63,"lead_sponsor_name":"BioMarin Pharmaceutical","has_results":false},{"nct_id":"NCT03694353","title":"Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":37,"lead_sponsor_name":"BioMarin Pharmaceutical","has_results":true},{"nct_id":"NCT04086511","title":"PANDA: PKU Amino Acid Evaluation","phase":"","overall_status":"COMPLETED","enrollment_count":37,"lead_sponsor_name":"Nutricia Research","has_results":false},{"nct_id":"NCT07406009","title":"The Psychosocial Functioning of Adults With Phenylketonuria.","phase":"","overall_status":"NOT_YET_RECRUITING","enrollment_count":30,"lead_sponsor_name":"Central Hospital, Nancy, France","has_results":false},{"nct_id":"NCT05051657","title":"Evaluation of the Express Plus Range","phase":"NA","overall_status":"COMPLETED","enrollment_count":28,"lead_sponsor_name":"Vitaflo International, Ltd","has_results":false},{"nct_id":"NCT03309345","title":"Body Composition, Energy Intake and Expenditure in People With Phenylketonuria","phase":"","overall_status":"UNKNOWN","enrollment_count":26,"lead_sponsor_name":"University of Glasgow","has_results":false},{"nct_id":"NCT06940193","title":"A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":20,"lead_sponsor_name":"Egoo Health Aps","has_results":false},{"nct_id":"NCT07255599","title":"Evaluation of BH4 Responsiveness in Our PKU Patients","phase":"PHASE1","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":20,"lead_sponsor_name":"Sohag University","has_results":false},{"nct_id":"NCT04679467","title":"Evaluation of PKU Sphere in Italy","phase":"NA","overall_status":"COMPLETED","enrollment_count":13,"lead_sponsor_name":"Vitaflo International, Ltd","has_results":false},{"nct_id":"NCT06694662","title":"PKU EASY Microtabs Plus - Acceptability and Tolerance","phase":"NA","overall_status":"COMPLETED","enrollment_count":10,"lead_sponsor_name":"Galen Limited","has_results":false},{"nct_id":"NCT07318909","title":"To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria","phase":"EARLY_PHASE1","overall_status":"NOT_YET_RECRUITING","enrollment_count":9,"lead_sponsor_name":"Gritgen Therapeutics Co., Ltd.","has_results":false},{"nct_id":"NCT04768348","title":"Natural History Clinical Study in Adult PKU","phase":"","overall_status":"TERMINATED","enrollment_count":7,"lead_sponsor_name":"Homology Medicines, Inc","has_results":false}],"total":17},"guidelines":[],"source":"Drug Landscape verified database"}