Phenylketonuria (PKU)

Phenylketonuria (PKU) is the indication for 2 drugs in DrugBank. It is covered by 2 curated, structured indications.

Also known as: Phenylketonuria · Phenylketonurias · PKU · Hyperphenylalaninaemia, Type I · PAH Deficiency
Classified under: Congenital, Hereditary, and Neonatal Diseases and Abnormalities
Mental Disorders
Nervous System Diseases
Nutritional and Metabolic Diseases
Urogenital Diseases
Indicated drugs: Pegvaliase
Sepiapterin

Resolves to

Vocabularies

MeSHM0016567ICD-10E70.0SNOMED CT154735006MedDRA10034871UMLSC0031485

What you can answer from here — as of November 01, 2026

2Indicated drugsWith each drug's targets and mechanism

Listed above

2Structured indicationsPopulation, line of therapy, route and combination as fields, not prose

93Clinical trialsPhase, status and sponsor resolved per trial

2Contraindicated drugsDrugs whose labels contraindicate them in this condition

8Drugs reporting it as an adverse effectWith incidence where the label states it

Which approved drugs for Phenylketonuria (PKU) share a target, and which companies have late-stage candidates against it?

Create a free account or log in to explore the full data card.