Palynziq is a medicine that is used to treat phenylketonuria (PKU) in adults and adolescents from 16 years of age. Patients with this genetic disease cannot process the amino acid phenylalanine from dietary protein, and as a result the amino acid builds up in the blood to abnormally high levels, causing problems in the nervous system. Palynziq is used in patients whose blood levels of phenylalanine have not been adequately controlled with other treatments. Palynziq was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 28 January 2010. Further information on the orphan designation can be found [EU/3/09/708](/en/medicines/human/orphan-designations/eu-3-09-708). Palynziq contains the active substance pegvaliase.
Therapeutic Indication
### Therapeutic indication Palynziq is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options.
Therapeutic Area (MeSH)
ATC Code
A16AB19
ATC Item
pegvaliase
Pharmacotherapeutic Group
Other alimentary tract and metabolism products
Active Substance (Summary)
INN / Common Names
EMA Name
Palynziq
Medicine Name
Palynziq
Aliases
N/A