This medicine is authorised for use in the European Union.


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.

Palynziq contains the active substance pegvaliase.

This EPAR was last updated on 29/05/2019

Authorisation details

Product details
Agency product number
Active substance
International non-proprietary name (INN) or common name
Therapeutic area (MeSH)
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
BioMarin International Limited
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Shanbally, Ringaskiddy
County Cork, P43 R298

Product information

03/05/2019 Palynziq - EMEA/H/C/004744 -


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Pharmacotherapeutic group

Other alimentary tract and metabolism products

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.

Assessment history

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