Palynziq
What Palynziq is used for
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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
Key facts
- Brand
- Palynziq
- Active substance
- pegvaliase
- Disease area
- Phenylketonurias
- Medicine group
- Other alimentary tract and metabolism products
- ATC code
- A16AB19
- Marketing authorisation holder
- BioMarin International Limited
- Authorised in the EU since
- 2019
Official information
In the USA
Same active substance: Palynziq
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Palynziq used for?
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.
What is the active substance in Palynziq?
Palynziq contains pegvaliase.
Who makes Palynziq?
The EU marketing authorisation holder is BioMarin International Limited.
Is Palynziq an orphan medicine?
Yes. Palynziq is designated an orphan medicine in the EU, for a rare disease (Phenylketonurias).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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