Palynziq - opinion on variation to marketing authorisation

Opinion

EMA has issued an opinion on a change to this medicine's authorisation

pegvaliase

Opinion

On 21 May 2026, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Palynziq. The marketing authorisation holder for this medicinal product is Biomarin International Limited.

The CHMP adopted an extension to the existing indication as follows:

Palynziq is indicated for the treatment of patients with phenylketonuria (PKU) aged 16 12 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options.

Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.

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New text in bold, removed text as strikethrough.

CHMP post-authorisation summary of positive opinion for Palynziq (VR/0000302032)

Adopted Reference Number: EMADOC-1700519818-3182005

Key facts

• Name of medicine:
  Palynziq
• EMA product number:
  EMEA/H/C/004744
• Active substance:
  pegvaliase
• International non-proprietary name (INN) or common name:
  pegvaliase
• Therapeutic area (MeSH):
  Phenylketonurias
• Anatomical therapeutical chemical (ATC) code:
  A16AB19
• Orphan:

  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.

• Marketing authorisation holder:
  BioMarin International Limited
• Date of opinion:
  21/05/2026
• Status:
  Positive