EU/3/09/708: Orphan designation for the treatment of hyperphenylalaninaemia

Pegylated recombinant phenylalanine ammonia lyase (pegvaliase)


This medicine is now known as pegvaliase.

On 28 January 2010, orphan designation (EU/3/09/708) was granted by the European Commission to BioMarin Europe Ltd, United Kingdom, for pegylated recombinant phenylalanine ammonia lyase for the treatment of hyperphenylalaninaemia.

The sponsorship was transferred to BioMarin International Limited, Ireland, in August 2016.

Pegylated recombinant phenylalanine ammonia lyase has been authorised in the EU as Palynziq since 3 May 2019.

Key facts

Active substance
Pegylated recombinant phenylalanine ammonia lyase (pegvaliase)
Intended use
Treatment of hyperphenylalaninaemia
Orphan designation status
EU designation number
Date of designation

BioMarin International Limited
County Cork P43 R298
Tel. +353 1479430

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Palynziq at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the orphan medicine assessment report PDF icon Palynziq : Orphan maintenance assessment report (initial authorisation) .

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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