EU/3/14/1347: Orphan designation for the treatment of pyridoxamine 5'-phosphate oxidase deficiency

Pyridoxal 5'-phosphate


On 15 October 2014, orphan designation (EU/3/14/1347) was granted by the European Commission to Great Ormond Street Hospital Foundation Trust, United Kingdom, for pyridoxal 5'-phosphate for the treatment of pyridoxamine 5'-phosphate oxidase deficiency.

Key facts

Active substance
Pyridoxal 5'-phosphate
Intended use
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Orphan designation status
EU designation number
Date of designation
Great Ormond Street Hospital for Children, NHS Foundation Trust
Great Ormond Street
London WC1N 3JH
United Kingdom
Tel. +44 (0)20 7405 9200

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

Patients' organisations

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

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

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

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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