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
Disease / condition
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Date of first decision
EU designation number

Review of designation

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

Sponsor's contact details

Great Ormond Street Hospital for Children, NHS Foundation Trust
Great Ormond Street
London WC1N 3JH
United Kingdom
Tel. +44 (0)20 7405 9200

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.

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