EU/3/18/1983: Orphan designation for the treatment of pyridoxamine 5'-phosphate oxidase deficiency

Pyridoxal 5'-phosphate

Table of contents


On 22 February 2018, orphan designation (EU/3/18/1983) was granted by the European Commission to Medicure Pharma Europe Limited, Ireland, 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
Medicure Pharma Europe Limited
Block 3 Harcourt Centre
Harcourt Road
Dublin 2
D02 A339 Ireland
Tel. +353 (0)1 449 4489

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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