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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
Disease / condition
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Date of decision
Orphan decision number

Sponsor's contact details

Medicure Pharma Europe Limited
Block 3 Harcourt Centre
Harcourt Road
Dublin 2
D02 A339 Ireland
Tel. +353 (0)1 449 4489

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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