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.
|Disease / condition||
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
|Date of decision||
|Orphan decision number||
Sponsor's contact details
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.