On 16 December 2019, orphan designation EU/3/19/2229 was granted by the European Commission to BioMarin International Limited, Ireland, for adeno-associated virus vector encoding human phenylalanine hydroxylase (also known as BMN 307) for the treatment of phenylalanine hydroxylase deficiency.
Adeno-associated virus vector encoding human phenylalanine hydroxylase
|Disease / condition||
Treatment of phenylalanine hydroxylase deficiency
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.