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
Treatment of phenylalanine hydroxylase deficiency
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Review of designation
Documents related to this orphan designation evaluation
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.
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: