EU/3/19/2229: Orphan designation for the treatment of phenylalanine hydroxylase deficiency
Adeno-associated virus vector encoding human phenylalanine hydroxylase
Table of contents
Overview
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.
Key facts
Active substance |
Adeno-associated virus vector encoding human phenylalanine hydroxylase
|
Intended use |
Treatment of phenylalanine hydroxylase deficiency
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/19/2229
|
Date of designation |
16/12/2019
|
Sponsor |
BioMarin International Limited |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Patients' organisations
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: