On 14 December 2018, orphan designation (EU/3/18/2109) was granted by the European Commission to Yes Pharmaceutical Development Services GmbH, Germany, for adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase (also known as HMI-102) for the treatment of phenylalanine hydroxylase deficiency.
Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase
|Disease / condition||
Treatment of phenylalanine hydroxylase deficiency
|Date of decision||
|Orphan decision 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;