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.
The sponsorship was transferred to Diamond Pharma Services Ireland Limited, Ireland, in June 2021.
Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase
Treatment of phenylalanine hydroxylase deficiency
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Review of designation
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.