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
Treatment of phenylalanine hydroxylase deficiency
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|August 2023||Please note that this product was withdrawn from the Union Register of orphan medicinal products in August 2023 on request of the Sponsor.|
|October 2022||The sponsorship was transferred from Diamond Pharma Services Ireland Limited, Ireland to Propharma Group The Netherlands B.V., Netherlands.|
|June 2021||The sponsorship was transferred to Diamond Pharma Services Ireland Limited, Ireland.|
Documents related to this orphan designation evaluation
EU/3/18/2109: Public summary of opinion on orphan designation: Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase for the treatment of phenylalanine hydroxylase deficiency (PDF/198.35 KB)
First published: 08/03/2019
Last updated: 08/08/2023
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: