EU/3/18/2109:

Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase

Overview

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.

 

Key facts

Active substance
Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase
Intented use
Treatment of phenylalanine hydroxylase deficiency
Date of designation
14/12/2018
Orphan designation status
Positive
EU designation number
EU/3/18/2109

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

Diamond Pharma Services Ireland Limited
Coliemore House
Coliemore Road
Dalkey
Co. Dublin
Ireland
Tel. +353 86067 3082
E-mail: frugiero@diamondpharmaservices.com

Patients' organisations

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.

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