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.

Key facts

Active substance
Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase
Disease / condition
Treatment of phenylalanine hydroxylase deficiency
Date of first decision
EU designation number

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

Yes Pharmaceutical Development Services GmbH
Bahnstraβe 42-46
61381 Friedrichsdorf
Tel. +4961727646465

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;

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