EU/3/19/2229: Orphan designation for the treatment of phenylalanine hydroxylase deficiency

Adeno-associated virus vector encoding human phenylalanine hydroxylase

Overview

On 16 December 2019, orphan designation EU/3/19/2229 was granted by the European Commission to BioMarin International Limited, Ireland, for adeno-associated virus vector encoding human phenylalanine hydroxylase (also known as BMN 307) for the treatment of phenylalanine hydroxylase deficiency.

Key facts

Active substance
Adeno-associated virus vector encoding human phenylalanine hydroxylase
Intended use
Treatment of phenylalanine hydroxylase deficiency
Orphan designation status
Positive
EU designation number
EU/3/19/2229
Date of designation
16/12/2019
Sponsor

BioMarin International Limited
Shanbally
Ringaskiddy, Co. Cork
Ireland
Tel. +353 1 479 430
E-mail: shanbally@bmrn.com

Review of designation

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

Documents related to this orphan designation evaluation

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.

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:

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