EU/3/20/2334

About

On 19 October 2020, orphan designation EU/3/20/2334 was granted by the European Commission to Real Regulatory Limited, Ireland, for adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene (also known as NGN-AGA) for the treatment of aspartylglucosaminuria.

Key facts

Active substance
Adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene
Disease / condition
Treatment of aspartylglucosaminuria
Date of first decision
19/10/2020
Outcome
Positive
EU designation number
EU/3/20/2334

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

Real Regulatory Limited
Unit 9A, Plato Business Park
Damastown
Dublin 15 D15 PA4C
Ireland
Tel: +35318851710
E-mail: Info@realregulatory.com

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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