EU/3/19/2197

About

On 21 August 2019, orphan designation EU/3/19/2197 was granted by the European Commission to Amicus Therapeutics Europe Limited, Ireland, for recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene (also known as AT-GTX-501) for the treatment of neuronal ceroid lipofuscinosis.

Key facts

Active substance
Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene
Disease / condition
Treatment of neuronal ceroid lipofuscinosis
Date of first decision
21/08/2019
Outcome
Positive
EU designation number
EU/3/19/2197

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

Amicus Therapeutics Europe Limited
Block 1 Blanchardstown Corporate Park
Ballycoolen Road
Dublin D15 AKK1
Ireland
Tel: +353 1 588 0836
E-mail: info@amicustherapeutics.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

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