EU/3/19/2197
Table of contents
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: