Orphan designation: Adeno-associated viral vector serotype 9 containing the human NPC1 gene Treatment of Niemann-Pick's disease, type C, 13/10/2023 Positive
Key facts
- Key facts
- Active substance
- Adeno-associated viral vector serotype 9 containing the human NPC1 gene
- Intended use
- Treatment of Niemann-Pick's disease, type C
- Orphan designation status
- Positive
- EU designation number
- EU/3/23/2843
- Date of designation
- Sponsor
FGK Representative Service GmbH
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. - 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:
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform: