EU/3/20/2287: Orphan designation for the treatment of Canavan disease
Adeno-associated virus serotype 9 containing the human ASPA gene
Table of contents
Overview
On 4 June 2020, orphan designation EU/3/20/2287 was granted by the European Commission to Raremoon Consulting Limited, United Kingdom, for adeno-associated virus serotype 9 containing the human ASPA gene (also known as BBP-812) for the treatment of Canavan disease.
The sponsorship was transferred to Raremoon Consulting Esp S.L., Spain, in March 2021.
Key facts
Active substance |
Adeno-associated virus serotype 9 containing the human ASPA gene
|
Intended use |
Treatment of Canavan disease
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/20/2287
|
Date of designation |
04/06/2020
|
Sponsor |
Raremoon Consulting Esp S.L. |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: