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.
Adeno-associated virus serotype 9 containing the human ASPA gene
Treatment of Canavan disease
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Review of designation
Documents related to this orphan designation evaluation
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: