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
Disease / condition
Treatment of Canavan disease
Date of first decision
EU designation number

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

Raremoon Consulting Esp S.L. 
Ronda De Sant Pere 33 Ppal 3 Y 4 
08010 Barcelona 
Tel. +34 518 88 03 22

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.

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