EU/3/20/2287: Orphan designation for the treatment of Canavan disease

Adeno-associated virus serotype 9 containing the human ASPA gene


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
EU designation number
Date of designation

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

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

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:

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