On 19 June 2015, orphan designation (EU/3/15/1509) was granted by the European Commission to AveXis EU, Ltd, Ireland, for adeno-associated viral vector serotype 9 containing the human SMN gene for the treatment of spinal muscular atrophy.

The sponsorship was transferred to Avexis Netherlands B.V., The Netherlands, in September 2018.

The sponsorship was transferred to AveXis EU Limited, Ireland, in September 2019.

Key facts

Active substance
Adeno-associated viral vector serotype 9 containing the human SMN gene
Medicine name
Disease / condition
Treatment of spinal muscular atrophy
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

AveXis EU Limited 
Block B, The Crescent Building
Northwood Crescent
Dublin 9
Co. Dublin D09 C6X8

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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