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.
Adeno-associated viral vector serotype 9 containing the human SMN gene has been authorised in the EU as Zolgensma since 18 May 2020.
Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec)
|Disease / condition||
Treatment of spinal muscular atrophy
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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.