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.
In December 2020, AveXis EU Limited changed name to Novartis Gene Therapies EU Limited.
The sponsorship was transferred to Novartis Europharm Limited, Ireland in July 2022.
This medicine is also known as onasemnogene abeparvovec.
Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec)
Treatment of spinal muscular atrophy
|Orphan designation status||
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Review of designation
|July 2022||The sponsorship was transferred to Novartis Europharm Limited, Ireland in July 2022.|
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: