On 24 August 2018, orphan designation (EU/3/18/2060) was granted by the European Commission to Biogen Idec Limited, United Kingdom, for adeno-associated viral vector serotype hu68 containing the human SMN1 gene for the treatment of spinal muscular atrophy.
The sponsorship was transferred to Biogen Netherlands B.V. The Netherlands, in May 2019.
The sponsorship was transferred to Nightstar Europa Limited, Ireland, in February 2019 and subsequently to Biogen Netherlands B.V., Netherlands, in September 2020.
This medicine is now known as timrepigene emparvovec.
adeno-associated viral vector serotype 2 containing the human REP1 gene (timrepigene emparvovec)
|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:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.