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. 

Key facts

Active substance
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

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

Sponsor's contact details

Biogen Netherlands B.V.
Prins Mauritslaan 13-19
1171 LP Badhoevedorp
The Netherlands
Tel. + 31 20 542 2140

Patients' organisations

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.

How useful was this page?

Add your rating