This medicine is now known as nusinersen.
On 2 April 2012, orphan designation (EU/3/12/976) was granted by the European Commission to Isis USA Ltd, United Kingdom, for antisense oligonucleotide targeted to the SMN2 gene for the treatment of 5q spinal muscular atrophy.
In April 2016, Isis USA Ltd changed name to Ionis USA Ltd.
The sponsorship was transferred to Biogen Idec Ltd, United Kingdom, in August 2016.
Update: Antisense oligonucleotide targeted to the SMN2 gene has been authorised in the EU as Spinraza since 30 May 2017.
The sponsorship was transferred to Biogen Netherlands B.V., The Netherlands, in September 2018.
Antisense oligonucleotide targeted to the SMN2 gene (nusinersen)
|Disease / condition||
Treatment of 5q spinal muscular atrophy
|Date of decision||
|Orphan decision number||
Review of designation
On 25 April 2017, the Committee for Orphan Medicinal Products (COMP) concluded its review of the designation EU/3/12/976 for Spinraza (nusinersen, previously known as antisense oligonucleotide targeted to the SMN2 gene) as an orphan medicinal product for the treatment of 5q spinal muscular atrophy. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. The COMP recommended that the orphan designation of the medicine be maintained1.
1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.
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;