Table of contents
Zolgensma is a gene therapy medicine for treating spinal muscular atrophy, a serious condition of the nerves that causes muscle wasting and weakness.
It is intended for patients with inherited mutations affecting a gene known as SMN1, who have either been diagnosed with SMA type 1 (the most severe type) or have up to 3 copies of another gene known as SMN2.
Spinal muscular atrophy is rare, and Zolgensma was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 19 June 2015. Further information on the orphan designation can be found here: ema.europa.eu/medicines/human/orphan-designations/eu3151509.
Zolgensma contains the active substance onasemnogene abeparvovec.
Zolgensma : EPAR - Medicine overview (PDF/122.81 KB) (updated)
First published: 27/05/2020
Last updated: 17/03/2023
Zolgensma : EPAR - Risk-management-plan summary (PDF/76.85 KB) (updated)
First published: 27/05/2020
Last updated: 13/03/2023
|Agency product number||
|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
Muscular Atrophy, Spinal
|Anatomical therapeutic chemical (ATC) code||
This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.
This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.
This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.
Novartis Europharm Limited
|Date of issue of marketing authorisation valid throughout the European Union||
17/05/2022 Zolgensma - EMEA/H/C/004750 - R/0021
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.
Other drugs for disorders of the musculo-skeletal system
Zolgensma is indicated for the treatment of:
- patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or
- patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 23-26 March 2020 (updated)27/03/2020