onasemnogene abeparvovec

This medicine is authorised for use in the European Union.


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 genes 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.

Zolgensma contains the active substance onasemnogene abeparvovec.

This EPAR was last updated on 17/08/2021

Authorisation details

Product details
Agency product number
Active substance
onasemnogene abeparvovec
International non-proprietary name (INN) or common name
onasemnogene abeparvovec
Therapeutic area (MeSH)
Muscular Atrophy, Spinal
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

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.

Conditional approvalConditional approval

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.

Publication details
Marketing-authorisation holder
Novartis Gene Therapies EU Limited
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Block B, The Crescent Building Northwood
Dublin 9 D09 C6X8

Product information

12/07/2021 Zolgensma - EMEA/H/C/004750 - IA/0016/G


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Pharmacotherapeutic group

Other drugs for disorders of the musculo-skeletal system

Therapeutic indication

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.

Assessment history

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