Zolgensma
What Zolgensma is used for
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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Advanced therapy: a gene, cell or tissue-engineered medicine.
- PRIME: a priority medicine that the EMA supported early because it may meet an unmet need.
- Conditional approval: approved on less complete data because the need is high; more data must follow.
Key facts
- Brand
- Zolgensma
- Active substance
- onasemnogene abeparvovec
- Disease area
- Muscular Atrophy, Spinal
- Medicine group
- Other drugs for disorders of the musculo-skeletal system
- ATC code
- M09AX09
- Marketing authorisation holder
- Novartis Europharm Limited
- Authorised in the EU since
- 2020
Official information
In the USA
Same active substance: Itvisma
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Zolgensma used for?
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.
What is the active substance in Zolgensma?
Zolgensma contains onasemnogene abeparvovec.
Who makes Zolgensma?
The EU marketing authorisation holder is Novartis Europharm Limited.
Is Zolgensma an orphan medicine?
Yes. Zolgensma is designated an orphan medicine in the EU, for a rare disease (Muscular Atrophy, Spinal).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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