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](/en/medicines/human/orphan-designations/eu-3-15-1509). Zolgensma contains the active substance onasemnogene abeparvovec.
Therapeutic Indication
### 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.
Therapeutic Area (MeSH)
ATC Code
M09AX09
ATC Item
onasemnogene abeparvovec
Pharmacotherapeutic Group
Other drugs for disorders of the musculo-skeletal system
Active Substance (Summary)
INN / Common Names
| Substance | CAS | Monograph |
|---|---|---|
| onasemnogene abeparvovec | N/A | Onasemnogene abeparvovec |
EMA Name
Zolgensma
Medicine Name
Zolgensma
Aliases
N/ANo risk management plan link.
