Itvisma is a gene therapy medicine for treating spinal muscular atrophy (SMA), a serious condition of the nerve cells (neurons) that causes muscle wasting and weakness, in people from 2 years of age with inherited mutations (changes) in the SMN1 gene. SMA is rare, and Itvisma was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 19 June 2015\. Itvisma contains the active substance onasemnogene abeparvovec.
Therapeutic Indication
Itvisma is indicated for the treatment of 5q spinal muscular atrophy (SMA) with a bi\-allelic mutation in the SMN1 gene in patients 2 years of age and older.
Therapeutic Area (MeSH)
ATC Code
M09AX09
ATC Item
N/A
Pharmacotherapeutic Group
Other drugs for disorders of the musculo-skeletal system
Active Substance (Summary)
INN / Common Names
EMA Name
Itvisma
Medicine Name
Itvisma
Aliases
N/ANo risk management plan link.
