Evrysdi is a medicine used to treat patients with 5q spinal muscular atrophy (SMA), a genetic disease that causes weakness and wasting of the muscles including the lung muscles. It is intended for patients with SMA type 1, type 2 or type 3, or those who have up to 4 copies of a gene known as SMN2. SMA is rare, and Evrysdi was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 26 February 2019. Further information on the orphan designation can be found here: [ema.europa.eu/medicines/human/orphan-designations/eu3192145](/en/medicines/human/orphan-designations/eu-3-19-2145). Evrysdi contains the active substance risdiplam.
Therapeutic Indication
### Therapeutic indication Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
Therapeutic Area (MeSH)
ATC Code
M09AX10
ATC Item
risdiplam
Pharmacotherapeutic Group
Other drugs for disorders of the musculo-skeletal system
Active Substance (Summary)
INN / Common Names
EMA Name
Evrysdi
Medicine Name
Evrysdi
Aliases
N/A
