The European Commission has approved the extension of the marketing authorisation for Evrysdi (risdiplam):
- Evrysdi (risdiplam) will become available from birth in SMA type I, II, III or for those with one to four copies of SMN2 (previously it was only available from the age of 2 months),
- This decision is based on the encouraging interim results of the RAINBOWFISH trial in presymptomatic SMA: six participants aged less than 6 weeks at the time of the first dose and treated for one year are all alive, without permanent ventilation and able to sit up, 67% can stand and 50% walk independently.
European Commission approves Roche’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA). Roche. 29 August 2023 press release.
