FDA Approves label Extension for Evrysdi for Infants with SMA Under 2 Months Old
The SMA Foundation’s partners, Roche and PTC Therapeutics, announced today that the FDA has approved a label extension for Evrysdi(risdiplam) to include babies under two months old with SMA. The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that pre-symptomatic babies treated with Evrysdi for at least one year were able to sit, stand and walk. Evrysdi is now approved in the US to treat SMA in children and adults of all ages. For more information, please see press releases from Roche/Genentech and PTC Therapeutics.