Roche Evrysdi gets CHMP recommendation for babies under two months old with spinal muscular atrophy

Basel: Roche has announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the extension of the Evrysdi (risdiplam) European Union (EU) marketing authorisation, which would include infants with genetically confirmed diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies, including from birth to below two months. The recommendation is based on an interim analysis from the ongoing RAINBOWFISH trial in pre-symptomatic babies with Type 1 SMA from birth to six weeks. In SMA, early treatment is critical to counteract ongoing and irreversible loss of motor neurons.3,4,5 A final decision regarding the approval is expected from the European Commission later this year.