FDA approves label extension for Evrysdi to include babies under two months old with spinal muscular atrophy.- Genentech/Roche
Genentech/Roche announced that the FDA has approved a label extension for Evrysdi (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment.
Evrysdi is now approved to treat SMA in children and adults of all ages. Of the babies with 2 or 3 copies of the SMN2 gene (n=6), 100% were able to sit after one year of treatment with Evrysdi, 67% could stand and 50% of infants could walk independently. All infants were alive at 12 months without permanent ventilation.
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