FDA approves Roche ’s Evrysdi for use in babies under two months with spinal muscular atrophy (SMA)

Approval based on interim RAINBOWFISH data which show pre-symptomatic babies treated withEvrysdi for at least one year were able to sit, stand and walkPrescribing information updated with FIREFISH data showing the majority of symptomatic babies treated withEvrysdi for at least two years could sit for at least five secondsEvrysdi has proven efficacy in babies, children and adults with more than 5,000 patients treated to dateBasel, 31 May 2022 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (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 t he 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 in the US 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.“The approval of Evrysdi for pre-symptomatic babies is particularly important, as early treatment of SMA, before symptoms start to arise, can help babies to achieve motor milestones,” said Richard Finkel, M...
Source: Roche Media News - Category: Pharmaceuticals Source Type: news