FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy (SMA)

发布时间： 2022-06-15 阅读：430次

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June 1, 2022 --Genentech, a member of the Roche Group, recently 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). Evrysdi is now approved to treat SMA in children and adults of all ages. 

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. 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 priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “Because of its efficacy in multiple settings, Evrysdi is now available for people with SMA from pre-symptomatic newborns to older adults. We are proud of this achievement, which has the potential to make a real difference to those living with SMA and their caregivers.”