Genentech’s Evrysdi (risdiplam) granted FDA priority review for treatment of presymptomatic babies under 2 months of age with spinal muscular atrophy


24 January 2022 - Interim data submitted to the FDA show majority of pre-symptomatic babies treated with Evrysdi for at least one year were able to sit, stand and walk within timeframes typical of healthy babies, as well as maintain swallowing.

Genentech today announced that the U.S. FDA has granted priority review of a supplemental new drug application for the use of Evrysdi (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy.

Read Genentech press release

Michael Wonder

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Michael Wonder

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US , Paediatrics , Dossier , Gene therapy