Genentech's Evrysdi Application Under FDA Priority Review For Use In Babies Below 2 Months

The FDA has granted priority review for Genentech's, a unit of Roche Holdings AG RHHBY supplemental marketing application for Evrysdi (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). 

• Evrysdi is already approved to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
• The submission incorporates interim data from the RAINBOWFISH study.
• The data showed that most pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting, standing, walking, and maintaining the ability to swallow following 12 months of treatment.
• No treatment-related serious adverse events were reported in any babies treated with Evrysdi through the interim safety analysis period (n=12).
• The latest results from the RAINBOWFISH study will be presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March 2022.
• Price Action: RHHBY shares are down 1.59% at $47.04 during the market session on the last check Tuesday.