Roche's Evrysdi Receives the EC’s Marketing Authorization Extension for Newborns with Spinal Muscular Atrophy
Shots:
- The EC has expanded the marketing authorization for Evrysdi to include infants with SMA Type 1, Type 2, or Type 3 or with one to four SMN2 copies from birth to ≤2mos.
- The approval was based on the interim analysis from the (RAINBOWFISH) study. All 6 infants incl. in the analysis were able to sit after 1yr. of treatment with Evrysdi i.e., 100% (6/6) while 67% (4/6) could stand, 50% (3/6) walked independently & all infants were alive at 12mos. without permanent ventilation
- The safety profile of Evrysdi in pre-symptomatic babies was consistent with the safety profile in prior trials. Evrysdi is currently being studied in the P-II/III trial (MANATEE) in combination with an anti-myostatin molecule targeting muscle growth for SMA
Ref: Globenewswire | Image: Roche
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