Shots:
The sNDA submission is based on the (RAINBOWFISH) study evaluating the efficacy, safety, PK & PD of Evrysdi in 25 patients aged birth to 6wks. with SMA
In an interim efficacy analysis, babies treated with Evrysdi maintained the ability to swallow & were able to feed orally while 80% (4/5) were able to sit, stand and walk independently & all participants met HINE-2 motor milestones of head control, sitting upright, rolling and crawling after 12mos. of treatment
Additionally, no treatment-related serious AEs were reported & the latest results from the will be presented at MDA in Mar 2022. The therapy has received PRIME designation from the EMA in 2018 & ODD from the US FDA in 2017
Ref: Businesswire | Image: Slide Genius
