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Genentech’s Evrysdi (risdiplam) Receives the US FDA’s Priority Review of sNDA for the Treatment of Spinal Muscular Atrophy

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Genentech’s Evrysdi (risdiplam) Receives the US FDA’s Priority Review of sNDA for the Treatment of Spinal Muscular Atrophy

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  • 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

Click here to­ read the full press release 

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