Genentech Presents Two Year P-II (JEWELFISH) Study Results of Evrysdi (risdiplam) for the Treatment of Spinal Muscular Atrophy at WMS 2022
Shots:
- The P-II (JEWELFISH) exploratory study evaluating Evrysdi in 174 patients aged 6mos. to 60yrs. with type 1, 2, or 3 SMA who received other investigational or approved SMA therapies
- The results showed an improved or maintained motor function & rapid increases in SMN protein levels sustained @2yr., longer-term safety data consistent with prior observed in earlier consistent safety profiles with exploratory efficacy. The therapy also showed a two-fold increase in median SMN protein levels over baseline @4wks. in all patient groups
- Low rates of discontinuation with a 5% rate/yr. over the 24mos. period. 96% with SMA viewed disease stabilization in a recent survey. Evrysdi received PRIME designation from the EMA and ODD from the US FDA
Ref: Businesswire | Image: Genentech
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