Shots:
The approval was based on interim efficacy & safety data from the (RAINBOWFISH) study evaluating the efficacy, safety, PK & PD of Evrysdi in 25 patients aged birth to 6wks. with SMA
The results showed that patients treated with Evrysdi were able to sit (100%) @1yr., 67% can stand, 50% can walk independently & infants were alive without permanent ventilation @12mos.
The Evrysdi prescription information has also been revised as part of the label extension including 2yr. pooled results from parts 1 & 2 of the (FIREFISH) trial which showed that 60% & 28% of infants were able to sit & stand without support for 5sec., 40% for 30sec. in symptomatic babies aged 1-7mos. @2yr. at the recommended dose (n=58)
Ref: Genentech | Image: Genentech
