Roche Reports Two-Year Data for Evrysdi (risdiplam) in Infants with Type 1 SMA

Shots:

• Roche reports the new 2-year data from Part 1 of the FIREFISH study assessing Evrysdi in infants aged 2-7mos. with symptomatic Type 1 SMA. The 2-year results in infants treated with Evrysdi showed that continued improvement and achieve motor milestones
• The exploratory analysis demonstrated 88% of infants were alive and required no permanent ventilation @2yrs., 59% of infants were able to sit without support for at least 5sec., 65% had maintained upright head control, 29% can turn themselves over and 30% were able to stand with support, 71% achieved a CHOP-INTEND score of ≥40 points and all infants increased their score from 12-24mos., all infants are alive in which 100% maintained the ability to swallow and 93% were able to feed orally
• Evrysdi is an SMN2 splicing modifier, being studied in 450 patients as part of a clinical program in SMA, with patients ranging from birth to 60yrs., and includes pre-symptomatic patients and those prior treated with other SMA-targeting therapies

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