Shots:

• The MAA submission is based on dose-finding Part 1 and confirmatory Part 2 of the FIREFISH and SUNFISH studies evaluating the efficacy and safety of Evrysdi (risdiplam) in symptomatic infants with type 1 SMA aged 2-7mos. and in people with types 2/3 SMA aged 2-25 yrs/ respectively
• The submission also includes data from JEWELFISH study evaluating patients with all types of SMA aged 1-60yrs. prior treated with other SMA therapies. Exploratory efficacy analysis from SUNFISH Part 1 showed Evrysdi improved motor function @24mos. of treatment- Part 2 of the FIREFISH study met its 1EPs of infants sitting without support for 5sec by month 12- as assessed by BSID-III- Part 2 of the SUNFISH trial demonstrated that change from baseline in MFM-32 score was greater at 12mos.
• Roche will grant $15M as milestones to PTC following MAA acceptance. Evrysdi is an SMN2-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q leading to SMN protein deficiency

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