Shots:

• The FIREFISH Part 2 study involves assessing risdiplam in 41 infants aged 1-7mos. with Type 1 SMA treated for 24mos.- followed by an open-label extension
• The study met its 1EPs with 29% of infants sitting without support for 5sec by 12mos. as assessed by the Gross Motor Scale of BSID-III- 43.9% infants were able to hold their head upright- 31.7% were able to roll to the side- 4.9% infants were able to stand with support as measured by HINE-2. In exploratory endpoint- 95% were alive @12mos. maintained the ability to swallow and 89% were able to feed orally
• Risdiplam is an SMN2 splicing modifier- designed to durably increase and sustain SMN protein levels both throughout the CNS and PNS and has received the FDA’s ODD in 2017- EMA’s PRIME designation in 2018 and FDA’s PR in Nov’2019 with its anticipated decision on approval by Aug 24- 2020

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Ref: Roche | Image: Roche