Shots:

• The SUNFISH part 2 study involves assessing risdiplam vs PBO in patients aged 2-25yrs. with non-ambulatory type 2 or 3 SMA
• The SUNFISH part 2 study resulted in meeting its 1EPs & 2EPs i.e. greater change from baseline in MFM-32 & improvement in RULM @12mos.- strongest responses in MFM-32 in younger patients aged 2-5 yrs. (78.1% vs 52.9% achieving ≥3 point increase)- disease stabilization in patients aged 18-25 yrs. (57.1% vs 37.5%- with stabilization defined as a ≥0 point increase)
• Risdiplam is an investigational SMN2 splicing modifier for SMA- designed to increase and sustain SMN protein levels both throughout the CNS and PNS and has received the US FDA’s PR designation in Nov’2019 with its anticipated decision for approval by May 24- 2019

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Ref: Roche | Image: Roche