Shots:

• The NDA submission is based on FIREFISH Part-1 and SUNFISH Part-2 studies. The FIREFISH (Part 1 & 2) study involves assessing Risdiplam in 21 & 41 infants aged 1-7mos. with SMA- followed by an open-label extension respectively
• The SUNFISH (Part 1 & 2) study involves assessing Risdiplam vs PBO in children & young adults aged 2-25yrs. with Type 2 or 3 SMA. Part 2 study resulted in meeting its 1EPs i.e- change in the Motor Function Measure 32 (MFM-32) scale- the safety profile is consistent with no new safety signals
• Risdiplam (PO) is an investigational SMN-2 splicing modifier- designed to increase and sustain SMN protein levels both via CNS and peripheral tissues of the body and has received FDA’s ODD and FT designation In Jan’17 & Apr’17 respectively with its expected PDUFA date as May 24- 2020

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