Roche Reports the US FDA’s Acceptance of NDA for Risdiplam and Granted Priority Review to Treat Spinal Muscular Atrophy

Roche Reports the US FDA’s Acceptance of NDA for Risdiplam and Granted Priority Review to Treat Spinal Muscular Atrophy

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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