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

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

Click here to­ read full press release/ article | Ref: Roche | Image: Roche

Tuba Khan

Tuba Khan is Senior Editor at PharmaShots. She is curious, creative, and passionate about recent updates and innovation in the Life sciences industry. She covers Biopharma, MedTech, and Digital health segments. Tuba also has an experience of digital and social media marketing and runs the campaigns independently. She can be contacted on tuba@pharmashots.com

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