Shots:

• The NDA submission is based on FIREFISH study assessing Risdiplam in infants with symptomatic SMA Type 1 & SUNFISH study in children and young adults with SMA Type 2 or 3
• FIRESISH study results: improvement in survival and motor milestones in infants. SUNFISH study results: improvement in motor function in people aged 2-25 with type 2 or 3 SMA
• Risdiplam (PO) is a therapy designed to increase and sustain SMN protein levels in the CNS & throughout the body. The therapy has received MHLW’s ODD on Mar 27- 2020- and is a subject to PR in Japan

 ­ Ref: Chugai | Image: Financial Times