Shots:

• The approval is based on the results from two studies i.e. FIREFISH & SUNFISH. The FIREFISH study involves assessing Evrysdi (dry syrup- 60mg) in infants with symptomatic Type 1 SMA- and the SUNFISH study evaluates Evrysdi in children & young adults aged 2-25yrs. with Type 2 or 3 SMA
• The 2-year data from the SUNFISH study demonstrate improvement or maintenance of motor function while the FIREFISH study continues to improve motor function & survival in babies
• The therapy has been approved as the first oral drug to be taken at home for SMA in Japan with proven efficacy in adults- children- and babies & received an ODD in Mar’19 & company filed a regulatory application in Oct’20

Ref: Roche | Image: Wikipedia