Shots:

• The company has reported the results from Part 2 of the FIREFISH study evaluating the efficacy and safety of Evrysdi in 41 infants aged 1-7 mos. with symptomatic Type 1 SMA
• The study met its 1EP i.e.- 29% of infants sitting without support for at least 5 sec.- 93% of infants were alive and 85% required no permanent ventilation @ 12 mos.- median duration of treatment (15.2mos.) with median age was 20.7mos- 90% achieved a CHOP-INTEND
• Additionally- the study met its 2EPs i.e.- 78% of infants classified as HINE-2 responders. The therapy also showed improvement in survival and motor milestones & the safety was consistent with its known safety profile

Ref: Roche | Image: Roche