Data for Evrysdi Published in NEJM shows Significant Improvement in Survival and Motor Milestones in Babies with SMA Type 1
30 July 2021
Data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety of Evrysdi™ (risdiplam) in babies aged 1-7 months old with symptomatic SMA Type 1, has been published in the New England Journal of Medicine. The study met its primary endpoint with 29% of infants sitting without support for at least five seconds by month 12, a milestone not seen in the natural course of the condition. Safety for Evrysdi™ in the FIREFISH Part 2 study was consistent with its known safety profile.
At the time of data analysis, the median duration of treatment with Evrysdi™ was 15.2 months and the median age was 20.7 months.
At month 12:
- 93% of infants were alive and 85% were free from permanent ventilation. Without treatment, the median age of death or permanent ventilation was 13.5 months in a natural history cohort.
- 90% had a motor function score increase of at least 4 points, with 56% achieving a score above 40; the median increase was 20 points.
In addition, the study met one of its secondary endpoints with 78% of infants classified as HINE-2 responders, which evaluated motor function through head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing and walking. Infants were classified as HINE-2 responders if more motor milestones showed improvement than worsened.
Data from part 1 of FIREFISH, a dose-determining study, were published in NEJM in February 2021.