New Three-Year Data for Roche’s Evrysdi™ Show Long-Term Improvements in Survival and Motor Milestones in Infants who have SMA Type 1
04 May 2022
Roche recently presented new three-year data from the FIREFISH study, including one-year data from the open label extension, reinforcing the long-term efficacy and safety of Evrysdi™ (risdiplam) in infants with symptomatic SMA Type 1.
The latest data from the FIREFISH study of risdiplam, which was presented at the 14th European Paediatric Neurology Society (EPNS) Congress, show that:
- 91% of infants treated with Evrysdi in the FIREFISH study were still alive at three years.
- Infants treated with Evrysdi maintained or continued to improve in measures of motor function, including their ability to swallow, sit without support, stand with support and walk while holding on.
- The study also showed overall continued reductions in serious adverse events and hospitalisations over time.
The FIREFISH study evaluated the efficacy and safety of Evrysdi in infants aged 1-7 months at the time of enrolment with Type 1 SMA. The study was in two parts:
- Part 1: a dose-finding period
- Part 2: evaluation of the efficacy and safety of risdiplam at the dose selected in Part 1.
The pooled population includes participants treated with Evrysdi™ at the approved dose for a minimum of three years.
Evrysdi™ is currently approved in 79 countries and the dossier is under review in a further 29 countries.