- More than twice as many babies (61% vs. 29%) were able to sit without support for at least five seconds after 24 months compared to 12 months of treatment
- Evrysdi increased survival and reduced need for permanent ventilation
- Evrysdi has proven efficacy across adults, children and babies 2 months and older
Roche today announced new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi™ (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 spinal muscular atrophy (SMA). The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to sit without support. The study also showed Evrysdi continued to improve survival, improve ability to feed orally and reduce the need for permanent ventilation*. Exploratory data suggested Evrysdi continued to improve the ability to swallow and reduce hospitalisations compared to the natural course of Type 1 SMA. Safety for Evrysdi was consistent with its established safety profile. These longer-term data build upon one-year pivotal findings from FIREFISH Part 2 and will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting being held virtually April 17-22, 2021.