top of page

Cure SMA Research & Clinical Care Meeting 2024 | News | Updates | iPharmaCenter

Roche Reveals Five-Year Data for Evrysdi Showing Major Improvements in Children with Severe Spinal Muscular Atrophy

Roche has announced new five-year results from the FIREFISH study, highlighting the long-term efficacy and safety of Evrysdi (risdiplam) in treating children with Type 1 spinal muscular atrophy (SMA). The findings reveal that a majority of the children treated with Evrysdi achieved or maintained critical motor milestones such as sitting, standing, and walking.

Key Outcomes of the Study

After five years of treatment, 91% of the children were alive, a significant improvement given that untreated children with Type 1 SMA typically do not survive beyond two years of age. The data also showed that 96% of the children could swallow, 80% could eat without the need for a feeding tube, and 59% could sit unsupported for at least 30 seconds. Additionally, by the end of the study, seven children could stand (three with support and four unaided), and six could walk with support.

Motor Function and Safety Results

The study assessed motor function using the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) and the Hammersmith Infant Neurological Examination 2 (HINE-2). These assessments confirmed that the children either maintained or continued to achieve motor functions over the five-year period.

Notably, no treatment-related adverse events led to discontinuation or withdrawal from the study. There was also a 66% reduction in the overall rate of adverse events from the first year to the fifth year of treatment, with the most common adverse events being upper respiratory tract infections (64%), fever (64%), and pneumonia (50%). Furthermore, hospitalizations decreased over time, with 22% of the children not requiring hospitalization since starting Evrysdi treatment.

Unique Features of Evrysdi

Evrysdi stands out as the only oral, non-invasive small molecule treatment for SMA that can be systemically delivered to both the central nervous system (CNS) and peripheral tissues. Roche leads its clinical development in collaboration with the SMA Foundation and PTC Therapeutics.

Ongoing and Completed Trials

Evrysdi has been evaluated in several global multicentre trials:

  • FIREFISH (NCT02913482): An open-label, pivotal trial for infants with Type 1 SMA, which met its primary endpoint.

  • SUNFISH (NCT02908685): A double-blind, placebo-controlled trial for individuals aged 2-25 with Types 2 or 3 SMA, which also met its primary endpoint.

  • JEWELFISH (NCT03032172): An exploratory trial assessing safety and tolerability in SMA patients aged 6 months to 60 years who had previously received other SMA treatments. Recruitment has been completed with 174 participants.

  • RAINBOWFISH (NCT03779334): A single-arm study evaluating Evrysdi in newborns up to six weeks old with genetically diagnosed SMA who were asymptomatic at the time of treatment. This study met its primary endpoint.

  • MANATEE (NCT05115110): A phase 2/3 trial combining Evrysdi with GYM329 (RG6237), an anti-myostatin molecule, to enhance muscle growth in children aged 2-10 years. This study is currently recruiting and has received Orphan Drug Designation from the FDA.

These extensive trials demonstrate Evrysdi’s potential to significantly improve the lives of those affected by SMA, underscoring its critical role in the ongoing management and treatment of this severe condition.


Recent Posts

See All


bottom of page