Five-year data for Evrysdi (risdiplam) show the majority of treated children with a severe form of spinal muscular atrophy (SMA) achieved or maintained the ability to sit, stand or walk.-Genentech/Roche

By the end of year 5, 91% of children treated with Evrysdi were alive, 81% were alive without permanent ventilation and the majority were able to sit without support for at least 30 seconds (59%). At the end of year 5, seven children were able to stand, three with support, four unaided and six could walk with support. Without disease modifying treatment, natural history studies indicate that children with Type 1 SMA would not only never be able to reach such milestones, but also not typically live past the age of two. The data were presented at the Cure SMA Research & Clinical Care Meeting, June 5 - 7, 2024.

“These long-term findings confirm the ongoing benefit of Evrysdi for children with Type 1 SMA,” said Professor Giovanni Baranello, M.D., Ph.D., UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, London, U.K. “Children treated with Evrysdi over 5 years have maintained or improved their ability to sit, stand and walk — critical skills for development and daily living. An overwhelming majority also maintained the ability to swallow and to eat without a feeding tube.”

Motor function abilities, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) and Hammersmith Infant Neurological Examination 2 (HINE-2), were maintained or continued to be achieved in those treated with Evrysdi. The FIREFISH results showed most children treated with Evrysdi also maintained their feeding and swallowing abilities. Of those assessed at year 5, 96% were able to swallow and 80% were able to feed without a feeding tube.

“This is the final readout of the FIREFISH study, which has provided a wealth of insights and data, helping to firmly establish Evrysdi as an important treatment option, improving the lives of children across the globe living with SMA,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “This would not have been possible without the commitment and dedication of the children and families who participated, as well as numerous healthcare professionals and patient support organizations to whom we are immensely thankful

No treatment-related adverse events (AEs) led to treatment discontinuation or withdrawal from the study. The overall rate of AEs decreased by 66% between year 1 and the final year of study. The most common AEs were upper respiratory tract infection (64%), pyrexia (64%) and pneumonia (50%). Hospitalizations declined over the 5-year treatment period and 22% of children did not require hospitalization at all since beginning treatment with Evrysdi. Evrysdi is the only oral, non-invasive small molecule SMA treatment designed to be systemically delivered to both the central nervous system (CNS) and peripheral tissues. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.