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Spinal Muscular Atrophy Patients Show Sustained Benefits from Evrysdi in Long-Term Study

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Spinal muscular atrophy (SMA) patients show sustained efficacy and safety of Evrysdi® (risdiplam) in children with Type 1, according to new five-year data from Genentech’s (a member of the Roche Group) open-label extension of the pivotal Phase II FIREFISH study. The findings highlight the long-term benefits of Evrysdi, a treatment designed to improve motor function and survival in children affected by this debilitating condition.

The FIREFISH extension study provides critical insights into the long-term outcomes for children with Type 1 SMA treated with Evrysdi. The study revealed that, after five years, 91 percent of children were alive, a significant achievement given that natural history studies indicate children with Type 1 spinal muscular atrophy are not expected to survive past two years without disease-modifying treatment.

The data also showed that 96 percent of children treated with Evrysdi could swallow, 80 percent could feed without a feeding tube, and 59 percent could sit without support for at least 30 seconds. Additionally, by the end of the five-year period, seven children could stand, with three doing so with support and four unaided. Furthermore, six children could walk with support.

The First Oral, Non-Invasive Spinal Muscular Atrophy Treatment Demonstrates Long-Term Benefits

Evrysdi is unique as the only oral, non-invasive small molecule treatment for SMA patients, designed to be delivered to both the central nervous system (CNS) and peripheral tissues. It functions as a survival motor neuron 2 (SMN2) splicing modifier, addressing SMA caused by mutations in chromosome 5q that lead to a deficiency in survival motor neuron (SMN) protein. The sustained ability to perform essential functions such as swallowing and feeding without assistance underscores the importance of this treatment in improving the quality of life for patients and their families.

“These long-term findings confirm the ongoing benefit of Evrysdi for children with Type 1 SMA,” noted Dr. Giovanni Baranello, PhD, from the UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital, UK. “Children treated with Evrysdi over five 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.”

Spinal Muscular Atrophy

Evrysdi’s Long-Term Efficacy Highlighted in Final FIREFISH Study Results, Transforming SMA Care

Dr. Levi Garraway, PhD, Genentech’s Chief Medical Officer and Head of Global Product Development, emphasized the significance of these findings, stating, “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.”

The data were presented at the 2024 Cure SMA Research & Clinical Care Meeting, showcasing the long-term benefits of Evrysdi and its potential to transform the standard of care for spinal muscular atrophy patients. In addition to the FIREFISH study, Roche released three-year data from the SUNFISH study in March 2022. These results confirmed the long-term efficacy and safety of Evrysdi in a broader patient population, including individuals aged between two and 25 years old with Type 2 or Type 3 spinal muscular atrophy.

The five-year data from Genentech’s Evrysdi study provides compelling evidence of the drug’s sustained efficacy and safety in treating children with Type 1 spinal muscular atrophy. The ability of Evrysdi to help children maintain or improve critical developmental skills over such an extended period marks a significant advancement in the treatment of this severe condition. As Genentech continues to explore and expand the applications of Evrysdi, these findings offer hope and improved quality of life for many patients and their families.

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Resource: Genentech, June 07, 2024

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