Evrysdi Therapy for Type 1 Spinal Muscular Atrophy Associated with Favorable 5-Year S&E Data

Five-year data from the open-label extension of the phase 2 FIREFISH (NCT02913482) clinical trial confirms the sustained efficacy and safety profile of Evrysdi (ridisplam; Genentech, South San Francisco, CA), a treatment for children with Type 1 spinal muscular atrophy (SMA). The results demonstrate that long-term treatment with Evrysdi is associated with reduced death and need for permanent ventilation, as well as improved ability to sit, stand, feed, and swallow as compared with natural history studies of untreated children with the disease. Results were presented at the Cure SMA Research & Clinical Care Meeting.

FIREFISH is an open-label, multi-center study assessing safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Evrysdi for the treatment of children aged 1 to 7 months with Type 1 SMA. At the end of 5 years:

• 91% of children treated with Evrysdi were alive.
• 81% were alive without permanent ventilation.
• 59% were able to sit without support for ≥30 seconds.
• 96% were able to swallow.
• 80% were able to feed without a feeding tube.

Additionally, 7 children treated with Evrysdi were able to stand, with 4 able to stand unaided. Six children were able to walk with support. According to a statement from Genentech, children treated with Evrysdi demonstrated maintained or continued achievement of motor function abilities at 5 years as measured by 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). No treatment-related adverse events (AEs) caused treatment discontinuation or study withdrawal, and the rate of AEs and hospitalizations decreased during the treatment period.

“These long-term findings confirm the ongoing benefit of Evrysdi for children with Type 1 SMA,” said Giovanni Baranello, MD, PhD, Professor at the University College London Great Ormond Street Institute of Child Health & Great Ormond Street Hospital. “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.”