Roche has reported four-year follow up data from the open-label extension of the FIREFISH study of Evrysdi (risdiplam) to treat Type 1 spinal muscular atrophy (SMA) in children.
The two-part study is designed to assess the safety and efficacy of the small molecule pre-mRNA splicing modifier Evrysdi in 58 infants aged one to seven months.
Out of the total, 37 infants were able to sit without support for at least five seconds at month 48, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III).
Motor functions were also evaluated using Hammersmith Infant Neurological Examination 2 (HINE-2) scale.
Between months 24 and 48, three infants were able to stand alone while one gained the ability to walk alone.
In addition, more than 95% of infants maintained their ability to feed by mouth and swallow up to month 48.
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Common adverse events of pyrexia, upper respiratory tract infection and pneumonia were observed during the study period.
Roche global product development head and chief medical officer Levi Garraway said: “The independence that comes with sitting, standing and walking is transformational for children with SMA, and their families, and we are very encouraged by how these skills increased over four years of Evrysdi treatment for many children in this study.
“Nine out of ten patients in our studies remain on Evrysdi long-term and these data underscore its importance as an option for people with SMA across a broad range of age and disease types.”
Evrysdi targets mutations caused in chromosome 5q of survival motor neuron-2 (SMN2) thereby increasing the production of SMN protein in the peripheral tissues and central nervous system.
Being developed in collaboration with PTC Therapeutics and SMA Foundation, Evrysdi is available in liquid form and can be administered daily at home orally or by feeding tube.