Roche has reported three-year findings from the FIREFISH clinical trial, where its Evrysdi (risdiplam) demonstrated durable improvements in survival and motor milestones in infants with symptomatic Type 1 spinal muscular atrophy (SMA).
A survival motor neuron 2 (SMN2) splicing modifier, Evrysdi is intended for treating SMA caused by mutations in chromosome 5q that lead to the deficiency of SMN protein.
It is given as daily at-home doses in liquid form through the mouth or using a feeding tube.
Evrysdi can boost and sustain the SMN protein production in the central nervous system (CNS) and peripheral tissues, to treat SMA.
The open-label, two-part trial in infants with Type 1 SMA of the age one to seven months at the time of enrolment had two parts.
Part 1 was a dose-escalation trial that enrolled 21 infants to evaluate the safety profile of risdiplam in infants and detect the dose for Part 2 as its primary goal.
The single-arm Part 2 trial assessed the efficacy and safety at the dose selected in Part 1 in 41 subjects who received treatment for two years and a subsequent open-label extension.
The latest data showed the durable safety and efficacy of Evrysdi in infants with Type 1 SMA.
Furthermore, 91% of subjects who received Evrysdi were alive following three years of therapy.
Subjects in the Evrysdi arm continued to have improvement or maintained motor functions, including swallowing ability, sitting or standing without having support and walking while holding on, between two and three years following treatment.
The overall continued decline in serious adverse events (SAEs) and hospital admission over course of time were also reported.
Roche Global Product Development chief medical officer and head Levi Garraway said: “These long-term results in babies treated with Evrysdi are very encouraging, with the vast majority improving or maintaining motor functions after three years.
“Without treatment, they would typically not survive beyond two years of age.
“Support for the compelling efficacy of Evrysdi continues to grow for a broad range of people, including infants with one of the most severe forms of SMA.”
In July last year, the company reported that Evrysdi met the primary goal of the FIREFISH Part 2 study in infants aged one to seven months with symptomatic Type 1 SMA.
Apart from FIREFISH, Evrysdi is being analysed in four other multicentre trials, SUNFISH, JEWELFISH, RAINBOWFISH and MANATEE, in SMA patients.