Roche’s Evrysdi Offers Survival Benefits in Babies With SMA

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.

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