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Five-year data from Genentech SMA study released

Spinal muscular atrophy (SMA) patients in the long-term extension Evrysdi study maintained or improved key developmental skills over the five-year period, data shows.

SMA EvrysdiSMA Evrysdi

New five-year data from Genentech (a member of the Roche Group)’s open-label extension of the pivotal Phase II FIREFISH study confirm the sustained efficacy and safety of Evrysdi ® (risdiplam) in children with Type 1 spinal muscular atrophy (SMA).

What did the FIREFISH extension study find?

The study found that after five years without treatment, 91 percent of children were alive. A total of 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. Genentech reported.

Furthermore, at the end of year 5, seven children could stand. Three of these children could do so with support; Four unaided and six could walk with support, the data showed.

The company highlighted that without disease modifying treatment, natural history studies indicate children with Type 1 SMA are not expected to survive past two years old.

Evrysdi is 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. As a survival motor neuron 2 (SMN2) splicing modifier, Evrysdi is designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency, according to Genentech.

Importance of the new Evrysdi data

over five years, an overwhelming majority [of children treated with Evrysdi] also maintained the ability to swallow and to eat without a feeding tube”

“These long-term findings confirm the ongoing benefit of Evrysdi for children with Type 1 SMA,” noted Dr Giovanni Baranello, PhD, 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.”

“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,” explained Dr Levi Garraway, PhD, Genentech’s Chief Medical Officer and Head of Global Product Development.

The data were presented at the 2024 Cure SMA Research & Clinical Care Meeting.

Roche released three-year data from the SUNFISH study in March 2022. The results confirmed the long-term efficacy and safety of Evrysdi in a broad patient population in individuals aged between two to 25 years old with Type 2 or Type 3 SMA.