Roche said that Evrysdi (risdiplam) has offered sustained efficacy and safety in children with type 1 spinal muscular atrophy (SMA) in the open-label extension portion of the FIREFISH trial.
Evrysdi is an oral, non-invasive small molecule SMA therapy intended to be delivered systemically to the central nervous system (CNS) as well as peripheral tissues.
The drug received PRIME designation from the European Medicines Agency (EMA) in 2018 and was granted orphan drug designation by the US Food and Drug Administration in 2017. Currently, Evrysdi has approval across more than 100 countries.
FIREFISH was an open-label, two-part clinical trial conducted in infants diagnosed with Type 1 SMA. At enrollment, the infants had a median age of approximately 5.5 months.
Among the 58 infants who completed the first year of treatment, 52 transitioned into the open-label extension study.
The five-year data revealed that 91% of children treated with Evrysdi remained alive, with 81% not reliant on permanent ventilation.
Furthermore, 59% of them demonstrated the ability to sit without assistance for at least 30 seconds.
By the fifth year, seven children achieved standing capability, with three needing support, four standing unaided, and six able to walk with assistance.
Additionally, motor function abilities, evaluated through different assessments, remained stable or continued to progress in individuals receiving treatment with Evrysdi.
Roche said the FIREFISH study’s results also indicated that most of the children treated with risdiplam sustained their feeding and swallowing capabilities.
By year five, 96% of those assessed were able to swallow, and 80% could feed without the need for a feeding tube.
Roche chief medical officer and global product development head Levi Garraway said: “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.
“This would not have been possible without the commitment and dedication of the children and families who participated, as well as numerous healthcare professionals and patient support organisations to whom we are immensely thankful.”
Roche spearheads the clinical advancement of Evrysdi through a partnership with the SMA Foundation and PTC Therapeutics.
The small molecule drug is presently undergoing or has undergone, evaluation in five global multicentre trials including SUNFISH, JEWELFISH, RAINBOWFISH, and MANATEE, involving individuals with SMA.
