Evrysdi linked to significantly improved outcomes in spinal muscular atrophy at 5 years

Key takeaways:

• At 5 years, 91% of pediatric patients treated with Evrysdi were alive, 81% without permanent ventilation.
• Data further showed 96% were able to swallow; 80% ate without a feeding tube at year 5.

Newly released data from the open-label extension of the FIREFISH study confirmed sustained safety and efficacy of Evrysdi in children with type 1 spinal muscular atrophy over 5 years.

The data were reported in a release from manufacturer Genentech and presented at the Cure SMA Research & Clinical Care Meeting earlier in June.

At 5 years, 91% of children treated with Evrysdi (risdiplam) were alive, 81% were alive without permanent ventilation and 59% of those treated were able to sit without support for at least 30 seconds. Additionally, data showed that seven children were able to stand, including three with support, four stood unaided and six could walk with support.

Results further showed 96% were able to swallow and 80% were able to feed without a feeding tube at year 5.

Motor function abilities, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III) and Hammersmith Infant Neurological Examination 2 (HINE-2), were sustained in those treated with Evrysdi, according to the release.

No treatment-related adverse events (AEs) led to treatment discontinuation or withdrawal from the study, with overall AE rate decreasing by 66% between year 1 and the final year of study. The most common AEs were upper respiratory tract infection, pyrexia and pneumonia.

Hospitalizations declined 50% over the 5-year treatment period and 22% of patients treated with the survival motor neuron 2 (SMN2) splicing modifier did not require hospitalization at all after treatment initiation, Genentech reported in the release.

“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 [spinal muscular atrophy],” Levi Garraway, MD, PhD, Genentech’s chief medical officer and head of Global Product Development, said in the release. “This would not have been possible without the commitment and dedication of the children and families who participated, as well as numerous health care professionals and patient support organizations to whom we are immensely thankful.”