Type 1 Children on Evrysdi Continue to Hit Milestones After 3 Years

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Children with spinal muscular atrophy (SMA) type 1 who started treatment with Evrysdi (risdiplam) in the first months of life are continuing to gain motor skills after three years of treatment, according to new data from the FIREFISH trial.

“These long-term results in babies treated with Evrysdi are very encouraging, with the vast majority improving or maintaining motor functions after three years,” Levi Garraway, MD, PhD, said in a press release. “Without treatment, they would typically not survive beyond two years of age.”

Garraway is chief medical officer and head of global product development at Genentech, a subsidiary of Roche, which developed Evrysdi in collaboration with PTC Therapeutics and the SMA Foundation.

“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,” Garraway said.

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Evrysdi is an oral medication designed to boost levels of the SMN protein, whose defect causes SMA. It is widely approved to treat SMA.

The Phase 2/3 FIREFISH study (NCT02913482) enrolled infants with type 1 SMA, who were treated with Evrysdi between the ages of 1 and 7 months. Two-year data from the study had showed most participants were still alive and did not require permanent ventilation, a marked departure from the natural course of the disease.

As of the data cut-off of Nov. 23, 2021, most of the children (91%) were still alive after three years on Evrysdi (based on data from 58 participants). There have been no additional deaths since prior results were reported.

After two years, 32 of 48 children with available data were able to sit unassisted for five seconds. All of these children retained this ability at three years, and an additional four children hit this motor milestone between years two and three. All 20 children who had been able to sit unsupported for 30 seconds at two years could still do so at three years, and an additional 15 children gained this motor milestone from years two to three.

From year two to year three, 36 children retained the ability to hold their heads upright, three children gained this ability, and no children with head control lost the ability. All 15 children able to pivot while sitting at year two could still do so at year three, and 11 children gained this ability from years two to three.

Five children gained the ability to stand with support from years two to three. Of seven children able to stand with support at year two, six retained this ability, while one was no longer able to stand supported at year three.

Two children gained the ability to walk with support from study years two to three. One child, who had already hit this motor milestone by year two, retained it at year three.

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The most common adverse events reported in the study were fever (60%), upper respiratory tract infection (57%), and pneumonia (43%). The most common serious adverse events were pneumonia (36%) and respiratory distress (10%). These safety events were largely related to the underlying disease, rather than a side effect of Evrysdi.

The rates of pneumonia and other adverse events have decreased over time in FIREFISH, as well as the rate of hospitalizations among participants.