Pre-symptomatic Infants Retain Swallowing Ability in Evrysdi Trial

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A half-full prescription medicine bottle bears a label reading 'Clinical Trials.'

After being treated with Evrysdi (risdiplam) for at least a year, pre-symptomatic infants with spinal muscular atrophy (SMA) have retained the ability to swallow, and most have been able to stand and walk within developmentally normal windows.

That’s according to new data from the RAINBOWFISH clinical trial presented at the World Muscle Society Virtual Congress by scientists at Roche, which markets Evrysdi in collaboration with PTC Therapeutics.

“These new data for Evrysdi may help extend the potential benefits of this medicine to the youngest SMA patients,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Roche, said in a press release.

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RAINBOWFISH (NCT03779334) is a Phase 2 trial that is testing the efficacy, safety, and pharmacological properties of Evrysdi given to infants as young as six weeks old with genetically confirmed SMA and who have not yet started to show disease symptoms. The trial is open-label and all participants are given one daily dose of the active medication.

The Roche-sponsored study is actively recruiting participants at multiple clinical sites around the world.

The new data come from five children who have been treated with Evrysdi for at least 12 months in the study. Prior data from these five children showed that all five were able to maintain head control, sit upright, roll over, and crawl.

The new findings show that, after a year on treatment, all five infants have maintained the ability to swallow, and are able to feed exclusively by mouth.

Furthermore, four of the five children were able to stand and walk independently, at ages considered developmentally appropriate by the World Health Organization.

The findings further support the efficacy of Evrysdi in very young patients, according to Roche. Currently, more than 4,000 people have been treated with Evrysdi.

Evrysdi is available in a liquid that can be taken by mouth or via a feeding tube. The medication works by increasing levels of the SMN protein, defects of which cause SMA.

Evrysdi was approved last year to treat all types of SMA in individuals ages 2 months and older in the U.S. In recent months, it has received similar approvals in Japan,  Europe, and Canada.