Genentech : New Data For Evrysdi Shows Improved Motor Function In Pre-Symptomatic Babies

Genentech, a member of the Roche Group (RHHBY), announced new interim data from two studies of Evrysdi or risdiplam; JEWELFISH and RAINBOWFISH. The new data showed improved motor function in pre-symptomatic babies after one year and confirmed safety profile in previously treated people with spinal muscular atrophy or SMA.

Data from JEWELFISH, an ongoing open-label study primarily evaluating the safety of Evrysdi in people aged 1 to 60 years who have been previously treated with another SMA-targeting therapy, including nusinersen and onasemnogene abeparvovec, showed the safety profile of Evrysdi and increase in SMN protein levels are consistent with those observed in other Evrysdi studies.

The data from JEWELFISH study also suggested stabilization in motor function at one year of treatment as measured by change from baseline in motor function measure (MFM-32).

Preliminary efficacy data from RAINBOWFISH, an ongoing open-label study evaluating Evrysdi in babies from birth to 6 weeks with pre-symptomatic SMA, showed that infants treated for 12 months achieved age-appropriate motor milestones, including sitting, standing and walking, and improvements in motor function.

Roche also presented preliminary efficacy data from RAINBOWFISH, which showed that of the 5 babies treated with Evrysdi for at least 12 months, all achieved sitting without support, rolling and crawling.

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