Genentech’s Evrysdi Granted FDA Priority Review for Spinal Muscular Atrophy

 

Interim data submitted to the FDA show majority of pre-symptomatic babies treated with Evrysdi for at least one year were able to sit, stand and walk within timeframes typical of healthy babies, as well as maintain swallowing –

– Evrysdi is approved in 70 countries and submitted in a further 31 with more than 4,500 patients treated to date –

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). The sNDA submission incorporates interim data from the RAINBOWFISH study, which shows the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting, standing, walking and maintained the ability to swallow following 12 months of treatment.

https://www.businesswire.com/news/home/20220124005816/en/Genentech%E2%80%99s-Evrysdi-risdiplam-Granted-FDA-Priority-Review-for-Treatment-of-Pre-Symptomatic-Babies-Under-2-Months-of-Age-With-Spinal-Muscular-Atrophy-SMA