SMA Newsroom

Oct 5, 2023

Community Update from Roche

We are pleased to announce that, in response to our standing request for important SMA clinical development and programme updates, Roche has shared an update on the RAINBOWFISH clinical trial, for which results from the primary analysis were presented at the 28th World Muscle Society (WMS) Congress.

As a reminder, RAINBOWFISH (NCT03779334) is an open-label study investigating risdiplam in 26 babies not yet presenting with symptoms, aged from birth to six week at first dose. This study was designed to evaluate the efficacy (benefit), safety (risk), pharmacokinetics and pharmacodynamics (how the drug behaves in the body) of risdiplam in presymptomatic babies. 

Roche has confirmed that the RAINBOWFISH clinical trial has met its primary endpoint of 80% of babies sitting without support for at least five seconds after 1 year of Evrysdi treatment. Without treatment these babies would never be able to sit. Today the following press release was issued summarising the study's safety and efficacy outcomes presented at the WMS Congress. This follows the news in August that the European Commission approved risdiplam for babies under two months old with SMA, based on an interim analysis of the RAINBOWFISH study. 

As always, Roche thanks the SMA community for its ongoing partnership and support, especially the many people and families living with SMA who have participated in the risdiplam clinical development programme. Roche continues to advance the science and understanding of risdiplam in SMA thanks to your continued collaboration.