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RAINBOWFISH Clinical Trial Results

RAINBOWFISH Clinical Trial Results

Page last checked: 9th October 2023
Last updated: 9th October 2023

 

At the 28th World Muscle Society (WMS) Congress, Roche confirmed that the RAINBOWFISH clinical trial has met its primary endpoint of 80% of babies sitting without support for at least five seconds after one year of treatment with risdiplam. Without treatment these babies would never be able to sit. 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.

Roche’s Press release >

Clinical Trials. Gov Rainbowfish Trial Page >

Read more about all Risdiplam clinical trials and their results >

23rd March 2022:

Updated interim data, presented virtually at the 2022 MDA Clinical and Scientific Conference on 13th – 16th March, demonstrated the safety and efficacy of Evrysdi for newborns. The majority of babies treated with Evrysdi for at least 12 months were able to stand and walk within timeframes typical of healthy babies.

14th June 2021:

Preliminary results were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting, which took place from 9th to 11th June 2021.

All five babies had been treated with risdiplam for at least 12 months:

Motor function:

  • Achieved sitting without support, rolling and crawling. Of the five, two had two SMN copies and three had >2 copies.
  • Four of the infants were able to stand unaided and walk independently. In addition, four babies reached a maximum score of 64 on the CHOP-INTEND scale, and one scored 63. Data on the primary endpoint, which is the number of infants sitting without support for at least five seconds, will be reported when all patients have reached one year of treatment.

Safety:

  • There were no Adverse Events (AEs) leading to withdrawal or study discontinuation.
  • The most common AEs were nasal congestion (33%), cough (25%), teething (25%), vomiting (25%), eczema (17%), abdominal pain (17%), diarrhoea (17%), gastroenteritis (17%), papule (rash; 17%) and pyrexia (fever; 17%).