Evrysdiâ„¢ Improves Motor Function in Pre-Symptomatic Babies

14 June 2021

In response to a request from SMA Europe, Roche has shared new interim data from two studies evaluating Evrysdi (also known as risdiplam) in people with SMA. The trials included pre-symptomatic babies (RAINBOWFISH) or patients who have been previously treated with other SMA-targeting therapies (JEWELFISH). The findings were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting, which took place from 9th to 11th June 2021.

Evrysdi improved motor function in pre-symptomatic babies after one year and was confirmed to be safe in people with SMA who have received alternative treatment.


The first study being presented was JEWELFISH, a trial evaluating the safety, tolerability and efficacy of risdiplam in people living with SMA Types 1-3, aged 1 to 60 years who had been previously treated with another SMA-targeting therapy, including nusinersen (Spinraza™) and onasemnogene abeparvovec (Zolgensma™). The JEWELFISH study has enrolled the broadest patient population ever studied in an SMA trial.


  • Safety of risdiplam was shown to be consistent with previous studies.
  • No treatment-related adverse events (AEs) leading to withdrawal or treatment discontinuation were observed, with some patients receiving treatment for more than three years.
  • The most common AEs in all patients were upper respiratory tract infection (17%), pyrexia (17%), headache (16%), nausea (12%), diarrhoea (11%), nasopharyngitis (10%) and vomiting (8%).
  • The most common serious AEs were pneumonia (2%) lower respiratory tract infection (2%), upper respiratory tract infection (2%) and respiratory failure (2%).

SMN levels:

All patients treated with risdiplam showed a sustained >2-fold increase in SMN protein levels when compared to the start of the trial, irrespective of previous treatment or SMA type.

Motor function:

Efficacy data indicated that people treated with risdiplam showed stabilisation in motor function after one year of treatment, as measured by change from baseline in motor function measure (MFM 32).

The study is open label (participants and researchers are aware of the treatment that is administrated) and ongoing with the full analysis conducted at month 24.


Preliminary data of the second study, RAINBOWFISH, was also presented. The study is evaluating the efficacy and safety of risdiplam in babies from birth to six weeks with pre-symptomatic SMA.

All five babies were 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.


  • There were no 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%).

RAINBOWFISH is open label and the only Roche-sponsored global clinical trial with risdiplam currently recruiting.

Further information

Roche’s Community Letter (attached to email) – 11th June 2021

Roche’s press release – 11th June 2011

About Evrysdi (risdiplam)

Risdiplam clinical trial results