News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Evrysdi improves motor function in pre-symptomatic babies after one year and its safety profile in previously treated people with SMA is confirmed

    In response to our request, Roche has shared new interim data from two studies evaluating risdiplam in people with SMA, who have previously been treated with other SMA-targeting therapies (JEWELFISH) and pre-symptomatic babies (RAINBOWFISH). The findings were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting, which is taking place from 9th to 11th June 2021.

    Evrysdi show improved motor function in pre-symptomatic babies after one year and confirm safety profile in previously treated people with SMA

    Evrysdi improves motor function in pre-symptomatic babies after one year and its safety profile in previously treated people with SMA is confirmedJEWELFISH

    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 and onasemnogene abeparvovec. The JEWELFISH study has enrolled the broadest patient population ever studied in an SMA trial.

    Safety:

    • Safety of risdiplam was shown to be consistent with previous studies.
    • No treatment-related adverse events (AEs) leading to withdrawal or treatment discontinuation 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 median SMN protein levels versus baseline (from the start of the trial), irrespective of which treatment was previously received or SMA type.

    Motor function:

    • Efficacy data indicated that people treated with risdiplam showed stabilisation in motor function at 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.

    Evrysdi improves motor function in pre-symptomatic babies after one year and its safety profile in previously treated people with SMA is confirmedRAINBOWFISH

    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 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, the number of infants sitting without support for at least five seconds, will be reported when all primary analysis patients have reached one year of treatment.

    Safety:

    • 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.

    More information