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 continues to improve motor function and survival in babies with Type 1 SMA

    Evrysdi continues to improve motor function and survival in babies with Type 1 SMAIn response to our request for regular updates, Roche has shared with us new two-year data from part 2 of its FIREFISH study. These longer-term findings will be presented at the 73rd American Academy of Neurology (AAN) Annual Meeting, which will be held virtually between 17th and 22nd April 2021.

    FIREFISH is assessing Evrysdi™ (risdiplam) in the treatment of infants aged 1-7 months at enrollment with symptomatic Type 1 SMA.

    The data build upon one-year findings from FIREFISH Part 2, which measures the ability to sit without support for at least five seconds as its primary endpoint. At 12 months, 29% (12/41) of infants treated with risdiplam achieved this milestone.

    This new data shows that at 24 months, infants treated with risdiplam:

    • Could sit without support for at least five seconds (61%; 25/41). In the natural course of Type 1 SMA, without treatment, children are not able to sit without support for five seconds.
    • Maintained the ability to feed orally (92%; 35/38). Further, exploratory data suggest similar maintenance in ability to swallow (95%; 36/38). In the natural course of the disease, infants with Type 1 SMA older than 12 months generally require feeding support.
    • Were alive (93%; 38/41) and 83% (34/41) were alive and free from permanent ventilation, an improvement compared to the natural course of the disease. Without treatment, the median age of death or permanent ventilation is 13.5 months.

    The findings also show that safety for risdiplam was consistent with its established safety profile observed in previous studies and there were no medicine-related adverse events leading to participants stopping treatment or withdrawing from the study.

    More information