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. Roche’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 SMA

    As part of our ongoing partnership and following our request to receive important and timely information about the risdiplam clinical development programme, Roche shares with us an update on part 2 of the FIREFISH clinical trial.

    FIREFISH Part 2 is a pivotal, global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 SMA.

    After 12 months of treatment, part 2 of the FIREFISH clinical trial has met its primary endpoint with:

    • 29% of infants sitting without support for five seconds by month 12, as assessed by a test which measures motor function, called “Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III)”. No infants achieve this milestone in the natural history of Type 1 SMA
    • 43.9% of infants were able to hold their head upright
    • 31.7% were able to roll to the side
    • 4.9% of infants were able to stand with support
    • 93% percent of infants were alive and 85.4% were event-free. Without treatment, the median age of death or permanent ventilation was 13.5 months in a natural history cohort
    • In an exploratory endpoint, 95% of infants, who were alive at 12 months, maintained the ability to swallow and 89% were able to feed orally. In contrast, in a natural history cohort, all infants with Type 1 SMA older than 12 months required feeding support.

    Safety for risdiplam in the FIREFISH study was consistent with its previously reported safety profile and no new safety signals were identified.

    The data is to be presented at the currently ongoing 72nd American Academy of Neurology (AAN) Annual Meeting and will be made available online via virtual presentation in the coming weeks.

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