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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 presents new data on SUNFISH & JEWELFISH trials at the Cure SMA Annual 2020 virtual Conference

    Roche presented new data on Part 1 of the SUNFISH trial, as well as on the JEWELFISH study, at the virtual Cure SMA Annual Conference, which took place between 8th and 12th June.

    Roche's risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 SMASUNFISH

    SUNFISH is a large (231 participants) global two-part study in children and adults, aged between 2 and 25 years, with Type 2 or 3 SMA. The dose-finding SUNFISH Part 1 (51 participants) includes a broad patient population, ranging from individuals unable to sit to those capable of walking, as well as people with scoliosis or joint contractures.

    Roche presented two-year data from this Part 1 of the SUNFISH trial. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data, as assessed by the Motor Function Measure (MFM) scale. In a weighted analysis comparing the data with a natural history comparative cohort, the MFM total change from baseline at month 24 was greater in patients receiving risdiplam with a 3.99 point difference. These results are consistent with the results of the Part 2 of the trial at 12 months in non-ambulatory patients, which demonstrated that change from baseline in total MFM32 score was greater in people treated with risdiplam, compared to placebo (with a 1.55 point mean difference).

     

    JEWELFISH

    JEWELFISH is a trial in people with all types of SMA, aged 6 months to 60 years and previously treated with other SMA therapies. Preliminary 12-month data showed that treatment with risdiplam led to rapid and sustained increases in SMN protein levels. In more detail, among the patients who completed 12 months of treatment with risdiplam, a median two-fold increase in SMN protein versus baseline was observed (n=18). Enrolment for the JEWELFISH study is now complete (174 participants).

    To date there have been no drug-related safety findings leading to withdrawal from either trial. The overall adverse event profile is similar to that observed in risdiplam trials of patients not previously treated with a SMA-targeting therapy.

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