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. Update on the clinical development of risdiplam

    Following our request to receive important and timely information about the risdiplam clinical development programme, Roche has shared with us an update on part 1 of the FIREFISH clinical trial.

    FIREFISH is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2.

    New 2-year data from Part 1 of the pivotal FIREFISH study of risdiplam in infants aged 1-7 months with symptomatic Type 1 SMA, showed that infants treated with the therapeutic dose of risdiplam (17/21) continued to improve and achieve motor milestones.

    This exploratory analysis showed that:

    • An estimated 88% of infants were alive and required no permanent ventilation at two years.
    • At two years:
      • 59% (10/17 vs. 7/17 at 1-year) of infants were able to sit without support for at least 5 seconds, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III).
      • 65% (11/17 vs. 9/17 at 1-year) had maintained upright head control,
      • 29% (5/17 vs. 2/17 at 1-year) could turn themselves over and
      • 30% (5/17 vs. 1/17 at 1-year) were able to stand either supporting weight or with support
    • After two years of treatment with Evrysdi:
      • 71% (12/17 vs. 10/17 at 1-year) of infants achieved a CHOP-INTEND* score of 40 points or more
      • All infants increased their score from month 12 to month 24
      • Of the infants alive at two years (n=14), 100% maintained the ability to swallow and 93% (13/14) were able to feed orally.

    Safety for risdiplam in the FIREFISH study was consistent with its previously reported safety profile and no new safety signals were identified. The most common adverse events (n=21) included fever (pyrexia; 71%), upper respiratory tract infection (52%), cough (33%), vomiting (33%), diarrhoea (29%) and respiratory tract infection (29%). The most serious adverse event that occurred in 24% of infants was pneumonia.

    The data were presented at the virtual 25th International Annual Congress of the World Muscle Society.

    In August, the FDA approved risdiplam for the treatment of SMA in adults and children 2 months of age and older for use in the U.S. At this time, risdiplam has been filed in 16 international markets: Australia, Brazil, Chile, India, Indonesia, Israel, Kuwait, Macedonia, Malaysia, Russia, Singapore, South Korea, Taiwan, Thailand, Ukraine and the United Arab Emirates. In addition, four health authorities worldwide are currently reviewing the application: Canada, China, EU and Switzerland.

    For more information