SMA Newsroom

Jul 29, 2021

Data for Evrysdi® published in New England Journal of Medicine shows significant improvement in survival and motor milestones in babies with Type 1 SMA

Data from FIREFISH Part 2, a pivotal global study evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies aged 1-7 months old with symptomatic Type 1 SMA, has been published in the New England Journal of Medicine.

The study met its primary endpoint with 29% of infants sitting without support for at least five seconds by month 12, a milestone not seen in the natural course of the disease. Safety for Evrysdi® in the FIREFISH Part 2 study was consistent with its known safety profile.

At the time of data analysis, the median duration of treatment with Evrysdi® was 15.2 months and the median age was 20.7 months. At month 12:

  • 93% of infants were alive and 85% were free from permanent ventilation. Without treatment, the median age of death or permanent ventilation was 13.5 months in a natural history cohort.
  • 90% had a motor function score increase of at least 4 points, with 56% achieving a score above 40; the median increase was 20 points.

In addition, the study met one of its secondary endpoints with 78% of infants classified as HINE-2 responders, which evaluated motor function through head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing and walking. Infants were classified as HINE-2 responders if more motor milestones showed improvement than worsened.Data from part 1 of FIREFISH, a dose-determining study, were published in NEJM in February 2021.

Evrysdi™ (risdiplam), is the third treatment approved for SMA in Europe. It is an orally-available compound, developed in collaboration with the SMA Foundation and PTC Therapeutics, which is used to increase the amount of SMN protein made by the SMN2 gene.