New Data for Roche’s Evrysdi (risdiplam) Demonstrate Long-Term Efficacy & Safety in a Broad Population of People who have SMA

17 March 2022

New Data for Roche’s Evrysdi (risdiplam) Demonstrate Long-Term Efficacy & Safety in a Broad Population of People who have SMA

Summary

The latest risdiplam (Evrysdi) data updates were presented virtually at the 2022 MDA Clinical and Scientific Conference on 13th - 16th March:

  • Long-term efficacy data from the SUNFISH study confirm increases in motor function are sustained at three years, while adverse events decreased over the same period;
     
  • Part 2 of SUNFISH demonstrated;
     
  • Latest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated with Evrysdi for at least 12 months were able to stand and walk within time frames typical of healthy babies;
     
  • More than 5,000 patients have been treated with Evrysdi to date, from newborns to people over 60 years of age

Roche presented new data from two trials of Evrysdi (risdiplam) in SMA, at the 2022 MDA Clinical and Scientific Conference.

Risdiplam is a small molecule, oral drug that targets and encourages the SMN2 "back-up" gene to produce greater amounts of functional SMN protein.
 

SUNFISH trials

New three-year data confirmed:

  • The long-term efficacy and safety of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA;
     
  • Evrysdi was well-tolerated over the three-year time period. The overall rate of adverse events (AEs) in SUNFISH decreased over three years and a trend towards a lower rate of serious adverse events (SAEs) was observed in the third year of treatment. Overall, AEs and SAEs were reflective of the underlying disease and no treatment-related AEs led to withdrawal from the study.
     
  • The increase in Motor Function Measure 32 (MFM32) total score from baseline, previously observed at year one, was maintained through year three;
     
  • The increases in Revised Upper Limb Module (RULM) and Hammersmith Functional Motor Scale Expanded (HFMSE) total scores from baseline were also sustained between year one and year three.

Two-year efficacy data from Part 2 demonstrate improvement in or stabilisation of motor function with Evrysdi compared to an untreated external control group:

  • The proportion of patients demonstrating a marked improvement (change ≥3 points) or stabilisation (change ≥0 points) were more likely in people who were on Evrysdi for 24 months than those in the untreated group.
     

RAINBOWFISH trial

Updated interim data in pre-symptomatic babies with SMA under two months of age demonstrated the safety and efficacy of Evrysdi for newborns. 

In January, the U.S. Food and Drug Administration (FDA) granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi to treat pre-symptomatic babies with SMA under two months of age.

To date, more than 5,000 people have been treated with Evrysdi in clinical trials, compassionate use or real-world settings. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
 

Further information:

The press release
About risdiplam