SUNFISH Clinical Trial Results
SUNFISH Clinical Trial Results
Page last updated: 14th April 2025
March 2025:
Positive long-term benefits of risdiplam in people with SMA shared at international conference.
At a recent international conference in the US, Roche shared new results from the SUNFISH study. The update focused on how risdiplam can help stabilise symptoms and support daily independence in people living with SMA.
The SUNFISH study looked at how risdiplam works in children and young adults aged 2 to 25 who have Type 2 or non-ambulant Type 3 SMA. At the start of the study, some people were given risdiplam and others were given a placebo (a treatment with no active medicine). After 12 months, everyone in the study began taking risdiplam and the study continued for a total of five years.
The key findings were:
- Stable motor function: people who took risdiplam showed improvements in their movement after one year. These improvements remained stable over the full five years of treatment. This was measured using a test called the MFM-32, which looks at how well people can move.
- Better independence: people over the age of 12, along with their caregivers, reported that daily tasks like getting dressed, washing and picking up objects either stayed the same or got easier. This was tracked using a tool called the SMA Independence Scale (see Feb 2024 Update below).
- Ongoing safety: the treatment was generally well tolerated. The side effects seen were mostly related to the underlying condition, not the medicine itself. Importantly, no one stopped taking risdiplam because of side effects.
These findings show that risdiplam can help people with SMA keep their motor function stable and support their ability to stay independent in daily life over the long term.
February 2024:
Independent living and measuring what matters
The latest risdiplam (Evrysdi) data updates were presented at the SMA Europe Congress in February 2024.
Roche has developed the SMA Independence Scale-Upper Limb Module (SMAIS-ULM). This has been done in close partnership with CureSMA, SMAEurope, and the SMA Foundation. The aim is to measure the level of support needed to perform activities of daily living (ADL) and to better capture the patient and caregiver experience.
At the February 2024 SMA Europe congress, Roche presented a review of the SMAIS-ULM data in Part 2 of the SUNFISH trial.
Across all sub age groups the results showed that a larger proportion of those treated with risdiplam were likely to maintain or gain independence in their daily living activities than those on placebo:
| Patient ages 12 – 25 years | Maintained or Gained Independence in Activities of Daily Living | |
|---|---|---|
| Treated with Risdiplam | Placebo | |
| Patient-reported outcome | 69.8% | 39.1% |
| Caregiver-reported outcome | 66.4% | 38.3% |
March 2023:
The latest risdiplam (Evrysdi) data updates were presented at the 2023 MDA Clinical and Scientific Conference in March 2023.
Outcomes for those with SMA Type 2 or 3, aged 2 – 25 years
Patients enrolled via Part 1 of the trial
Patients involved in Part 1 continued to receive treatment at the dose selected from the 12-week dose-finding study.
- Risdiplam significantly improved motor function after 48 months of risdiplam treatment:
- MFM-32 (Motor function measure which assesses 32 items) total change from baseline was greater in patients receiving risdiplam compared with natural history data.
Patients enrolled via Part 2 of the trial
- Risdiplam significantly improved motor function after 36 months of treatment:
- The proportion of patients demonstrating a marked improvement (change ≥3 points) or stabilisation (change ≥0 points) in MFM-32 score were more likely in people who were on risdiplam than those in the untreated group.
2022
17th March: Roche shared 3-year, longer-term data from the SUNFISH trial at the 2022 Muscular Dystrophy Association Virtual Clinical & Scientific Conference.
2021
17th March: Roche shared new, 2-year, longer-term data from part 2 of the SUNFISH trial to be presented at the 2021 Muscular Dystrophy Association Virtual Clinical & Scientific Conference.
2020
16th June: Roche presented two-year data on Part 1 of the SUNFISH trial, at the virtual Cure SMA Annual Conference.
6th February: Roche presented 1-year data from the pivotal Part 2 of their SUNFISH trial at the 2nd International Scientific and Clinical Congress on SMA in Evry, France.
2019
12th November: Roche announced positive data from part 2 of the SUNFISH trial.
May: Roche presented positive data from its SUNFISH study at the 2019 American Academy of Neurology Annual Conference.
2018
14th June: Roche announced all available screening slots in SUNFISH had been allocated by the active study centres, which meant that no new sites and countries would be activated for SUNFISH. This included the UK.
2017
11th July: PTC Therapeutics presented an update on their SUNFISH trial of their splice-modifying drug, RG7916, at the 2017 Cure SMA Conference in Orlando, USA.
2016
27th September: Roche have announced that they were to begin recruiting SMA patients to the SUNFISH trial.