Roche’s Risdiplam Meets Primary Endpoint in SUNFISH Trial in People with SMA Type 2 or 3 SMA

12 November 2019

  • Risdiplam caused statistically significant improvements in the overall study population with SMA Type 2 or 3 
  • No treatment-related safety issues leading to study withdrawal were seen in any risdiplam trial to date
  • The data will be shared with health authorities globally

Risdiplam (previously known as RG7916) is an orally-administered, small molecule that encourages the “back-up gene”, SMN2, to produce more of the functional SMN protein. This drug is being tested in four different clincial trials: FIREFISH, SUNFISH, JEWELFISH & RAINBOWFISH (click here for more information).

The SUNFISH trial is a two-part study in people aged 2-25 years with SMA Types 2 or 3. Part 2 evaluated motor function using a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA.

Roche has just announced positive data from this part 2 of SUNFISH. The study met its primary endpoint as seen through motor function tests after one year of treatment with risdiplam, compared to placebo. No treatment-related safety issues, leading to study withdrawal, have been seen in any risdiplam trial to date. Safety for risdiplam was consistent with its known safety profile and no new safety signals were identified. Data from the SUNFISH study will be presented at an upcoming medical congress.

Further information:

The press release 

Risdiplam Clinical Trial Updates

Modulation of the SMN2 ‘Back-up’ Gene 

About the SUNFISH study