What is the purpose of our SUNFISH clinical trial?

SUNFISH is a two-part study evaluating risdiplam in people with Type 2 and 3 SMA between 2 and 25 years of age. It is placebo controlled, with two out of every three participants receiving risdiplam and one receiving placebo.

The objective of Part 1 was to assess the safety profile and concentration of risdiplam at different dose levels as well as the level of SMN protein in blood. Part 1 has been completed and the participants from Part 1 of SUNFISH are continuing to receive risdiplam and are now enrolled in the open-label extension phase of the study.

Part 2 is the pivotal part of the study and will assess efficacy and safety of risdiplam after 12 months of treatment. Enrolment in SUNFISH Part 2 is complete.