Roche Presents New Two-Year Data From SUNFISH (Part 2) Trial

17 March 2021

Roche has shared new, 2-year, longer-term data from part 2 of the SUNFISH clinical trial of risdiplam. These data will be presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference taking place from 15th to 18th March.

SUNFISH part 2 is an ongoing, global study evaluating the efficacy and safety of risdiplam in people aged 2-25 years, who have SMA Type 2 or who are non-ambulant and have SMA Type 3. The study is placebo-controlled, which means that patients were treated with risdiplam or with placebo then risdiplam (patients in the placebo group received the placebo for 12 months, followed by risdiplam treatment for 12 months).

These new results of nearly a year of additional follow-up, show that people treated with risdiplam continue to demonstrate sustained benefit from the treatment. People who have SMA Types 2 and 3 who remain untreated, normally show a decline in motor function over time.

In more detail, the gains in motor function seen after 12 months of risdiplam treatment either continued to increase or were maintained at month 24 across a number of primary and secondary endpoint measures. In addition, the number of serious adverse events, high-grade adverse events and treatment-related adverse events observed, decreased in the second year versus the first year, further demonstrating the favourable safety profile of risdiplam.

Further information

Roche's press release

About risdiplam