Roche's risdiplam meets goals in spinal muscular atrophy trial

Swiss pharma giant Roche (ROG: SIX) today announced positive data from the pivotal Part 2 of SUNFISH, a study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA).

The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment with risdiplam, compared to placebo. No treatment related safety findings 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.

“The positive outcome of this trial is an important milestone for people with Type 2 or 3 SMA, too many of whom remain untreated,” said Dr Levi Garraway, Roche’s chief medical officer and head of global product development. “SUNFISH is the largest placebo-controlled study ever undertaken in Type 2 or 3 SMA patients. We thank the SMA community for their partnership and look forward to sharing these results with regulators and bringing risdiplam to people living with this condition,” he added.

Competitors in SMA space