Roche posts positive SUNFISH pivotal results for risdiplam

Swiss pharma giant Roche (ROG: SIX) has presented one-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA).

The study showed that change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32) was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). The Revised Upper Limb Module (RULM), a key secondary endpoint, also showed an improvement (1.59 point difference; p=0.0028). Safety for risdiplam in the SUNFISH study was consistent with its known safety profile. Data were presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy from February 5-7 in Evry, France.

Under development with SMA and PTC Therapeutics