Roche ’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy

Basel, 6 February 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented 1-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)[1]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),[2] 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 5-7 February in Evry, France. As anticipated, exploratory subgroup analyses showed that the strongest responses in MFM-32 versus placebo were observed in the youngest age group (2-5 years) (78.1% vs 52.9% achieving ≥3 point increase). Importantly, disease stabilisation was observed in the 18-25 years age group (57.1% vs 37.5%, with stabilisation defined as a ≥0 point increase), which is the goal of treatment for those with more established disease.“Risdiplam is the first potential treatment to have pivotal placebo-controlled data in a broad population of patients, including children, teenagers and adults,” said SUNFISH principal investigator Eugenio Mercuri, M.D., Ph.D....
Source: Roche Investor Update - Category: Pharmaceuticals Source Type: news