Journal Article

SUNFISH Part 2: 24-month efficacy and safety of risdiplam in Type 2/3 SMA

Vuillerot, Carole, Baranello, Giovanni, Deconinck, Nicolas, Kletzl, Heidemarie, Mazzone, Elena, Yeung, Winnie, Martin, Carmen, Servais, Laurent, Gerber, Marianne

2022

Overview

SUNFISH (NCT02908685) is a multicentre, two-part, randomised (2:1, risdiplam:placebo), placebo-con- trolled, double-blind study in individuals with Type 2/3 spinal muscular atrophy (SMA; inclusion criteria 2–25 years at enrolment). SUNFISH investigates efficacy and safety of risdiplam, a centrally and periph- erally distributed oral survival of motor neuron 2 pre-mRNA splicing modifier. Risdiplam (EVRYSDI™) has been approved by the US Food and Drug Administration for the treatment of individuals with SMA, aged 2 months and older.In Part 1 (N=51) the safety, tolerability and pharmacokinetics/pharmacodynamics of different risdiplam dose levels were assessed. Part 2 (N=180) assessed the efficacy and safety of the Part 1-selected dose of risdiplam versus placebo in Type 2 and non-ambulant Type 3 SMA. Individuals received risdiplam or placebo for 12 months; all individuals then received risdiplam until Month 24, when they had the oppor- tunity to enter the open-label extension phase.In Part 2, the primary outcome of the study was met, showing a statistically significant difference in change from baseline in 32-item Motor Function Measure total score at Month 12 between individuals treated with risdiplam (n=120) and placebo (n=60). No treatment-related safety findings leading to withdrawal were reported. Here we present SUNFISH Part 2 data after 24 months’ [e-mail]16

• Publisher: BMJ Publishing Group Ltd,BMJ Publishing Group LTD
• Subject: ABN Abstracts 2021 / Neuromuscular diseases