Safety update: risdiplam clinical trial program for spinal muscular atrophy

<h3></h3> The risdiplam clinical development program consists of four trials: FIREFISH (NCT02913482) in Type 1 SMA (inclusion criteria [IC]: aged 1–7 months at enrollment) SUNFISH (NCT02908685) in Types 2/3 SMA (IC: aged 2–25 years at enrollment) JEWELFISH (NCT03032172) in SMA patients previously receiving RG7800, nusinersen, olesoxime or onasemnogene abeparvovec (IC: aged 6 months–60 years at enrollment) RAINBOWFISH (NCT03779334) in pre–symptomatic SMA (IC: birth–6 weeks of age at first dose) Previous analyses of 465 patients with symptomatic SMA from FIREFISH, SUNFISH and JEWELFISH (clinical cut-off dates [CCODs] 23 Nov 2021, 6 Sep 2021 and 31 Jan 2022, respectively) and 18 presymptomatic SMA patients from RAINBOWFISH (CCOD 1 Jul 2021) showed that risdiplam has a favourable safety profile, with no treatment-related adverse events (AEs) leading to treatment withdrawal. No serious AEs (SAEs) were reported in RAINBOWFISH where the AE profile appeared reflective of age. In symptomatic SMA patients, the MedDRA standard organ classes with the highest number of AEs were infections and gastrointestinal AEs. The overall rate of AEs decreased with continued treatment. SAEs declined in Type 1s but remained stable in Types 2/3. Here we will present updated safety analyses from the risdiplam studies.