Summary

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• This summary is based on the review of five systematic review(s)/meta-analysis(es). ^[1-5]
• Efficacy in Motor Function Improvement: Risdiplam demonstrated statistically significant improvements in motor function measures, including the Hammersmith Functional Motor Scale Expanded (HFMSE) and the 32-item Motor Function Measure (MFM32), across phenotypes 1 and 2/3 (spinal muscular atrophy (SMA)) patients. In the RAINBOWFISH study, presymptomatic infants with two or three the survival motor neuron 2 gene (SMN2) copies showed motor milestones achievement, with some delays noted for those with two copies. In Type 1 SMA, risdiplam improved survival and motor function compared to nusinersen.
• Systematic Review Findings: Motor function improvements were consistently observed with earlier treatment initiation, regardless of therapy (nusinersen, onasemnogene abeparvovec, risdiplam). However, respiratory function improvements were inconsistent, and no significant changes in nutritional outcomes were reported.
• Indirect Treatment Comparisons: While risdiplam showed improved motor function and survival in Type 1 SMA compared to nusinersen, comparisons with onasemnogene abeparvovec were inconclusive due to differences in study populations.
• Risdiplam was associated with a 16% incidence of adverse events, similar to other SMA therapies such as nusinersen and onasemnogene abeparvovec, with no statistically significant differences in the occurrence of severe adverse events between risdiplam and nusinersen.
• Serious adverse events (SAEs) were rarely classified as treatment-related across therapies, with post-lumbar puncture syndrome frequently reported in nusinersen studies but not risdiplam.
• There is no population types or subgroups information available in the reviewed studies.