Summary

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• Evrysdi (risdiplam) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
• This summary is based on the review of five systematic review(s)/meta-analysis(es). ^[1-5]
• CHOP-INTEND (SMA Type 1): After 12 months of treatment with risdiplam, 57% of participants achieved a CHOP-INTEND score ≥ 40 points. Over half of these participants were able to feed orally and maintain head control.
• Motor Function Measures (SMA Type 2/3): Risdiplam increased MFM32 scores by 2.09 points, RULM scores by 1.73 points, and HFMSE scores by 1.00 point. However, the effect on respiratory function in this group was inconsistent.
• Comparison with Nusinersen: For SMA Type 2/3, nusinersen resulted in greater increases in HFMSE (4.90 points) and RULM (3.70 points) compared to risdiplam's 0.87 and 1.73 points, respectively. Nusinersen also increased HINE-2 scores by 5.21 points.
• Adverse Events: 16% of participants treated with risdiplam experienced adverse events. Serious adverse events could not be quantified due to a lack of reported cases.
• Comparison with Other Drugs: No statistically significant differences were observed between risdiplam and nusinersen in the rate of adverse events (OR: 0.93; 95% CI: 0.51, 1.7; p = 0.82) or severe adverse events (OR: 0.77; 95% CI: 0.47, 1.27; p = 0.31).
• The evidence source includes studies on SMA phenotypes Type 1 and Types 2/3, with varying effectiveness of risdiplam observed by phenotype, particularly in motor function improvements for Type 1, and no specific subgroup safety concerns identified, but adverse events reported in 16% of participants without detailed subgroup-specific data.