Drug updated on 9/4/2024
Dosage Form | Solution (oral; 60 mg [0.75 mg/mL]) |
Drug Class | Survival of motor neuron 2 splicing modifiers |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
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Summary
- 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.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Evrysdi (risdiplam) Prescribing Information. | 2024 | Genentech, Inc., South San Francisco, CA |
Systematic Reviews / Meta-Analyses
Document Title | Year | Source |
---|---|---|
Efficacy of risdiplam in spinal muscular atrophy: A systematic review and meta-analysis. | 2023 | Pharmacotherapy |
Safety and efficacy of nusinersen and risdiplam for spinal muscular atrophy: a systematic review and meta-analysis of randomized controlled trials. | 2023 | Brain Sciences |
Evaluation of the therapeutic efficacy and tolerability of current drug treatments on the clinical outcomes of paediatric spinal muscular atrophy type 1: a systematic review. | 2023 | Paedeatric Respiratory Reviews |
Efficacy of risdiplam in spinal muscular atrophy: a systematic review and meta-analysis. | 2023 | Pharmacotherapy |
How does risdiplam compare with other treatments for types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison. | 2022 | Journal of Comparative Effectiveness Research |