Drug updated on 10/28/2024
| Dosage Form | Solution (oral; 60 mg [0.75 mg/mL]) |
| Drug Class | Survival of motor neuron 2 (SMN2) splicing modifiers |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
Latest News
Summary
- 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 HFMSE and MFM32, across phenotypes 1 and 2/3 (SMA) patients. In the RAINBOWFISH study, presymptomatic infants with two or three 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.
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 |
|---|---|---|
| Systematic Review of Presymptomatic Treatment for Spinal Muscular Atrophy | 2024 | International Journal of Neonatal Screening |
| An updated systematic review on spinal muscular atrophy patients treated with nusinersen, onasemnogene abeparvovec (at least 24 months), risdiplam (at least 12 months) or combination therapies | 2024 | European Paediatric Neurology Society |
| Efficacy of risdiplam in spinal muscular atrophy: A systematic review and meta-analysis | 2024 | 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 |
| 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 |