Drug updated on 3/28/2024
| Dosage Form | Solution (oral: 60 mg powder for reconstitution) |
| Drug Class | Survival of motor neuron 2 splicing modifiers |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.
Summary
- Risdiplam (Evrysdi) is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.
- Two systematic reviews/meta-analyses were reviewed to gather information about this drug's efficacy and safety profile.
- The first review found that after 12 months of Evrysdi treatment, 57% of participants with SMA type 1 achieved a score greater than or equal to 40 points on the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders, indicating improved motor function.
- In patients with SMA types 2/3, risdiplam was shown to increase scores on various measures including the Motor Function Measure by an average range from approximately one point up to three points; however its impact on respiratory function was inconsistent across studies.
- Adverse events related to Evrysdi occurred in around 16% percent participants according to one study but serious adverse events could not be quantified due lack data availability suggesting it has a relatively safe profile overall based upon available evidence.
- An indirect comparison between risidiplam and other approved treatments such as nusinersen and onasemnogene abeparvovec suggested that risidiplam may offer superior survival rates and motor function improvement compared specifically against nusinersen in Type-1 SMA cases although comparisons against other drugs were inconclusive due population differences among trials studied.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Evrysdi (risdiplam) Prescribing Information. | 2022 | 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 |
| 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 |
| Clinical and pharmacoeconomic combined report: risdiplam (Evrysdi). | 2021 | CADTH |
| Assessment report: Evrysdi. | 2021 | EMA |