Drug updated on 5/17/2024

Dosage FormSolution (oral; 60 mg [0.75 mg/mL])
Drug ClassSurvival of motor neuron 2 splicing modifiers
Ongoing and
Completed Studies


  • Indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

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  • Risdiplam (Evrysdi) is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It has shown significant improvements in motor function among patients with SMA types 1 and 2/3 after a year of treatment, enhancing oral feeding capability and head control.
  • The efficacy and safety of risdiplam in treating SMA were analyzed in five systematic reviews/meta-analyses.
  • Compared to nusinersen, another drug used for treating SMA, risdiplam demonstrated statistically significant improvements in metrics such as the Hammersmith Functional Motor Scale - Expanded (HFMSE), Revised Upper Limb Module (RULM), though with lesser magnitude. An indirect comparison suggested that risdiplam could potentially offer superior survival rates and improved motor function than nusinersen specifically for Type 1 SMA.
  • In terms of the safety profile, no statistically significant increase in adverse events or severe adverse events was observed between treatments using risdiplam compared to those using nusinersen or onasemnogene abeparvovec. Approximately 16% of participants experienced adverse effects during trials involving risdiplam; however, due to the lack of reported cases, it is difficult to quantify the incidence rate of serious side effects.
  • Efficacy analyses included a wide range of patient ages/types, ranging from pediatric to adult with type 1 through type 3 SMAs, indicating potential consideration for early initiation to maximize clinical benefit since outcomes tend to be partly dependent on the stage of disease at which treatment is initiated.
  • Comparisons between risdiplam and onasemnogene abeparvovec, as well as nusinersen, were challenged by substantial differences in study populations, making it hard to draw definitive conclusions about their relative effectiveness based solely on these indirect comparisons.