Risdiplam is better than nusinersen for the treatment of type 1 spinal muscular atrophy (SMA), according to a systematic literature review and indirect treatment comparison published in the Journal of Comparative Effectiveness Research.

To indirectly compare risdiplam with other approved SMA treatments, a team of researchers led by Neil Hawkins, MSc, MBA, PhD, compared individual patient data from risdiplam trials with aggregated data from published studies of nusinersen and onasemnogene abeparvovec.

The results showed that risdiplam treatment led to better survival and motor function compared to nusinersen. The researchers could not draw any concrete conclusions from the indirect comparison analyses of risidiplam and onasemnogene abeparvovec in type 1 SMA and of risdiplam and nusinersen in type 2 and 3 SMA because there were substantial differences in study populations.

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“Overall, these results support the use of risdiplam as a superior alternative to nusinersen for the treatment of patients with type 1 SMA,” the researchers wrote.

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“Further analyses in other outcomes and populations (e.g, presymptomatic patients, adolescents and adults) with forthcoming evidence from clinical trials and real-world studies may provide additional evidence of comparative effectiveness.”

Risdiplam is an oral, disease-modifying treatment approved by the US Food and Drug Administration in 2020 to treat patients with SMA aged 2 months and older.

It works by including exon 7 in the mRNA from the SMN2 gene, thereby increasing the amount of functional SMN protein made from this gene. 

Other disease-modifying treatments for SMA are the splice corrector nusinersen and the gene therapy onasemnogene abeparvovec. Nusinersen must be administered intrathecally because it cannot cross the blood-brain barrier, while onasemnogene abeparvovec is given as a single intravenous infusion.


Ribero VA, Daigl M, Martí Y, et al. How does risdiplam compare with other treatments for types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison. J Comp Eff Res. Published online January 18, 2022. doi:10.2217/cer-2021-0216