Peer-Reviewed Publication

A systematic review/indirect treatment comparison of risdiplam versus other treatments for Type 1 - 3 SMA

How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison

Valerie Aponte Ribero, Monica Daigl, Yasmina Martí, Ksenija Gorni, Rachel Evans, David Alexander Scott, Anadi Mahajan, Keith R Abrams, Neil Hawkins


To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). 

Patients & methods: 

Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. 


In Type 1 SMA, studies of risdiplam and nusinersen included similar populations. Indirect comparison results found improved survival and motor function with risdiplam versus nusinersen. Comparison with onasemnogene abeparvovec in Type 1 SMA and with nusinersen in Types 2/3 SMA was challenging due to substantial differences in study populations; no concrete conclusions could be drawn from the indirect comparison analyses. 


Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA.