A meta-analysis of the efficacy and safety of the onasemnogen abeparvovec in SMA

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Brazilian researchers have compiled data from the literature on one of three innovative drugs designed to treat children with type 1 spinal muscular atrophy (SMA1). The onasemnogene abeparvovec (Zolgensma®) is the first gene therapy product to have marketing authorisation for this indication:

  • the authors selected four publications corresponding to three clinical trials (START, STR1VE-US and STR1VE-EU), none of which was comparative,
  • with a minimum follow-up of 12 months, the survival rate of infants treated was 97.56%,
  • the CHOP-INTEND score was greater than or equal to 40 in 87.28% of children,
  • slightly more than half of them experienced treatment-related adverse events.

Although the efficacy of Zolgensma® has been well confirmed, the risk of complications associated with this new type of therapy and the lack of hindsight in these studies are matters for concern.

 

Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis. Fernandes BD, Krug BC, Rodrigues FD et al. PLoS One. 2024 May 7;19(5):e0302860.

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