Onasemnogene abeparvovec-xioi

(Zolgensma®)

Onasemnogene abeparvovec-xioi

Drug updated on 12/11/2024

Dosage FormInfusion (intravenous: 5.5 mL, 8.3 mL)
Drug ClassAdeno-associated virus (AAV) vector-based gene therapies
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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Summary
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  • This summary is based on the review of six systematic review(s)/meta-analysis(es). [1-6]
  • Onasemnogene abeparvovec (Zolgensma) demonstrated motor function improvement in spinal muscular atrophy (SMA) patients, particularly in Type 1 SMA, where it showed a favorable effect in achieving motor milestones (e.g., independent sitting, walking) compared to nusinersen, though no significant respiratory or nutritional improvements were noted.
  • In SMA Type 1, onasemnogene abeparvovec provided longer event-free survival (EFS) and a higher likelihood of motor milestone achievement than nusinersen, with indirect comparisons suggesting continued benefits over nusinersen at 24 months.
  • Earlier treatment initiation and higher baseline function across SMA types correlated with better motor function outcomes, consistently observed across treatments, including onasemnogene abeparvovec, nusinersen, and risdiplam.
  • Adverse events were common across treatments, though seldom classified as treatment-related; post-lumbar puncture syndrome was frequently reported in nusinersen studies.
  • No significant safety concerns or specific adverse effects were highlighted for onasemnogene abeparvovec compared to other treatments.
  • There is no population types or subgroups information available in the reviewed studies.

Product Monograph / Prescribing Information

Document TitleYearSource
Zolgensma (onasemnogene abeparvovec-xioi) Prescribing Information.2024Novartis Gene Therapies, Inc., Bannockburn, IL

Systematic Reviews / Meta-Analyses

Clinical Practice Guidelines