Onasemnogene abeparvovec-xioi

(Zolgensma®)

Onasemnogene abeparvovec-xioi

Drug updated on 9/4/2024

Dosage FormInfusion (intravenous: 5.5 mL, 8.3 mL)
Drug ClassAdeno-associated virus 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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  • Zolgensma (onasemnogene abeparvovec-xioi) is 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.
  • This summary is based on the review of six systematic review(s)/meta-analysis(es). [1-6]
  • Onasemnogene abeparvovec demonstrated significant improvements in motor function, particularly in presymptomatic participants, with CHOP INTEND scores increasing by 11.06 points at 3 months and 14.14 points at 6 months post-infusion. Motor milestones, such as sitting independently and walking, were more frequently achieved compared to nusinersen.
  • Indirect comparisons indicated that risdiplam may offer improved survival outcomes compared to nusinersen, although the comparison with onasemnogene abeparvovec was challenging due to study heterogeneity. Onasemnogene abeparvovec showed a favorable effect on event-free survival and overall survival compared to nusinersen.
  • Common adverse events reported included pyrexia, vomiting, thrombocytopenia (79.3%), and elevated aminotransferases (71.7%), with greater incidence in patients older than 8 months.
  • Drug-related adverse events were noted post-treatment, but detailed safety outcomes were not consistently specified across all studies, with some reporting rare occurrences.
  • 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.2023Novartis Gene Therapies, Inc., Bannockburn, IL

Systematic Reviews / Meta-Analyses

Clinical Practice Guidelines