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Onasemnogene abeparvovec-xioi

(Zolgensma®)

Zolgensma®

Drug updated on 11/4/2024

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 SMA (spinal muscular atrophy) 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

Document TitleYearSource
An updated systematic review on spinal muscular atrophy patients treated with nusinersen, onasemnogene abeparvovec (at least 24 months), risdiplam (at least 12 months) or combination therapies2024European Journal of Paediatric Neurology : Ejpn : Official Journal of the
Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data2022European Journal of Paediatric Neurology : Ejpn : Official Journal of the
Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy2022Advances in Therapy
How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison2022Journal of Comparative Effectiveness Research
Matching-adjusted indirect treatment comparison of onasemnogene abeparvovec and nusinersen for the treatment of symptomatic patients with spinal muscular atrophy type 12021Current Medical Research and Opinion
Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments2021Orphanet Journal of Rare Diseases

Clinical Practice Guidelines

Document TitleYearSource
Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy.2021Muscle & Nerve
From clinical trials to clinical practice: practical considerations for gene replacement therapy in sma type 1.2019Pediatric Neurology