Drug updated on 12/11/2024
Dosage Form | Infusion (intravenous: 5.5 mL, 8.3 mL) |
Drug Class | Adeno-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.
Latest News
Summary
- 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 Title | Year | Source |
---|---|---|
Zolgensma (onasemnogene abeparvovec-xioi) Prescribing Information. | 2024 | Novartis Gene Therapies, Inc., Bannockburn, IL |
Systematic Reviews / Meta-Analyses
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy. | 2021 | Muscle & Nerve |
From clinical trials to clinical practice: practical considerations for gene replacement therapy in sma type 1. | 2019 | Pediatric Neurology |