Drug updated on 9/4/2024
Dosage Form | Infusion (intravenous: 5.5 mL, 8.3 mL) |
Drug Class | Adeno-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
- 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 Title | Year | Source |
---|---|---|
Zolgensma (onasemnogene abeparvovec-xioi) Prescribing Information. | 2023 | 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 |