Summary

This AI-generated content is provided without warranty, with no liability accepted for reliance on it. Learn more.

• 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.