(Onasemnogene abeparvovec-xioi)


Drug updated on 11/16/2023

Dosage FormInfusion (intravenous: 5.5 mL, 8.3 mL)
Drug ClassAdeno-associated virus vector-based gene therapy
Ongoing and Completed StudiesClinicalTrials.gov


  • 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.

Product Monograph / Prescribing Information

Document TitleYearSource
Zolgensma (onasemnogene abeparvovec-xioi) Prescribing Information.2023Novartis Gene Therapies, Inc., Bannockburn, IL

Systematic Reviews / Meta-Analyses

Document TitleYearSource
Assessment report: Zolgensma.2020EMA