Clinical review report: Onasemnogene abeparvovec (Zolgensma) (Novartis Pharmaceuticals Canada Inc.) : indication: for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and: 3 or fewer copies of SMN2 gene; or infantile-onset SMA.
Onasemnogene abeparvovec (Zolgensma) has a Health Canada indication for the treatment of pediatric patients with 5q SMA with bi-allelic mutations in the SMN1 gene and 3 or fewer copies of the SMN2 gene or infantile-onset SMA. Onasemnogene abeparvovec is a non-replicating, recombinant, adeno-associat...
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| Format: | eBook |
| Language: | English |
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Ottawa (ON)
Canadian Agency for Drugs and Technologies in Health
May 2021, 2021
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| Edition: | Version: final (with redactions) |
| Series: | CADTH common drug review
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | Onasemnogene abeparvovec (Zolgensma) has a Health Canada indication for the treatment of pediatric patients with 5q SMA with bi-allelic mutations in the SMN1 gene and 3 or fewer copies of the SMN2 gene or infantile-onset SMA. Onasemnogene abeparvovec is a non-replicating, recombinant, adeno-associated virus vector that utilizes the adeno- associated virus serotype 9 (AAV9) capsid to deliver a stable, fully functional human SMN transgene. Onasemnogene abeparvovec is given as a 1-time intravenous infusion with a recommended dose 1.1 × 1014 vector genomes (vg)/kg. In addition, prednisolone treatment should be given at 1 mg/kg per day (or equivalent) 1 day prior to infusion and continued for 30 days, after which it can be subsequently tapered over 28 days |
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| Physical Description: | 1 PDF file (111 pages) illustrations |