Onasemnogene Abeparvovec (Zolgensma)
Keyword(s):
CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments, with the exception of Quebec. This review assesses onasemnogene abeparvovec (Zolgensma), given as a single-dose IV infusion of 1.1 × 1014 vector genomes/kg Indication: For the treatment of pediatric patients with 5q SMA with biallelic mutations in the survival motor neuron 1 (SMN1) gene and: 3 or fewer copies of the SMN2 gene or infantile-onset SMA