Onasemnogene Abeparvovec (Zolgensma)

In the March 2021, CADTH Canadian Drug Expert Committee recommended that onasemnogene abeparvovec be reimbursed for treatment of pediatric patients with 5q spinal muscular atrophy (SMA) biallelic mutations in survival motor neuron 1 (SMN1) gene, if certain conditions were met. A request was received to develop clinical criteria could used identify SMA older than 180 days who are most likely benefit from abeparvovec. The advice this report, a expert panel, is not necessarily evidence-based, but has been developed based on experience and expertise implementation panel members and, as such, represents experience-informed opinion.