Onasemnogene Abeparvovec (Zolgensma)

Abstract

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