onasemnogene abeparvovec (Zolgensma)

following a full submission assessed under the orphan medicine process:

onasemnogene abeparvovec (Zolgensma®) is accepted for restricted use within NHSScotland.

Indication under review: treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.

SMC restriction: for the treatment of

• patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or
• pre-symptomatic patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene, where patients are expected to develop SMA type 1

In a phase III study of patients with symptomatic SMA type 1 treated with onasemnogene abeparvovec, survival was significantly better than a historical control cohort. In addition, motor milestones achieved generally exceeded the natural history of SMA type 1.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.