nusinersen (Spinraza)

following a full submission assessed under the orphan medicine process:

nusinersen (Spinraza®) is accepted for restricted use within NHSScotland.

Indication under review: for the treatment of 5q Spinal Muscular Atrophy.

SMC restriction: for use in patients with pre-symptomatic 5q SMA.

In a phase II single-arm study, 80% of pre-symptomatic patients with a genetic diagnosis of 5q Spinal Muscular Atrophy (SMA) who received treatment with nusinersen did not require respiratory intervention. Additionally, 92% of patients achieved the motor milestone of walking alone. Comparative evidence against risdiplam or onasemnogene abeparvovec is lacking.

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.