nusinersen (Spinraza)

following a reassessment through the ultra-orphan framework:

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

Indication under review: for the treatment of 5q spinal muscular atrophy.

SMC restriction: Patients with symptomatic type 2 or type 3 (later-onset) 5q spinal muscular atrophy.

In a double-blind, randomised, controlled phase III study in patients aged 2 to 12 years with later-onset spinal muscular atrophy (SMA), there was a significant improvement in motor function from baseline to 15 months, assessed by the Hammersmith Functional Motor Scale Expanded, in the nusinersen group compared with the sham control group.

Improvements were maintained in an open-label extension study and were supported by real-world evidence.

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.