following a full submission considered under the ultra-orphan process:
nusinersen (Spinraza®) is accepted for restricted use within NHSScotland.
Indication under review: for the treatment of 5q spinal muscular atrophy (SMA).
SMC restriction: patients with symptomatic type 1 SMA (infantile onset)).
In randomised, controlled, phase III studies of children with SMA, nusinersen treatment was associated with significant improvements in motor function compared with a sham injection. In infants with type I SMA, nusinersen significantly prolonged the time to permanent assisted ventilation or death.
This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of nusinersen. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
The license holder has indicated their intention to resubmit.
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- Medicine name:
- nusinersen (Spinraza)
- SMC ID:
- Treatment of 5q spinal muscular atrophy.
- Pharmaceutical company
- Biogen Idec Ltd
- BNF chapter
- Musculoskeletal and joint diseases
- Submission type
- Full submission
- Date advice published:
- 07 May 2018