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.


 From July 2019 nusinersen can also be prescribed for patients with types 2 and 3 SMA under the ultra-orphan pathway - see additional notes below


Download detailed advice342KB (PDF)


Medicine details

Medicine name:
nusinersen (Spinraza)

Treatment of 5q spinal muscular atrophy.

Pharmaceutical company
Biogen Idec Ltd
BNF chapter
Musculoskeletal and joint diseases
Submission type
Date advice published
07 May 2018
Additional notes

A new ultra-orphan definition and approach for assessing medicines for extremely rare conditions started in October 2018. Further information is available on our ultra-orphan process overview webpage.

Nusinersen for SMA has been categorised as an ultra-orphan according to the new definition. From July 2019 it can be prescribed for patients with types 2 and 3 SMA for a period of up to three years while further evidence on its effectiveness is generated. The company will then provide an updated submission for reassessment to allow a final decision on its routine use in this patient group in NHSScotland.   

You can find out more in the Scottish Government’s guidance on the new approach.