nusinersen (Spinraza)

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.

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From July 2019 nusinersen (Spinraza) can be prescribed for patients with types 2 and 3 SMA within the ultra-orphan pathway while further evidence on its effectiveness is generated. At the end of the data collection period the company will provide an updated submission for reassessment to allow a decision on its routine use in NHSScotland.