We are extremely happy to share the long-awaited news: the Scottish government will be making Spinraza available to all people with spinal muscular atrophy in Scotland.



The Scottish Medicines Consortium has decided to grant the Ultra Orphan Drug designation to Spinraza, a decision that paves the way for the drug to be routinely offered on the Scottish NHS.

Starting already in April, everyone with SMA who is eligible to access the Scottish healthcare system will be able to receive Spinraza injections, thus starting the process to reversing the disease effects.

We are grateful for all those whose incessant efforts have led to this decision. Below, we are sharing a statement by Biogen, the pharmaceutical company behind Spinraza, prepared on our request.

Biogen statement to SMA community re: ultra-orphan designation for Spinraza® (nusinersen) in Scotland The Scottish Medicines Consortium (SMC) has granted nusinersen ultra-orphan designation, allowing the medicine to be reviewed in spinal muscular atrophy (SMA) types 2 and 3 (later onset) through its new ultra-orphan appraisal process. Nusinersen has been routinely available to SMA type 1 (infantile onset) patients in Scotland since May 2018, however later onset patients do not currently have routine access via the NHS. The SMC’s new process, which is more appropriate for nusinersen than the previous review route, launched in October 2018 and allows greater flexibility in evaluating medicines for severe, very rare diseases for which there is a high level of unmet need. The SMC’s decision is in line with recommendations submitted to the Scottish Government in 2016 following a review of the system to allow a greater degree of flexibility for the assessment of ultra-orphan medicines. It means ultra-orphan medicines, such as nusinersen, could be made available on the NHS in Scotland for at least three years while further efficacy data are gathered, ahead of a final decision on routine availability. Biogen is extremely encouraged by the collaboration and flexibility shown throughout the appraisal process in Scotland – the SMC took just four months to review the evidence and make their decision on access for type 1 patients. The review of nusinersen via this new process represents an increased possibility for SMA patients in Scotland beyond type 1, who are in desperate need, to access the medicine. What happens now? Biogen welcomes the opportunity to work with the SMC to formalise the data collection and reimbursement arrangements. Subject to successful sign off, nusinersen is expected to be routinely available across types 1, 2, and 3 from April 2019. Between now and April 2019, Biogen will continue to work with all stakeholders in Scotland to finalise the data collection required to aid the SMC’s future assessment of the value of nusinersen. What is the current status across the rest of the UK? Appraisals of nusinersen remain in progress in England, Wales, and Northern Ireland, with decisions expected in early 2019. Biogen is working closely with the relevant authorities to expedite timelines where possible to make the treatment available to patients as swiftly as possible, and bring equality in access with the 24 countries in Europe (and many more across the world) already making nusinersen available.

TreatSMA comment

TreatSMA shares the excitement of all the Scottish families. We are continiuing to fight for the Spinraza treatment to be available for all who live in England, Wales and Northern Ireland, the same way as it is available in Scotland and most of the developed world.