Nusinersen, now to be marketed as SpinrazaTM, is a disease-modifying therapy for the treatment of SMA, manufactured by pharmaceutical company Biogen.
On 1st June 2017, it was approved by the European Commission “for the treatment of 5q spinal muscular atrophy”. This refers to a mistake in a gene on the fifth chromosome in the chromosomal region labelled ‘q’ which affects those with SMA Types 1, 2, 3 and 4. This is the first treatment ever for SMA to reach this stage.
You can read the 117 page document with the detailed science and full details of the various clinical trials that lie behind this decision here.
If you already know about nusinersen, you may want to go straight to these pages for an update on what is happening now:
If this is all new to you, we suggest the summaries below may be helpful:
Clinical Trial Results
In Biogen’s open-label, uncontrolled studies in individuals who had, or were likely to develop childhood-onset SMA Types 1, 2 or 3, some individuals treated with nusinersen showed improvement, including:
- achieving physical milestones which they would not have reached without treatment
- maintaining physical milestones which they would not have done without treatment
- surviving longer than expected considering the typical course of their condition
There have been no clinical trials with people with adult onset SMA Type 4.
The SMA Type 1 Expanded Access Programme
Currently (June 2017), nusinersen is only potentially available in the UK to children with onset of symptoms of SMA before six months of age (consistent with SMA Type 1) via a ‘compassionate use’ or Expanded Access Programme (EAP).
As access to this programme is evolving all the time, it is not possible to keep an accurate record on this website of which centres are accepting eligible children from which geographical areas for the EAP. Talk first to your medical team about what is possible for your child in your area. You can also contact us to see if we have any further updates to help you. Phone 01789 267 520 or email email@example.com
Although Biogen provides the drug free of charge, lack of capacity in centres and lack of a national system for allocating places equitably to eligible children has prevented the EAP being rolled out fully to all those children who could benefit. You can read more about the EAP, including what we are doing about advocating for equitable access here.
Wider UK Access
We hope to know by June / July 2017 if, when and how the treatment will be assessed for provision in the UK. Its availability depends on a recommendation by the National Institute for Health and Care Excellence (NICE), NHS England, the Scottish Medicines Consortium and other authorities in the devolved nations to decide whether to fund the drug in England, Scotland, Wales and Northern Ireland. Availability may also depend on the treatment readiness of specialist centres. You can read more about this here.
Find out what stage other drugs have reached in our: