(last updated July 24th 2017)
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.
The SMA Type 1 Expanded Access Programme
Currently, 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). 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 this month 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. The full NICE process (including possible appeals) takes an absolute minimum of 43 weeks, and this process can take much longer. You can read more about this here.
You can also read:
- how nusinersen works here
- the full clinical trial results here
- what happens after a successful clinical trial here
- the 117 page document European Medicines Agency (EMA) Assessment Report released on 1st June with the detailed science and further details of the various clinical trials that lie behind this decision here
- Biogen’s 9th June position on price and access to treatments in Europe here
- June 6th meeting notes (NHS clinicians, managers, NHS England representatives),summarising NHSE's current position that it will not fund the administration costs of the EAP and the action plan here
- the All Party Parliamentary Group (APPG) July 18th meeting outcome which included an announcement by NHSE that, following the release of the EMA Assessment Report, they are reviewing their interim EAP policy position here
Please also see:
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.
Find out what stage other drugs have reached in our: