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Nusinersen (SpinrazaTM)

(last updated 18th October 2017)

Nusinersen, now to be marketed as SpinrazaTM, is a disease-modifying therapy for the treatment of SMA, manufactured by pharmaceutical company Biogen. In Biogen’s clinical trials 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.

On 1st June 2017, SpinrazaTM 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 SMA Type 1 via a ‘compassionate use’ or Expanded Access Programme (EAP). You can read about the programme here.

Wider UK Access

Nusinersen's 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.

What is happening about access now?

We share the frustration of the SMA community that although there are now some 60 children with SMA Type 1 receiving Biogen / NHS England funded treatment via the EAP, there has still been no announcement from NICE about whether it will assess the treatment for continued and wider provision by the NHS. The UK continues to lag behind the US and Europe. The UK continues to fail the SMA community.

SMA Support UK:

  • is continuing to try to find out why there is no announcement from NICE - we had expected to hear in July
  • is talking to clinicians to see whether submitting a Policy Proposition Route (see NHSE letter) about expanding the SMA Type 1 EAP criteria would be worthwhile
  • has asked MDUK as part of their Fasttrack campaign, to highlight nusinersen in national media as a strong example of what is wrong with the system. We have asked them to work with Rare Diseases UK which has also been working hard on these wider access issues
  • has contacted RDUK and asked them to work with MDUK so that there is a strong coordinated joint lead
  • has committed to support this work
  • is concerned that Biogen has said it will review the future of the SMA Type 1 EAP at the end of this calendar year and that its decision will depend on what progress there has been with NICE’s appraisal of the treatment. We continue to do all we can to find out why this is so delayed and will urge Biogen to keep the programme open.
  • is in close contact with TreatSMA  - working in our different ways to maintain pressure and give voice to the SMA community

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

Please also see: 

Q AND AS ABOUT NUSINERSEN'S AVAILABILITY IN THE UK

What we are doing to progress access to the treatment in the UK

How you can help

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