All your questions answered on Spinraza

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Spinraza SMA treatment

We have heard today that Spinraza, the highly effective treatment for spinal muscular atrophy (SMA), has not been recommended for NHS use in draft guidance by the National Institute for Health and Care Excellence (NICE).

We know that this is a complex topic and so have compiled the below information to help you understand what the treatment is, what today’s decision means, and what we need to see happen to get access now.

You can read more about SMA and the support and information MDUK offers for those living with the condition here.

Spinraza is currently being assessed by NICE, after it released an appraisal consultation document giving its initial opinion of the treatment as negative. Giving the reasons for its recommendation, NICE says existing data for Spinraza does not show the impact of the treatment on all types of SMA and the long-term benefits. NICE does, however, recognise that Spinraza provides “important health benefits” for patients with early-onset and later-onset SMA.

Muscular Dystrophy UK and the SMA charities are calling for NICE to approve the treatment for children with the most severe form of the condition. We would also like to see a temporary scheme – known as a managed access agreement – put in place for people with other types of SMA, so long-term evidence can be collected on its effectiveness.


Developed by pharmaceutical company Biogen, Spinraza is the first and only treatment for SMA, and has already proven to be highly effective for many.

Without access to Spinraza, children with SMA will irreversibly lose the ability to perform simple tasks, and babies with the most severe form of the condition – SMA Type 1 – are unlikely to see their second birthday.

Clinical trials have shown significant improvement in children’s motor function; for the first time, children with SMA Type 1 are able to crawl and even walk. Families of children who have received the drug often report noticeable improvements from the start of treatment. The drug proved so effective in clinical trials with children with SMA Type 1 that in August 2016 they were stopped early so all the children on the trial could access the drug.

The drug also has promise for children and adults with the other types of SMA; motor function for children with SMA Type 2 and 3 was significantly improved in trials, although data so far submitted to NICE refers only to children.

While the drug was highly effective in most treated children, there were some who did not respond to the treatment. Some patients may also be too weak to receive treatment.

Children with SMA Type 1 can currently access Spinraza – but for most it is through a scheme with no guarantees of future provision.


England, Wales and Northern Ireland: children access the drug through an Expanded Access Programme (EAP) set up by Biogen in 2016 following successful clinical trials. This provides the drug for free while assessments into its provision are made. However, there are no guarantees that the EAP will continue; most other countries now fund access, leaving England looking increasingly out of step.

Spinraza is currently being appraised by NICE for use on the NHS in England. NICE gave the drug a negative opinion in an appraisal consultation document published on 14 August and is due to make a final recommendation in November 2018.

Scotland: in May, the Scottish Medicines Consortium (SMC), the Scottish equivalent of NICE, approved Spinraza for use on the NHS for children with SMA Type 1, replacing the EAP.


Children and adults with SMA Type 2, 3 and 4 do not currently have access to the treatment on the NHS in the UK.

While other countries have now arranged access for at least those with SMA Type 1, patients in England, Wales and Northern Ireland remain reliant on the EAP scheme put in place by the drug company.

Part of the delay has been because of the unsuitability of NICE’s appraisal routes. There are only two: a general route, through which Spinraza would be too expensive; and a specialist treatment route, unsuitable for Spinraza as it has a wider applicability than a specialist treatment. This is not the first time that a treatment to help people with muscle-wasting conditions has been heavily delayed. We need to see an overhaul of these routes to ensure rare disease drugs aren’t held up in future.

We await updates on other possible routes to access for Spinraza:

  • a managed access agreement (MAA), to provide interim access while NICE’s lengthy procedures are conducted, was promised at the end of 2017 but is yet to have any details released about when such a scheme would come into force and who would be eligible. There are more details on this below.
  • the government announced a scheme to fast-track approval for five treatments a year in November 2017. Muscular Dystrophy UK has lobbied hard for treatments like Spinraza to be eligible for the scheme, but there has been no further update.

An MAA is a temporary scheme that would allow patients with SMA to access Spinraza, so that data can be gathered on the treatment’s effectiveness without stopping access. The aim of an MAA is to temporarily deliver a treatment through the NHS while a longer-term agreement to provide it is agreed. The length of time a MAA is implemented can vary, but three to five years is typical.

Muscular Dystrophy UK has worked closely with SMA Support UK, The SMA Trust and Treat SMA to lobby for access for all those who could benefit from access.

There is a wealth of data that shows Spinraza to be clinically effective, and we are calling on NICE to approve the drug for babies with SMA Type 1. We would also like to see an MAA put in place for children with SMA Type 2 and 3 so that further evidence can be gathered on Spinraza’s effectiveness.

Muscular Dystrophy UK met with the Health Minister Steve Brine in July 2018 to put across our concerns, and he promised to request an urgent update. We are also calling for an urgent meeting with Biogen to discuss next steps to ensure continued access to Spinraza.

NICE has acknowledged that an MAA was being looked at for Spinraza but the committee said that ‘the details of the company’s proposed MAA were vague and currently insufficient for it to assess whether it could be an option’. It was recognised that an MAA could reduce the risk for the NHS, but that such an agreement would have to carry the support of NHS England, patients, carers and clinicians. However, NICE also commented that any MAA would still require the cost to be within a range that could be considered cost-effective.

Before individual European countries examine a treatment, they are first of all licensed by the European Medicines Agency (EMA). Spinraza was recommended in April 2017 by the EMA to be licensed as a treatment for SMA Types 1, 2, 3, 4. In June 2017, the European Commission finalised the recommendation.

Spinraza is available in 20 European countries for children and some adults with all types of SMA. Negotiations are under way in other countries. More widely, Spinraza was approved in the United States in December 2016, and since then has gained additional approvals in South Korea, Canada, Japan, Brazil and Australia.

Spinraza is administered through an injection into the spinal canal in an established procedure known as an ‘intrathecal injection’. This delivers medication through the lower back via a lumbar puncture and directly into the central nervous system. This procedure is performed under the direction of healthcare providers experienced in administering lumbar punctures and requires special precautions – either sedation or general anaesthetic.

The list price of Spinraza is £75,000 per vial. At list price, the total annual treatment cost is £450,000 for the first year and £225,000 for subsequent years. However, the final prices for treatments delivered on the NHS are subject to commercial negotiations that are not made public, and so the actual cost would remain unknown. Biogen proposed a commercial arrangement, which would apply if the technology had been recommended.

Two trials, called ENDEAR and CHERISH, were carried out by Biogen. There are also three ongoing studies: NURTURE, SHINE and EMBRACE.

In August 2016, the drug proved so effective in clinical trials with children with SMA Type 1 that they were stopped early so that all the children on the trial could access the drug. Biogen then opened its EAP for Spinraza, offering the drug free to all children with SMA Type 1 who showed signs of SMA before six months of age. Clinical data indicates that Spinraza has shown, in some cases, to improve survival and motor function.

Read our news story to find out what you can do, including taking part in a consultation on the treatment.

  • AAP: Accelerated Access Programme. A government scheme to fast-track breakthrough treatments announced in November 2017. We are waiting to hear if rare disease drugs will be eligible for it.
  • EAP: Extended Access Programme. A special scheme currently providing access to Spinraza for those with SMA Type 1 from the pharmaceutical company Biogen.
  • EMA: European Medicines Agency. Pharmaceutical treatments typically need to get a licence for marketing a drug from the EMA before they are assessed NICE.
  • HST: Highly Specialised Technology route. One of the two routes, along with STA, through which NICE assess treatments.
  • MAA: Managed Access Agreement. This is an agreement to provide treatment on the NHS while more data is gathered on its efficacy, and we want to see one put in place for Spinraza.
  • NICE: National Institute for Health and Care Excellence. NICE is in charge of recommending whether treatments should be funded on the NHS.
  • PACE: Patient and Clinical Experts. A special meeting held by the SMC to garner views.
  • SMA: spinal muscular atrophy.
  • SMC: Scottish Medicines Consortium. The SMC is in charge of recommending treatments for provision on the NHS in Scotland.
  • STA: Single Technology Appraisal route. The main route through which NICE assesses treatments.


  • August 2016: the drug proves so effective in clinical trials with children with SMA Type 1 that they are stopped early so that all the children on the trial can access the drug. Biogen then opens its EAP for Spinraza, offering the drug free to all children with SMA Type 1 who showed signs of SMA before six months of age.
  • 21 April 2017: the drug is recommended by the EMA to be licensed as a treatment for SMA Types 1, 2, 3, 4.
  • 1 June 2017: the European Commission finalises the recommendation by the EMA.
  • August 2017: NHS England agrees to support delivery costs for Spinraza, but only for children with two copies of the SMN2 gene (around 80 percent of children), as these were the only children involved in the clinical trials.
  • November 2017: the Department of Health announces a new, fast-track route into the NHS for ‘breakthrough’ medicines and technologies, known as the Accelerated Access Pathway (AAP). Five drugs a year will benefit – but no criteria are given and the scope of the scheme is unclear.
  • November 2017: the SMC starts its appraisal of Spinraza.
  • January 2018: NICE announces it will begin its appraisal of Spinraza for SMA Types 1, 2, 3 and 4 through its Single Technology Appraisal (STA) route. Discussions of an interim scheme, known as an MAA, also gets underway between manufacturers Biogen, NHS England, and NICE.
  • 13 March 2018: families and charities make submissions and give evidence on the benefits of Spinraza at a Patient and Clinical Experts (PACE) meeting held by the SMC.
  • 3 April 2018: the SMC holds a second meeting for final submissions from charities and families on why Spinraza should be approved in Scotland.
  • 7 May 2018: the SMC’s appraisal of Spinraza is published. It recommends it for use on the NHS in Scotland for children with Type 1 SMA – but not Types 2 and 3.
  • 14 August 2018: NICE publishes its appraisal consultation document for Spinraza, setting out provisional recommendations for not approving the treatment for use on the NHS in England. This was originally expected to be released in June.


Find out more about how you can help us fight for access for Spinraza.

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