About Spinraza

Spinal muscular atrophy (SMA) is a devastating condition which, in the most severe cases, leaves babies with a life expectancy of rarely more than two years. Spinraza (also known as nusinersen) has been developed by pharmaceutical company Biogen and is the first and only treatment for people with SMA. It was approved by the European Medicines Agency (EMA) in June 2017. Clinical trials showed the treatment led to a significant improvement in children’s motor function, allowing them to achieve, or maintain, physical milestones that they would never reach without treatment, and to survive longer than expected considering the typical course of the condition. Some children who would never have sat independently have been able to and some have also been able to crawl and even walk.

Assessment of access to Spinraza in England

The National Institute of Health and Care Excellence’s (NICE) process for assessing Spinraza got underway in January 2018. Since then we have submitted evidence to NICE on the impact of the condition.

NICE held their first committee meeting on Spinraza on 27 June 2018. On 14 August 2018 NICE gave their initial decision on Spinraza which was negative. They said that a lack of data and the high cost of the treatment meant they could not recommend it for use on the NHS. This decision was subject to a consultation and then discussed at a second committee meeting in October.  

Alongside SMA Support UK, The SMA Trust and Treat SMA we are urging Biogen, NHS England and NICE to quickly reach a positive conclusion to Managed Access Agreement discussions taking place alongside the NICE assessment, which would allow access to Spinraza for a certain amount of time while further data and evidence is gathered. 

We greatly appreciate the backing of all our parliamentary supporters who have been raising awareness of this issue in questions and debates, as well as meeting families at drop-in events.

The third NICE committee meeting to discuss access to Spinraza took place on Wednesday 6 March and we are still awaiting the outcome of that meeting.

Expanded Access Programme for Spinraza

Spinraza was shown to be so effective in clinical trial that Biogen agreed to provide the drug to all children with Type 1 SMA showing symptoms before six months of age through a UK-wide Expanded Access Programme (EAP). However, the scheme only provides the treatment for a percentage of the potential beneficiaries of Spinraza and is not sustainable as a long-term solution. The EAP closed to new patients with Type 1 SMA in November 2018. All those patients originally on the programme are continuing to receive treatment. 

What assessment has been undertaken in Scotland?

The Scottish Medicines Consortium announced on 7 May 2018 that it has approved the spinal muscular atrophy (SMA) treatment Spinraza for use by children in Scotland with symptomatic SMA Type 1. This is the first treatment for SMA to receive approval for use on the NHS.

SMA has been designated a very rare disease which would mean it would be considered differently under the pathway for new medicines for very rare diseases which was recommended by the independent Montgomery Review on Access to New Medicines published in December 2016.

The pathway was introduced in October 2018 and in an announcement on 13 February 2019, Spinraza is set to be approved for Types 2 and 3 from April 2019 as part of the new pathway process.

For more information, please contact Clare Lucas on c.lucas@musculardystrophyuk.org or call 020 7803 4838.

Keep in touch