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 was the first treatment for people with SMA.
Spinraza
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Current situation
It is currently available to eligible people with SMA through a Managed Access Agreement (MAA) in England (which also covers Wales and Northern Ireland) and MDUK is a member of the Managed Access Oversight Committee (MAOC).
A MAA is a process used to collect further evidence about the effectiveness and impact of a treatment over several years following an initial appraisal by NICE. These are used when there is evidence of benefit but limited data from clinical trials and other studies, usually due to the rare nature of a condition. We were heavily involved in the initial appraisal process and were a member of the Managed Access Oversight Group.
What's next?
MAAs conclude with another appraisal by NICE. Spinraza is being appraised in 2024 at the same time as a second SMA treatment, risdiplam (also known as Evrysdi). This process is called a Multi Technology Appraisal (MTA) and it is due to conclude in late 2024. We will be working in partnership with SMA UK and Treat SMA throughout the appraisal process and in April 2024 made a joint submission to the process presenting the experience and views of the SMA community.
More information on the NICE appraisal of risdiplam and Spinraza can be found here.
Spinraza was accepted for use within NHSScotland in 2018 under the ultra-orphan process, and its availability was widened in 2019.
More information on the SMC’s appraisal of Spinraza can be found here.
Support and information
Spinal muscular atrophy
Spinal Muscular Atrophy (SMA) is a rare, genetically inherited neuromuscular condition. It causes progressive muscle weakness and loss of movement due to muscle wasting (atrophy). This may affect crawling and walking ability, arm, hand, head and neck movement, breathing and swallowing.
Research
We fund groundbreaking research to learn more about muscle wasting conditions and lead us to new treatments. We’ve already made advances that would have been unthinkable just 10 years ago, and we are determined to go even further and faster.
Our support services
We are here for everyone, but we understand that support isn’t one-size-fits-all. Speak to us. We can tailor our support to meet your unique needs.
Our helpline is open Monday-Thursday 10am-2pm.