Risdiplam (also known as Evrysdi) is a treatment for SMA, manufactured by Roche.
Risdiplam
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How does it work?
It is an oral syrup medicine and can be taken once a day after meals. The liquid drug works by modifying the SMN2 gene to produce increased amounts of SMN protein, which is essential for the health of nerve cells that control muscle movement.
Current situation
Risdiplam is currently available in England, Wakes and Northern Ireland through a Managed Access Agreement (MAA). This 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. Risdiplam is being appraised in 2024 at the same time as a second SMA treatment, spinraza (also known as nusinersen). 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.
In February 2022, risdiplam was accepted for use within NHS Scotland under the orphan process.
More information on the SMC’s appraisal of Risdiplam 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.
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