The Medicines and Health Products Regulatory Agency (MHRA) has given marketing authorisation to Evrysdi®▼(risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in individuals with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
This means that those eligible are now able to receive risdiplam from birth where previously they could only receive treatment from 2 months of age.
What does this mean for people living with SMA in the UK?
Now all individuals with types 1-3 SMA can receive risdiplam from birth. In England, Northern Ireland and Wales eligible individuals can receive risdiplam from birth under the existing managed access agreement (MAA), whereas, those in Scotland are able to access risdiplam via the NHS and this extension will be automatically incorporated. This may take a few weeks to be rolled out to new patients.
What does this mean for the MAA?
The MAA will close in 2025 as planned when evidence will be re-assessed by the National Institute for Health and Care Excellence (NICE). We are working with NICE to ensure we have all the data they need to make a thorough and fair assessment and will provide updates to you as and when able.
