NICE, NHS and Roche Agree a 3-year Managed Access Agreement for Risdiplam for SMA Types 1, 2 or 3

19 November 2021

We’re absolutely delighted that Risdiplam, this third treatment for SMA, will now be possible in England via a Managed Access Agreement (MAA) for individuals who are 2 months or older with a diagnosis of SMA Types 1, 2 or 3 or who have a diagnosis of SMA, are pre-symptomatic and have between one to four SMN2 copies.

All the documents the National Institute of Health and Care Excellence (NICE) has published about this recommendation, including their Final Appraisal Document (FAD), the Managed Access Agreement (MAA) and Appendix A about the use of disease modifying medicines for SMA after onasemnogene abeparvovec (Zolgensma) treatment, are on their website here. This recommendation is currently subject to the possibility of an appeal by stakeholders. If there is no appeal, NICE expects to issue final guidance on 16th December.

We have asked NHS England & Improvement (NHSE&I), Roche and clinicians many questions about the MAA and the roll-out of this programme. They have given us as much information as possible at the moment. If there is no appeal, the programme should start be rolled out from 15th January 2022:

The hard work and advocacy for access by SMA UK, MDUK, TreatSMA, all the SMA Community and clinicians has paid off. Our thanks to everyone who responded to surveys, made submissions and spoke at committee meetings. We continue to work for a similar outcome in Scotland and access in all devolved nations as well as the introduction of newborn screening for SMA.

You can read the comments and press releases of key stakeholders and others here:

Access across the UK

Risdiplam offers children, young people and adults who live with this progressive and complex neuromuscular condition, an oral medication that can be taken daily at home to combat the deterioration they otherwise face. Many are powerchair users who have shielded throughout the pandemic and face huge challenges with travel to hospital for treatment - Risdiplam offers so much more flexibility in people’s lives. We continue to work for a similar outcome in Scotland and access in all devolved nations. We understand that Wales and Northern Ireland are likely to follow NICE’s recommendation. 

Newborn Screening is a must

The full transformative potential of risdiplam and all treatments for SMA won’t be realised until we have newborn screening (NBS) for SMA. Every five days in the UK, a baby is born with the condition. If treatments available now are delivered at birth, these children have the best chance of long and healthy lives. We urge the UK National Screening Committee to swiftly progress their review of the case for newborn screening in the UK.

More about what’s happening about NBS for SMA in the UK