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Breaking news – Risdiplam to be made available on the NHS in Scotland

4 February 2022

A drug that helps treat Spinal Muscular Atrophy (SMA) will be funded for use on the NHS in Scotland, it was announced today. 

The news came this morning from the Scottish Medicines Consortium (SMC) and means there will now be UK-wide access to Risdiplam for those living with certain types of SMA. 

What is Risdiplam and who can access it?

Risdiplam is an oral drug manufactured by Roche. It is taken daily and increases SMN protein levels by targeting the SMN2 gene.

Risdiplam will be available to  everyone with SMA Types 1 to 3 or who have between 1 to 4 copies of SMN2. 

The SMC said this guidance will take effect 90 days after today’s announcement. 

Further details on access to Risidiplam in Scotland can be read on the SMC website here.

Those seeking guidance on access should speak with their specialist healthcare team for advice. 

Why is this news important?

SMA is a devastating condition that, prior to improved condition management and the advent of new treatments in 2016, rarely saw children with the most severe types live beyond their second birthday. 

Risdiplam can be taken at home, making it an accessible and flexible treatment option. 

Clinical evidence shows that Risdiplam improves the ability to sit up, stand or walk for people diagnosed with SMA Types 1, 2 and 3. There is also some evidence suggesting that people with type 1 SMA live for longer on Risdiplam. 

What was our role in the process?

Working closely with SMA UK, we reached out to the SMA community to hear their views on Risdiplam. 

We then represented the patient voice in October 2021 via our joint submission to the SMC, in which we advocated for the SMC to Risdiplam for use on the NHS in Scotland. 

At that stage, the SMC was not planning on recommending Risdiplam for funding. 

Roche requested a Patient and Clinician Engagement (PACE) meeting, which was held in January 2022. 

During the meeting, the SMC heard from key stakeholders including our Sheonad Macfarlane, Chair of MDUK’s Scottish Council, who shared her family’s experience of living with SMA.  

The PACE meeting also heard from our very own Dr Kate Adcock, Director of Research and Innovation at MDUK, as well as SMA UK’s Liz Ryburn. 

What do we think?

Dr Kate Adcock said: “Today’s announcement is brilliant news for families in Scotland who are affected by SMA. We’ve worked closely with SMA UK to represent the SMA community throughout this whole process and we are delighted that eligible patients in Scotland will soon be able to access Risdiplam on the NHS. We are really proud to have worked so hard alongside SMA UK to campaign for this drug to be approved for use.” 

Alistair Emslie-Smith MBE, a Trustee of SMA UK, said: “This is wonderful and life-changing news for so many with SMA in Scotland who will be eligible for treatment under the terms of this agreement – adults, for whom no treatment options have so far been available, and those children for whom the alternative therapy has not been appropriate. It brings new hope to us all. As someone living with SMA, I am proud that our Scottish SMA community, SMA UK, and MDUK have worked so well together and advocated so strongly for access to this treatment.”

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