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Page last updated: 23rd January 2023

Our Advocacy and Access Diary

1st June: The U.S. Food and Drug Administration (FDA) approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with SMA. This meant that risdiplam is now approved in the US to treat SMA in children and adults of all ages. The data behind this decision had been submitted to the European Medicines Agency (EMA).

7th February: The Scottish Medicines Consortium (SMC) announced that the NHS in Scotland will fund those who are 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. This followed assessment by the SMC of a full submission under their orphan drug appraisal process.

January 2022: A Patient and Clinician Engagement (PACE) meeting was held. This had been requested byt the pharmaceutical company when the SMC in October 2021, said it was not minded to recommend risdiplam for funding .

It seemed this PACE meeting made a critical difference to the decision-making. Maria Elena Farrugia, Consultant Neurologist at Glasgow (adults), was a key advocate for access, as were Joan Turner and Sheonad Laidlaw who shared their experiences of living with SMA and accessing risdiplam so powerfully, and Kate Adcock (MDUK) and Liz Ryburn (SMA UK).

"As someone living with SMA myself and a Trustee of SMA UK I am proud that our Scottish SMA community, our charity and MDUK have worked so well together and advocated so strongly for access to this treatment."

Alistair Emslie-Smith MBE, SMA UK Trustee

“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.”

Dr Kate Adcock, MDUK’s Director of Research and Innovation

16th December: NICE issues final guidance giving the go ahead for risdiplam

10th December: SMA UK releases Risdiplam Treatment webinar to cover the many questions coming from the community.

4th October: SMA UK and MDUK made their joint submission to the Scottish Medicines Consortium. This included:

  • the community’s responses to our recent survey
  • information from our autumn 2020 community surveys and submission to NICE – of the 137 survey responses, 10 were from people from Scotland
  • our June 2021 consultation response to NICE which included results of UK-wide surveys on the impact of SMA (including January 2018 survey results from Scotland).

2nd September: We sent a survey to the Community so that we could include people’s views in our submission

13th August: SMC announces decision to resume review of risdiplam with a deadline for submissions of 4th October 2021.

3rd August: SMA UK, MDUK and TreatSMA wrote to the SMC to raise their concerns about the further delays with processing Roche’s submission for risdiplam.

13th July: Our Support Team Manger was one of the Patient Experts at the second NICE committee

September 2020 – July 2021: We worked with local neuromuscular teams, Trusts and Roche, wherever there were access challenges for people with SMA Type 1 or 2 who were eligible for the Risdiplam Early Access to Medicines Schemes – with some considerable success. When the scheme closed, 241 people in the UK had access.

23rd June: SMA UK responded to the NICE Consultation.

2nd June: NICE says ‘no’ to recommending risdiplam for NHSE funding but asks for comments on its consultation paper.

21st May: Risdiplam was given GB marketing authorisation from the MHR. Roche announces it will submit an application in June for NHS provision in Scotland. The EAMS scheme for people with SMA Type 1 or 2 for whom nusinersen is not clinically suitable continued for those already enrolled. The EAMS now closed to new people, unless they had been identified before the MHRA announcement.

11th May: Our Support Team Manger was one of the Patient Experts at the first NICE committee meeting. Our contribution included the reading of a powerful statement from a Ally Allen, parent of a child who has SMA Type 3 and had to date been excluded from all treatments.

30th March: Risdiplam received marketing authorisation in the European Union (EU) for the treatment of 5q SMA in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3, or with 1-4 SMN2 copies. Under the terms of the UK’s departure from the European Union, EU marketing authorisation applied to Northern Ireland. Risdiplam remained an unlicensed medicine in the rest of the UK.

29th January: SMA UK worked with REACH clinicians to answer the community’s many questions about access to the risdiplam EAMS.

23rd December: Following considerable advocacy efforts by SMA UK and other patient groups, Cambridge University Hospital NHS Trust joined the EAMS bringing the total of Trusts taking part to more than 15.

30th October: SMA UK’s & MDUK make their Joint Submission to NICE Re: Risdiplam.

23rd September: Roche answered questions collated by SMA UK from MDUK, Treat SMA and the community about access to the EAMS / Compassionate Use Programme

17th September: The Medicines and Healthcare Products Regulatory Agency (MRHA) announced access to Risdiplam for those who have SMA Type 1 or Type 2, aged 2 months and older, who are not suitable for authorised treatments.

28th August: NICE announced it would re start its review of risdiplam with a view to publishing its guidance on 21st July 2021.

17th August: The European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) for risdiplam for the proposed use in patients with SMA. EMA granted the marketing application with accelerated assessment, which meant that the evaluation process could be reduced from 15 to 9 months, if the accelerated assessment timetable is maintained. This led to a planned compassionate use eligibility criteria adaptation in the UK, to include SMA Type 2 patients who have exhausted all therapeutic options.

7th August: The U.S. Food and Drug Administration (FDA) approved risdiplam for the treatment of 5q SMA in adults and children 2 months of age and older

20th February: SMA UK and MDUK submit Scoping Response to NICE.

25th November: FDA grants priority review to risdiplam for the treatment of SMA.

17th December: The European Medicines Agency grants risdiplam prime designation.

17th January: RG7916 granted orphan drug status in the US.