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Page last updated: 17th March 2023

Our Advocacy and Access Diary

9th January: NICE announces that recently, a small number of children treated in England, particularly those who are older and weighing more than 13.5 kg, have been noted to have suspected significant adverse drug reactions affecting the liver following the administration of Zolgensma. In view of this, NHS funded treatment with Zolgensma in England is temporarily paused in children older than 12 completed months as per NICE guidance. Wales, Northern Ireland and Scotland follow this advice while the MHRA investigates further.

9th February: The NICE Committee Meeting re the review of Zolgensma treatment for pre-symptomatic SMA is taking place today. We are very grateful to both the patient and clinical experts speaking and hope for a positive outcome. The publication date for the final decision is expected on 19th April.

16th March: NICE reported that Zolgensma is now recommended as an option for treating pre-symptomatic SMA in babies aged 12 months and under, who have up to 3 copies of the SMN2 gene. Previously, babies diagnosed symptomatically could only access Zolgensma through an MAA.

15th June: NICE opened its review of the one year Managed Access Agreement (MAA) of pre-symptomatic zolgensma. In response to comments, NICE has positively commented: "the committee will consider the entire presymptomatic population (1-3 SMN2 copies). If evidence allows, consideration by SMN2 copy number may be considered. The scope has been updated to reflect this". See final scope.

11th August: SMA UK and MDUK make joint submission and nominate possible patient expert(s) willing to speak at the NICE committee meeting scheduled for 9th February 2023. The selection of experts finally rests with NICE.

8th March: In the morning, NHS England announced landmark agreement for access to Zolgensma for eligible children who have SMA Type 1.

In the afternoon, the Scottish Medicines Consortium announced access for: infants where both copies of the SMN1 gene are ‘faulty’ and who have a clinical diagnosis of SMA Type 1; pre-symptomatic infants where both copies of the SMN1 gene are ‘faulty’, and up to 3 copies of the SMN2 gene, who are expected to develop SMA Type 1.

13th April: SMA UK responded to NICE’s evaluation consultation supporting their recommendations. We acknowledged that no current evidence or modelling was presented to demonstrate that Zolgensma could also provide health benefits for a proportion of children with SMA Type 2 and 3 and urged Novartis Gene Therapies to continue to work to address this gap in access to the treatment.

21st April: SMA UK hosted Zolgensma Treatment for SMA Type 1 webinar with leading health professionals focusing on access in England

7th July: NICE issued final guidance.

November: ‘Zolgensma: Six Months On’ webinar with leading Health Professionals from the Zolgensma National Multidisciplinary Team (NMDT) discuss how the programme is going with Liz Ryburn from SMA UK.

6th January: AveXis advised NICE of an update to their regulatory timings. NICE advised therefore, that the first committee discussion scheduled to take place on Thursday 26th March will not now take place.

AveXis started to roll out its Global Compassionate Use Programme this month for children age under 2 years who have SMA. It accepted clinical referrals and then globally randomly allocatee 100 doses of Zolgensma a year via a fortnightly draw. SMA Europe’s statement set out its concerns.

5th March: Following SMA Europe’s January statement about the Global access programme. AveXis global teams had been meeting with clinical and patient representatives from SMA Europe and from the SMA Asia Pacific Alliance to discuss alternative options. SMA REACH’s statement confirmed that the UK would not take part.

27th March:

European Commission recommends Zolgensma for conditional approval.

SMA UK requests NICE to include appraisal as a therapeutically-critical topic in covid-19 impacted work programme.

3rd April: NICE confirms appraisal of Zolgensma is not defined as therapeutically critical

19th May: European Commission report approves zolgensma.

13th July: To facilitate the clinical introduction and use of Zolgensma, a group of prominent European neuromuscular experts provided eleven consensus statements covering many aspects of treatment.

28th August: NICE widens scope of its appraisal of zolgensma in line with the European marketing authorisation to include those who have 5qSMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene. If evidence allows, consideration may be given to a sub-group of people who are pre-symptomatic. This widens the possibilities and is what SMA UK and other patient groups had been advocating.

8th October: SMA UK’s and MDUK’s two patient experts join two others nominated by TreatSMA and the Ally Cadence Trust and two clinical experts to give evidence to NICE’s first committee meeting.

9th December: SMA UK & MDUK make joint submission to the Scottish Medicines Consortium strongly supporting access. Our Patient Representatives later attended the Patient and Clinician Engagement (PACE) meeting.

28th May: AveXis received USA approval for Zolgensma treatment for children with SMA age 2 years or less.

10th June: NICE announces appraisal of Zolgensma for infants with SMA Type 1 will follow the highly specialised technology (HST) route.

25th June: SMA UK launches survey asking the community for their views on the possible provision of Zolgensma by NHS England for infants who have SMA Type 1.

8th August: SMA UK and MDUK’s joint access to Zolgensma – the survey we ran, summary of the drug treatment, survey results and submission sent to NICE.

20th December: AveXis announced limited global access to Zolgensma in January 2020 for eligible children under 2 years of age. Clinicians and SMA patient groups in SMA Europe welcomed the news, but had concerns about how it would operate. AveXis agreed to further discussions in the new year but remained committed to the launch. It was not known if UK regulations would allow UK patients to be part of this programme. Eligible children in this age group were able to access nusinersen treatment.

19th September: NICE Opens Draft Scoping Consultation for AveXis Gene Therapy for SMA Type 1.

17th October: SMA UK’s submission to NICE’s Scoping Consultation on AVXS-101 treatment for infants with SMA Type 1 asks that it follows the highly specialised technology (HST) route.

6th November: Our Support Team Manager attended NICE’s scoping meeting for AVXS-101 advocating for an HST route for the appraisal and speaking up for access.