August is SMA Awareness Month, and we’re continuing our Every Piece of the Puzzle campaign, celebrating the people, partnerships and milestones that have shaped SMA UK and the wider SMA community. This piece of the puzzle is an overview to securing access to Zolgensma.
Read more detail about access to Zolgensma here.
The journey to securing access to Zolgensma for people living with SMA has been a long one, involving years of advocacy, consultation and collaboration between patient organisations, clinicians, NHS bodies and regulators.
Since 2018, SMA UK has worked alongside families, healthcare professionals and partners including Muscular Dystrophy UK (MDUK) and TreatSMA to ensure the voices of the SMA community were heard throughout the appraisal process. From responding to consultations and gathering patient evidence to speaking at committee meetings and hosting educational events, we have consistently championed equitable access to this life-changing treatment.
2018: The appraisal process begins
In September 2018, NICE opened its draft scoping consultation for AveXis’ gene therapy, then known as AVXS-101, for infants with SMA Type 1.
SMA UK submitted a response calling for the treatment to be assessed through NICE’s Highly Specialised Technologies (HST) programme, recognising the rarity and severity of SMA. In November, our Support Team Manager attended NICE’s scoping meeting to advocate for the HST route and highlight the importance of timely access for families.
2019: Gathering evidence from the SMA community
Momentum continued in 2019 when Zolgensma received approval in the United States for children with SMA aged two years and under.
In June, NICE announced that it would evaluate Zolgensma through the HST programme. To support this process, SMA UK launched a community survey to gather the experiences and views of people affected by SMA. Working with MDUK, we submitted this evidence to NICE in August, ensuring that lived experience played a central role in the appraisal.
Towards the end of the year, AveXis announced a limited global access programme for eligible children. While patient organisations across Europe welcomed the opportunity, there were significant concerns about how the programme would operate fairly, and it remained unclear whether UK families would be able to participate.
2020: Advocacy through a challenging year
The COVID-19 pandemic brought additional challenges to the appraisal process. Committee meetings were delayed following changes to regulatory timelines, and the planned NICE discussions did not proceed as originally scheduled.
Meanwhile, the European Commission recommended and later granted conditional approval for Zolgensma, expanding momentum across Europe.
Throughout the year, SMA UK continued to advocate for the broadest possible access. We successfully called for NICE to widen the scope of its appraisal to include people with 5q SMA who have a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene. This represented an important step towards recognising the needs of pre-symptomatic infants as well as those already showing symptoms.
Our patient experts, nominated jointly with MDUK, gave evidence at NICE’s first committee meeting in October, sharing the real-world impact of SMA and the importance of timely treatment. Later that year, SMA UK and MDUK also made a joint submission to the Scottish Medicines Consortium in support of access in Scotland.
2021: A landmark year for access
March 2021 marked a major milestone.
NHS England announced a landmark agreement that made Zolgensma available for eligible children with SMA Type 1. On the same day, the Scottish Medicines Consortium announced access for eligible infants with SMA Type 1, as well as pre-symptomatic infants with up to three copies of the SMN2 gene who were expected to develop the condition.
SMA UK welcomed these decisions while continuing to advocate for broader access. In our response to NICE’s consultation, we highlighted the lack of evidence available for children with SMA Types 2 and 3 and encouraged Novartis Gene Therapies to continue developing the evidence needed to address this gap.
Throughout the year, we also supported families by hosting webinars with leading healthcare professionals, explaining how access would work in England and providing updates six months after implementation from the Zolgensma National Multidisciplinary Team.
2022: Reviewing access for pre-symptomatic treatment
In June 2022, NICE began its review of the one-year Managed Access Agreement (MAA) for pre-symptomatic use of Zolgensma.
Following consultation responses, NICE confirmed that it would consider the entire pre-symptomatic population with one to three copies of the SMN2 gene, rather than limiting its review to specific subgroups.
SMA UK and MDUK submitted further evidence in August and nominated patient experts to contribute to NICE’s committee discussions, continuing our commitment to ensuring that patient experience remained central to decision-making.
2023: Expanding access
In March 2023, NICE recommended Zolgensma as a treatment option for pre-symptomatic babies aged 12 months and under who have up to three copies of the SMN2 gene.
This decision represented another significant milestone, extending routine access beyond the previous Managed Access Agreement and ensuring that eligible babies could receive treatment before symptoms develop, when outcomes have the greatest potential to improve.
Looking ahead
The story of Zolgensma demonstrates the power of sustained collaboration between families, clinicians, researchers and patient organisations.
While significant progress has been made, SMA UK continues to advocate for equitable access to treatments and ongoing research that improves outcomes for everyone affected by SMA. Every consultation, evidence submission, patient story and expert contribution has helped shape the treatment landscape we see today, and we remaincommitted to ensuring that people living with SMA can benefit from advances in care as they become available.
Community Story
“However, this isn’t just Ollie’s story, as he is now one of many children who has received Zolgensma in the UK. We know that if children are treated from birth, then the onset of symptoms can be stopped in its tracks, and that’s why advocating for newborn screening has been such an important part of our lives since we learnt about SMA.”
