Two treatments for spinal muscular atrophy (SMA), nusinersen and risdiplam, will now be permanently available on the NHS after several years of limited access to assess their effectiveness and value for money.
For years, the SMA community has been pushing for access to nusinersen (Spinraza) and risdiplam (Evrysdi). While these treatments were initially recommended by the National Institute for Health and Care Excellence (NICE) in 2019 and 2021 respectively, access was restricted through Managed Access Agreements (MAAs).
Under these agreements, treatment was available only to certain groups and for a fixed period, during which additional data was collected. MAAs are used when there is not yet enough evidence to support routine use, allowing more evidence to be generated before NICE reassesses the evidence. Throughout this period, we worked closely with people living with SMA and the charities SMA UK and Treat SMA to ensure that patient experience and real-world impact were captured and reflected in decision making.
Today’s announcement means there are now three treatment options available for people with SMA, including Zolgensma (onasemnogene abeparvovec), a one-time gene therapy. This progress reflects the strength and persistence of the SMA community – and the life-changing difference these treatments are making to people’s lives.
“Within a few days of starting risdiplam, my arms felt stronger. I could feel myself breathing more deeply. And I started being able to eat foods I hadn’t managed in nearly a decade due to my weak swallow.”
Access to nusinersen and risdiplam is expanding compared with eligibility under the MAAs. People who are permanently ventilated will now be able to access either treatment.
There are some differences in eligibility between the two treatment options:
However, treatment with risdiplam or nusinersen will not be routinely available after treatment with Zolgensma, unless the gene therapy is considered not to have been effective.
This decision means that people living with SMA will now have greater certainty about long term access to treatment, and more opportunity to make informed choices about their care over time.
Following approvals for both treatments in Scotland, today’s announcement means access to all SMA treatments is moving towards UK‑wide availability, as Wales and Northern Ireland typically align with NICE decisions. This is an important step towards consistent, equitable care for everyone living with SMA right across the UK.
Over the last couple of weeks, four treatments for muscle wasting and weakening conditions have been approved by NICE, following decisions on treatments for Duchenne muscular dystrophy and myasthenia gravis.
A decade ago, people living with muscle wasting conditions had no treatments designed specifically for their condition. These recent decisions mean there are now ten. This is a major milestone – one that would have felt unimaginable not so long ago – and it reflects the persistence, determination and collective voice of a community that has worked tirelessly to ensure the realities of living with a muscle wasting condition are understood, heard and reflected in decision‑making.
But progress cannot stop here. Everyone living with a muscle wasting condition deserves timely access to treatments that may benefit them, and we will continue to support research and advocate for equitable access until that is possible for all.
“This is incredibly positive news for people living with spinal muscular atrophy and their families. Permanent approval of nusinersen and risdiplam brings long awaited certainty and recognises the difference these treatments are making in people’s lives. It also reflects years of determination from the SMA community, alongside clinicians and charities, to ensure lived experience sits at the heart of decision making.
Taken together with other recent approvals, this progress highlights why we must keep pushing until everyone living with a muscle wasting condition can benefit from effective treatments.”
Becca lives with spinal muscular atrophy (SMA) type 2. She shares her reflections on how the treatment landscape has changed in her lifetime, the improvements she’s seen from Risdiplam, and the importance of improving access to treatment for all.
