NHS England Announces Access to Zolgensma for Eligible Children who have SMA Type 1

08 March 2021

NHS England’s announcement of this landmark agreement is positive news for parents of some children who have SMA Type 1 who may be eligible for access to this one-time gene therapy treatment.

NHS England has said it is: "...ready to fast-track the introduction of the highly complex and innovative gene therapy and will not wait until NICE publish final guidance to get going. This approach is backed by NICE given the importance of administering the one-off treatment as early as possible."

Read NHS England’s announcement

Read Novartis Gene Therapies' Community Statement

Is my child eligible?

We expect the following children to have access to this new service:

  • infants newly diagnosed with SMA Type 1 up to 12 months of age (those aged 7-12 months upon recommendation of a National Multidisciplinary Team (MDT));
  • any pre-symptomatic infants identified who have two ‘faulty’ SMN1 genes, up to 3 SMN2 copies and are likely to develop SMA Type 1

We understand that NHS England’s agreement states:

"The terms of the deal mean that some young children that currently fall outside the NICE recommendation criteria will also be eligible to be considered for treatment by a national multidisciplinary clinical team (MDT) made up of the country’s leading experts in the treatment of SMA.

This means as many as 80 babies and young children could potentially benefit from the life-changing gene therapy a year.

At the moment, we don’t know what criteria the MDT will agree such as if a child is currently receiving any treatment. We are aware the European clinical consensus statement suggests a safe upper weight limit for treatment of 13.5kg. 

We don’t have any timeframe for how long it will take to set up the MDT. We have asked these questions and will publish the answers as soon as we have them.

Setting up the Service

We know every day matters and ‘time is neurons’, but we do also know that it will take time to set up access to this new treatment. Specialist ‘gene-therapy treatment Centres’ in England are in the process of being approved to provide this brand-new national service. 

We don’t yet have any timeframe for how long it will take the Centres to start treatment or the rate at which children will be able to be treated. We have asked these questions and will publish the answers as soon as we have them.

What can I do now?

We know this will be an anxious time for families whose child might be eligible for treatment. We don’t have any guidance yet on anything you can do now, but clinicians know all families who have a child who has SMA Type 1. This announcement is likely to be new to clinicians who will need to work with the MDT to find out eligibility criteria and what happens next with referral processes. We will publish any information or guidance we receive about this.

What will happen in Scotland, Wales and Northern Ireland?

We are expecting the Scottish Medicines Consortium to make their recommendation on Monday 8th March at 2pm. At the moment we don’t know what paths Wales and Northern Ireland will follow. They usually follow NICE guidance but may follow Scotland.

Further questions and thoughts

SMA UK, MDUK, TreatSMA patient groups and clinicians all strongly advocated for access to Zolgensma treatment.

In anticipation of a positive decision, we had already gathered a wide range of questions from all patient groups about this treatment and how it would be delivered. These have been sent to NHS England, Novartis Gene Therapies and the clinical network. As soon as we have answers we will publish them. If you have further questions, please do let us know so that we can pass them on.

We are very fortunate in the UK to have clinicians already with experience of delivery of the treatment via the clinical trials. They have been planning for the possibility of this service being set up for some time. They and our other expert clinicians across the country are in close contact with international colleagues, so we can be assured that when the MDT does make treatment decisions, they will be well informed in terms of both clinical safety and potential benefits for each child.

SMA UK believes this ground-breaking gene therapy offers real hope for the future. We truly welcome this decision, so parents in England whose children are covered by this agreement will now be able to discuss a choice of NHS-funded drug treatments with their clinician.

We are however aware that many across the UK are still struggling to get access to treatment that should be available to them or still have no access to treatment at all. As a matter of urgency, we will continue to advocate for clinically safe and potentially beneficial treatment options to be both approved for and delivered to all.

Further information:

What is Zolgensma and how does it work?