Promising treatment for SMA Type 1 available on the NHS in England

MDUK is delighted to bring you the news that a highly complex, innovative gene therapy is to become available on the NHS in England for the treatment of spinal muscular atrophy (SMA) Type 1.

Babies born with SMA Type 1, the most common form of the condition, are unable to sit or roll unaided. Studies have shown that after a single infusion treatment of Zolgensma, babies with SMA Type 1 were able to reach milestones such as breathing without a ventilator, sitting up on their own, and crawling and walking.

MDUK has been working with SMA UK, TreatSMA patient groups and clinicians to advocate for access to this innovative treatment. Dr Kate Adcock, MDUK’s Director of Research and Innovation said:

This truly exciting announcement comes in the wake of promising clinical trial results. Zolgensma is a one-off treatment that delivers the missing SMN1 gene to nerve cells, and making it available will change lives. As many as 80 babies and young children who are born every year with this rare condition could potentially benefit from the life-changing gene therapy.

“We’re delighted that NHS England and the manufacturer, Novartis, have reached an agreement; this will bring hope to families in England facing this diagnosis. Until we know more about the detail of how families will get access to the treatments, we suggest families speak to their consultants.”

NHS England announced today (8 March) that they’re ready to fast-track the introduction of Zolgensma, which they describe as a ‘life-saving’ gene therapy. They want to make this treatment available as early as possible, without waiting until NICE publishes its final guidance. NICE has backed this approach, given the importance of administering Zolgensma as early as possible.

With this new deal, some young children with SMA Type 1 who currently fall outside of NICE’s recommendation criteria will also be eligible for consideration for treatment. A national multi-disciplinary team comprising leading UK experts in the treatment of SMA will assess each child.

NICE has also today opened a consultation on its draft recommendations for the use of Zolgensma on the NHS. We will be working with SMA-UK on a formal response to the consultation. If you’d like to get involved, you can find out more on the NICE website.