Zolgensma, a one-time gene therapy for infants with SMA Type 1, has been approved by the Scottish Medicines Consortium for restricted use within NHS Scotland.
Today’s exciting news comes following a joint submission to the SMC from Muscular Dystrophy UK and SMA UK, and is the first time a health technology appraisal of Zolgensma has been concluded in Europe.
What is Zolgensma and how is it used?
Zolgensma, also known as onasemnogene abeparvovec, addresses the genetic cause of SMA by delivering a functional copy of the SMN1 gene into nerve cells.
This gene is needed to make a protein called the survival motor neuron (SMN) protein, which is critical for the function of the nerves that control muscles.
Zolgensma is given as a single, one-time dose into the bloodstream.
Who has the SMC approved Zolgensma for?
The SMC has said Zolgensma can be given by NHS Scotland in two restricted circumstances. These are:
- For infants where both copies of the SMN1 gene are faulty and who have a clinical diagnosis of SMA type 1.
- For some affected infants before they develop symptoms of SMA.
A document outlining detailed advice can be read here and we recommend contacting your clinical team for further information on Zolgensma eligibility.
What was MDUK’s involvement in the SMC’s appraisal?
MDUK, SMA UK and a third patient group, TreatSMA, all strongly advocated for access to this treatment. Clinicians also advocated for its approval.
We submitted evidence that included families’ first-hand experiences of Zolgensma, including a parent whose daughter received treatment aged 10.5 months old.
They said: “She has made huge strides in motor skills and is able to stand independently and take a few steps with support. We continue to see improvements 1.5 years post treatment. She has no issues in terms of respiratory and swallow functions, and her fine motor skills are age-appropriate.”
What do we think?
Dr Kate Adcock, Director of Research and Innovation at MDUK, said:
“Today’s announcement that the SMC has recommended Zolgensma for restricted use within NHS Scotland truly is fantastic news.
Zolgensma is a ground-breaking one-time treatment that offers significant positive impacts on motor milestones and has the potential to improve the lives of children and their families who are affected by this rare disease.
While Zolgensma is a treatment and not a cure for SMA, today’s news means more affected children will be able to access the world around them, attend nursery and school, and join in with their family. We are delighted at the SMC’s decision.”
What is happening outside of Scotland?
In other exciting news, NHS England has today announced access to Zolgensma for eligible children who have SMA Type 1.
MDUK has been working with SMA UK, TreatSMA patient groups and clinicians to advocate for access to this innovative treatment. More information on this can be read here.
We know that the SMA community will have many queries that need answering. SMA UK has gathered questions from patient groups and has requested answers from appropriate organisations.
The questions can be read here.