AveXis, which developed the treatment, announced the news on its website. You can read its press release in full here.
What is Zolgensma?
Zolgensma is a promising new one-time gene therapy for some infants with SMA Type 1. It’s still early days, but clinical trials have shown it to be effective, with some babies living longer and some even learning to stand and walk.
If Zolgensma is approved in the UK, it will be the second treatment available in the NHS for SMA Type 1, as Spinraza was approved last year. For the first time, families would have a choice of treatments.
What has the EC said?
The EC has approved the treatment "for patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene".
What happens next?
The EC's decision will be applicable to all 27 European Union member states, as well as Iceland, Norway, Liechtenstein and the United Kingdom.
In the UK, treatments need to be further approved before being available in the NHS. NICE assesses treatments for use in England, while the Scottish Medicines Consortium does so in Scotland. Wales and Northern Ireland typically follow NICE’s guidance.
What do we think?
This is an important step in making Zolgensma available in the NHS, and we're pleased progress is being made.
We will continue to work closely with other SMA charities, AveXis, and clinicians so patients in the UK can ultimately access the treatment, and to provide all the information and support the SMA community needs.