We’re delighted to announce that NICE’s appraisal committee has today published its final evaluation determination document. Subject to any appeal by consultees, this may be used as the basis for NICE’s guidance on the use of Zolgensma in the NHS in England. This follows NICE’s evaluation of the clinical trial evidence and cost-effectiveness. As we have previously reported, Zolgensma is a potentially life-changing gene therapy for spinal muscular atrophy (SMA) Type 1.
Four centres in England will offer Zolgensma, a single-dose infusion that offers significant positive impacts on motor milestones. It has the potential to improve the lives of children born with this very rare disease, which affects nerve cells in the spinal cord, affecting movement and, sometimes, breathing. Without treatment, children born with SMA Type 1 rarely see their second birthday.
Zolgensma addresses the genetic cause of SMA by delivering a functional copy of the SMN1 gene into nerve cells. This gene is critical for the function of the nerves that control muscles.
This gene therapy has been available on the NHS in Scotland since March; you can read more about that here. MDUK, SMA UK and a third patient group, TreatSMA, all advocated for access to this treatment, as did clinicians working with families affected by SMA.
For more detailed information, please read this Q&A. It was developed with input from the company that makes Zolgensma, clinicians and NHS England.