NICE widens scope of its appraisal of Zolgensma
This will now be in line with the European marketing authorisation and will now also include those who have 5q SMA a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene and, if evidence allows ,consideration may be given to a sub-group of people who are pre-symptomatic. This widens the possibilities and is what patient groups have been advocating.
NICE will evaluate clinical trial and cost effectiveness evidence and we would expect them to note the European clinical consensus on its use.
Muscular Dystrophy UK has been working with other patient and clinical representatives ahead of the committee meeting, which will be held on 8 October 2020. The expected publication date on the NHS website is 25 March 2021.