NICE Widens Scope of its Appraisal of Zolgensma

28 August 2020

This will now be in line with the European marketing authorisation and will now also include those who have 5qSMA with a bi-allelic mutation in the SMN1 gene and up to 3 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. SMA UK and other patient and clinical representatives will take part in the committee meeting on 8th October. The expected publication date, on the NHS website is 25th March 2021.