NICE Announces Appraisal of the Treatment Zolgensma for Infants with SMA Type 1

10 June 2019

NICE is to review whether it will recommend ZolgensmaTM (under its full name: onasemnogene abeparvovec) for provision by NHS England for the treatment of infants with SMA Type 1.

SMA UK will be making a submission to NICE by the deadline of 31st July. We will be asking the Community for their views on this treatment and working with the other patient groups. NICE’s first committee meeting to review AveXis’ application and all the submissions will be on 31st October 2019.

In our submission at the scoping stage of this process we advocated that it be considered as a Highly Specialised Technology (HST) and are pleased to say NICE came to this conclusion. You can read our submission here.  We also attended the scoping meeting to further voice this view.

NICE will post updates of any progress with its work on this ‘topic’, here.

Read more about the NICE process and what happens in the rest of the UK, here.