NICE publishes its Final Evaluation Document on Zolgensma

04 June 2021

Today NICE has confirmed as follows:

Onasemnogene abeparvovec is recommended as an option for treating 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1 in babies, only if:

  • they are 6 months or younger, or
  • they are aged 7 to 12 months, and their treatment is agreed by the national multidisciplinary team.

It is only recommended for these groups if:

  • permanent ventilation for more than 16 hours per day or a tracheostomy is not needed
  • the company provides it according to the commercial arrangement (see section 3).

For babies aged 7 to 12 months, the national multidisciplinary team should develop auditable criteria to enable onasemnogene abeparvovec to be allocated to babies in whom treatment will give them at least a 70% chance of being able to sit independently. 

Onasemnogene abeparvovec is recommended as an option for treating presymptomatic 5q SMA with a bi-allelic mutation in the SMN1 gene up to 3 copies of the SMN2 gene in babies. It is recommended only if the conditions in the managed access agreement are followed.

You can read the publication and why it made these recommendations here.

There is now an appeal period closing on 18th June. Final publication is scheduled for 7th July 2021.

NHS England’s parallel landmark agreement remains in place.

For more information on progress of the roll out of the programme:

Updates from the National Multidisciplinary Team

England Q and As