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Page last updated: 29th March 2023

As a result of the National Institute of Health and Care Excellence (NICE) final guidance, issued in July 2021, which is on their website, the following children may access an assessment for this treatment:

(a) Children with SMA Type 1 up to 6 months of age.

(b) Children with SMA Type 1 over 6 months of age and below 12 months of age. (NICE states in its guidance that treatment should be given to babies in whom treatment will give them at least a 70% chance of being able to sit independently. They have given the National Multidisciplinary Team (NMDT) the task of developing what criteria will assist them with this prediction).

NHS England’s agreement, clarified in March 2021, enables the following groups of children to have access to an assessment for Zolgensma treatment:

(c) Children with SMA Type 1 who are over 12 months old within the scope of the drug’s European Medicines Agency (EMA) marketing authorisation. (This eligibility was reinstated following a temporary pause from 9th January– 28th March 2023 while the Medicines and Healthcare product Regulatory Agency – MHRA – investigated and resolved safety concerns:

UK Investigation into Zolgensma safety concerns 9th January – 28th March 2023

(d) Children with SMA Type 1 who have been using treatment e.g., with nusinersen (Spinraza) or Risdiplam (Evrysdi) and fall within the scope of the European Medicines Agency marketing authorisation.

(e) NICE’s review of the one-year Managed Access Agreement for access to Zolgensma by pre-symptomatic infants, completed on 15th March 2023, made permanent the possibility of access to Zolgensma by any identified pre-symptomatic SMA infants aged 12 months and under who have a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.


The age bands and any other criteria that apply to each group:

  • Aged six months and younger refers to calendar months and includes children below 7 months of age (e.g. if a child is born on the 1st January 2021, they will be 7 months old on the 1st August).

NICE’s Final Determination Document states that for this group:

"Children who are having more than 16 hours per day of non-invasive ventilation, or who have a tracheostomy, are not eligible."

  • Aged 7 – 12 months refers to calendar months and includes children who are 7 months old up to when they turn 13 months (e.g. if a child is born on the 1st January 2021, they are 7 months old on 1st August and turn 13 months old on the 1st February 2022).

NICE’s Final Determination Document states that for this group:

"Children who are having more than 16 hours per day of non-invasive ventilation, or who have a tracheostomy, are not eligible.

The NMDT 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."