NICE’s Guidance for NHS-Funded Access to Zolgensma Published

07 July 2021

Today, NICE’s recommendations for NHS-funded access to Zolgensma that were first announced in March, have now been published and finalised. These will continue to run in parallel to the additional agreement also made in March by NHS England. Thanks to the many stakeholders who have got us to this positive place. They read 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 type 1 SMA 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.

1.2 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.

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

There is further information about the decision on NICE’s website.

The MAA review will start in July 2022 when Novartis Gene Therapies should have data from their SPR1NT Trial.

While we're pleased this guidance has been published, no current evidence has been presented that Zolgensma could also provide health benefits for a proportion of children with SMA Type 2 and 3 and so we will continue to urge Novartis Gene Therapies to work to address this gap in access to treatment. 

Read the latest updates from the NMDT and NHS England, here.