It addresses the genetic cause of SMA by delivering a functional copy of the SMN1 gene into nerve cells. This gene is critical for the function of the nerves that control muscles.
On 7 July 2021 NICE published final guidance for the treatment, making it available to the following groups of children:
- Babies with SMA type 1 if they are 6 months or younger
- Babies aged 7 to 12 months if their treatment is agreed by the national multidisciplinary team.
- Pre-symptomatic babies identified as having SMA, up to three copies of the SMN2 gene if their treatment is agreed by the national multidisciplinary team
Children with SMA type 1 who are not included in the groups identified in the NICE recommendations above, but who fall within the scope if the European Medicines Agency (EMA) marketing authorisation for Zolgensma, may be considered for treatment with the drug. This may include:
- Children with SMA type 1 who are over 12 months old, but eligible within the EMA marketing authorisation based on their weight and other clinical factors
- Children with SMA type 1 who have been using treatment including Nusinersen (Spinraza) or Risdiplam (Evrysdi) and fall within the EMA marketing authorisation for Zolgensma.
More information on NICE’s guidance can be found here
Zolgensma was accepted for restricted use within NHS Scotland by the Scottish Medicines Consortium in March 2021. More information about its assessment can be found here.