Zolgensma (also known as onasemnogene abeparvovec) is the gene therapy for SMA made by Novartis Gene Therapy
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 with SMA type 1, 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
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.