Indication

For the treatment of:

  • patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene and a clinical diagnosis of SMA type 1, or
  • patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene

Medicine details

Medicine name:
onasemnogene abeparvovec (Zolgensma)
SMC ID:
SMC2311
Pharmaceutical company
Novartis Gene Therapies
BNF chapter
Central nervous system
Submission type
Full
Publication due date:
Q1 2021
SMC meeting date:
Q1 2021
Patient group submission deadline:
30 November 2020