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