ZOLGENSMA (Novartis Pharmaceuticals Australia Pty Ltd)
Product name
ZOLGENSMA
Date registered
Evaluation commenced
Decision date
Approval time
199 (255 working days)
Active ingredients
onasemnogene abeparvovec
Registration type
NCE/NBE
Indication
ZOLGENSMA (injection for intravenous infusion) (onasemnogene abeparvovec) is indicated for the treatment of paediatric patients less than 9 months of age with symptomatic or pre-symptomatic spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and 1 to 3 copies of the SMN2 gene.
Registration process
Orphan drug
Sponsors receive a fee waiver to help bring medicines for a small population to market.