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Zolgensma onasemnogene abeparvovec APPROVED
Drug Profile
ModalityGene therapy
RouteIV
Therapy AreaNeurology
Launch2019-05-24
US LOE2033-05-24
Peak Sales Est$1500M
Formulations[{"id":"zolgensma-iv","doses":"2.0x10^13 vg/kg","route":"IV","setting":"HOSPITAL","frequency":"Singl
Companies
NVS (ORIGINATOR)100%
Mechanism: SMN1 gene replacement
Expert: Adeno-associated virus serotype 9 vector delivering functional SMN1 gene
Everyday: Delivers a working copy of a gene to replace a faulty one
Targets: ["SMN1"]
Revenue History
PeriodRevenue ($M)
2024$1,320M
2025$1,280M
Q4 2024$340M
Q4 2025$310M
Programs (1)
IndicationStageKey StudyRegional Status
SMAAPPROVEDSTR1VE[{"stage":"APPROVED","region":"US","approval_date":"2019-05"}]
Notes
AAV9 gene therapy for SMA. One-time cure. Premium pricing.
Data from Supabase · Updated 2026-03-24