Zolgensma(onasemnogene abeparvovec)
Zolgensma (onasemnogene abeparvovec) is a gene pharmaceutical. Onasemnogene abeparvovec was first approved as Zolgensma on 2019-05-24. It is used to treat spinal muscular atrophy in the USA. It has been approved in Europe to treat spinal muscular atrophy.
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Commercial
Therapeutic Areas
Therapeutic Area | MeSH |
|---|---|
| nervous system diseases | D009422 |
Trade Name
FDA
EMA
Zolgensma
Drug Products
FDA
EMA
Reference product - 351(a)
Reference product - 351(a)
Interchangeable product - 351(k)
Interchangeable product - 351(k)
Biosimilar product - 351(k)
Biosimilar product - 351(k)
Labels
FDA
EMA
Brand Name | Status | Last Update |
|---|---|---|
| zolgensma | Biologic Licensing Application | 2025-02-05 |
Indications
FDA
EMA
Indication | Ontology | MeSH | ICD-10 |
|---|---|---|---|
| spinal muscular atrophy | EFO_0003823 | D009134 | G12.1 |
Agency Specific
FDA
EMA
Expiration | Code | ||
|---|---|---|---|
onasemnogene abeparvovec, Zolgensma, Novartis Gene Therapies, Inc. | |||
| 2031-05-24 | Reference product excl. | ||
| 2026-05-24 | Orphan excl. | ||
Patent Expiration
No data
HCPCS
Code | Description |
|---|---|
| J3399 | Injection, onasemnogene abeparvovec-xioi, per treatment, up to 5x10^15 vector genomes |
Clinical
Clinical Trials
23 clinical trials
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Indications Phases 4
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Spinal muscular atrophy | D009134 | EFO_0003823 | G12.1 | 3 | 1 | 9 | 2 | 8 | 23 |
| Muscular atrophy | D009133 | — | — | 3 | — | 9 | 1 | 7 | 20 |
| Atrophy | D001284 | — | — | 3 | — | 8 | 1 | 7 | 19 |
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Spinal muscular atrophies of childhood | D014897 | Orphanet_83419 | G12.1 | 1 | 1 | 3 | — | 1 | 6 |
| Genetic therapy | D015316 | — | — | — | — | 1 | — | — | 1 |
| Muscular diseases | D009135 | EFO_0002970 | G72.9 | — | — | 1 | — | — | 1 |
| X-linked bulbo-spinal atrophy | D055534 | Orphanet_139557 | — | — | — | 1 | — | — | 1 |
Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Neuromuscular manifestations | D020879 | — | — | — | 1 | — | — | — | 1 |
Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Healthy volunteers/patients | — | — | — | 1 | — | — | — | — | 1 |
Indications Without Phase
No data
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | Onasemnogene abeparvovec |
| INN | onasemnogene abeparvovec |
| Description | Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It works by providing a new copy of the SMN gene that produces the SMN protein.
|
| Classification | Gene |
| Drug class | — |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | — |
Identifiers
| PDB | — |
| CAS-ID | 1922968-73-7 |
| RxCUI | — |
| ChEMBL ID | CHEMBL4297240 |
| ChEBI ID | — |
| PubChem CID | — |
| DrugBank | DB15528 |
| UNII ID | MLU3LU3EVV (ChemIDplus, GSRS) |
Target
No data
Variants
No data
Financial
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Zolgensma – Novartis
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Tabular view
Estimated US medical usage
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Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 2,290 documents
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Safety
Black-box Warning
Black-box warning for: Zolgensma
Adverse Events
Top Adverse Reactions
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2,396 adverse events reported
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